The creation of the Bespoke Gene Therapy Consortium (BGTC) in the US is welcome news, especially if it achieves its ambition to accelerate the delivery of more gene therapies for rare diseases. The new body is headed and funded by FDA and the National Institutes of Health (NIH), with other stakeholders including small biotechs, major pharma brands, and non-profit organizations.

One clear benefit is the chance to finally tackle the problem of affordability and return on investment. Today this presents a significant barrier for more niche therapies that are expensive and complex to develop and study, and whose target populations are small. If a standardized platform can be agreed upon, via which each drug developer can share findings and build on what’s gone before, the potential to reduce costs and accelerate approvals could be considerable.

The real beneficiaries, though, will be patients. If the BGTC achieves its aims, patients could see readier access to the breakthrough treatments they long for.

The new US consortium offers a glimpse of the role regulators can play to drive markets forward, as facilitators rather than merely gatekeepers. It paves the way for a shared platform that could help propel each company forward with new products.

The hope is that Europe will pick up the baton now. Take the known concept of “master files,” adopted by the EU (for vaccine antigens and plasma). If master files for gene therapy vectors were logged on a common platform, there could come a time when it’s possible to add a gene of interest for each new development, rather than details of all vector-related safety aspects.

Anything that would allow for greater convergence around clinical trial design, particular studies, tests/assays would be welcome, too—even the pooling of safety data—making advances in the treatment of rare diseases more viable.

Although it might take a while to establish an enabling platform, the hope for the future is that the BGTC and any equivalent consortia that emerge over time, will come up with a roadmap quite quickly to support at least some level of useful community cooperation.

We’re already seeing a growing push from pharma companies for a regulatory framework that better meets their needs. Up to now, for instance, each new product has needed to be vetted in its own right. This has presented a barrier for developers of new therapies with a limited safety database to work from—even if these have shared the same vector backbone. Tests showing the biodistribution and effectiveness of the transduction of cells, for example, would have to be repeated again and again.

There are some encouraging signs that regulators want to drive positive change. For one thing, they’re beginning to proactively hire people with expertise in advanced therapies so that they more confidently assess and approve new treatments. They’re also looking more holistically at data now, especially around the patient impact.

Ultimately, we all need to work toward a situation in which “advanced” therapies are viewed as standard treatments that we know how to regulate, so that patients suffering from rare diseases that affect their quality of life every day can dare to imagine a better future. The BGTC is a big step in the right direction.

Head of Biopharma Excellence and Chief Medical Officer (Biopharma), PharmaLex

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For biopharma innovation to succeed and accelerate, companies must stop duplicating their efforts and share valuable information.

New Bespoke Gene Therapy Consortium Is Welcome Development

On Sept. 23, 2021, six chief commercial officers (CCOs) joined 

 to discuss the future of sales and marketing in biopharma, a topic that is especially relevant due to the rate of acceleration brought on by the COVID-19 pandemic. While a healthy dose of weary change was already in progress within the industry over the past few years, March 2020 quickly ushered in a new way of fundamentally approaching the world and its stakeholders on all fronts. Panelists shared their insights and visions for delivering innovation and health care solutions in the future, while considering products, informed/empowered patients, healthcare professionals (HCPs), and payers. Topics discussed were aplenty with particular themes interweaving themselves throughout the discussion: playing catch-up to other industries, digital innovation, the rising value of patients’ perception, the nature and medium of communication, the changing face and approach of sales and marketing teams, and the importance of flexibility and agility. The event was moderated by Michael J. Hennessy Jr., president and CEO of MJH Life Sciences, 

Following are highlights of the conversation (answers have been edited for length and clarity): 

View the following video excerpts from this roundtable:

Sales Reps and Digital vs. In Person Approaches – Part 1

Sales Reps and Digital vs. In Person Approaches – Part 2

The Role of the Regional Marketing Manager

Preventative Medicine, Population Health, and Branding

As more data is available and patients become more empowered, the days of a one-size-fits-all advertising and marketing approach seem to no longer be sustainable. How does this new patient affect what the ideal biopharma company will look like in 2030, and how will it respond?

Lauren D’Angelo, Chief Commercial Officer, Alpha Cognition: 

We need to embrace the power of digital innovation and adopt a customer-first business model. Traditionally, we’ve been so focused on selling the features and benefits of our product. Now that patients are more aware of their health and focused on holistic health goals, it’s going to be more about expanding our current offerings of drugs and moving beyond just focusing on the product, and instead becoming an active partner in the patients’ management of their overall health. It’s more about what we can bring to the table and the role that we can play in the patient’s journey by focusing on continuous patient outcomes, versus only our product.

Kristin Yarema, PhD, Chief Commercial Officer, Atara

We’ve always thought about patients as one of the key customers in the healthcare system, along with physicians and payers. But we have to think about them as a more important customer, and our focus has to start much earlier.

We’re seeing openness from regulators; for example, with patient-focused drug discovery and development. There’s a real interest in understanding what patients want from their medications versus just what physicians want, or think patients want from their medications. And there’s an openness to build those kinds of patient-reported outcome measures, endpoints, and claims into development programs. I think we’re making a lot of progress there already, but it’s a somewhat new frontier and a tremendously exciting opportunity.

Mark Plinio, Chief Commercial Office, Evelo Biosciences, Inc.:

 I don’t think we’ve treated our patient populations as true customers. There are a lot of other industry examples of building customer loyalty, but pharma continues to lag. We should surround the patient with a customer-service approach that builds their loyalty over time. Patients are digesting information from so many different sources about various treatments, and they have a decision that goes beyond price. There are times when we make a product or service choice where we’re not just looking at the price—instead, we go to that brand because we trust it and trust the experience we’ve had in the past. Overall, I don’t think we do a good enough job in pharma of building an environment that builds trust and brand loyalty.

Todd Nichols, Senior Vice President, Chief Commercial Officer, Alkermes, Inc.:

 I don’t think we can predict five to 10 years out. If there is anything that the last 18 months have taught us, it’s that agility and flexibility right now are key. And one of the things that we have really learned at Alkermes especially is that patients are consuming information in various formats and modalities, and we’ve got to be extremely flexible right now.

In Alkermes’ space of serious mental illness and addiction, we spend a lot of time with caregivers. We believe in community activation. Our industry has had a black eye in the past, but some of the solutions that we’ve brought to the marketplace in the last 18 months have been phenomenal, and there’s a chance for us to build trust within these communities and with patients.

Paul Friel, Chief Commercial Officer, Beyondspring, Inc.:

 Other industries are so ahead of where we are today. Maybe it’s because of the present complexity or the regulatory environment, but I think the Amazons, Facebooks, and Apples of the world have shown us what can be done. I think our customers, patients, their families, their caregivers, and our physicians see that we are behind.

My very first role at a company was in consumer packaged goods at Procter & Gamble. Every discussion at those companies starts with “What customer did you talk to most recently? What were your insights from that conversation?” And every conversation started with grounding in the here and now with customers, their needs, and how they’re being met. If we can bring that mentality and those learnings from other industries, and leverage new technologies that allow us to interact with our patients in ways that just haven’t been possible before, that will be incredibly powerful.

 Our industry is the keeper of vast amounts of data. We invest in enormous amounts of research trying to understand the behaviors of our customers and patients, and we have to make sure that all of our future and new product planning feeds that data in. Our customers want options, and their patients want options. That’s the reason why our industry thrives. We’re going to have to be very deliberate to make sure that we’re allocating those resources appropriately to meet their needs.

What new steps will the industry be taking in both preventative medicine and population health, and how will that affect pharma branding in the future?

 As we look toward the next several years, we’re going to have to figure out how we make medicine more affordable for the broader population. I think every one of us on this call is probably involved in therapeutic areas where we have fairly high-priced drugs that have focused on a much smaller population of sufferers, typically the more severe or the rare. Over time, we’re going to need to be in a position where we are able to approach this differently and shift to a model that might be more volume-based.

This shift will also require different stances on the part of the payer. We’ve all been at the receiving end of the payer over time—many times price and availability drive a treatment choice that’s not necessarily right for the patient. I think the model is going to change, and we have to actively be part of that change, otherwise it will happen to us.

 I’ll pick up on the theme of affordability. How do our customers think about preventative medicine? Regardless of the therapeutic area, we’re seeing examples on a consistent basis of customers forming large partnerships. We all know the integrated delivery models, the models that have a payer involved. These are topics that are of big interest to our customers.

An encouraging trend we see now is that they’re building scale with various partnerships—there’s a void there that life sciences companies like ours can fill by showing we are sincere and able to talk about patients holistically, and not just about a single therapeutic option. We all know that patients need various options, they need access. One way to do it is through pricing models. The other way is to do it through value creation, such as disease-state education and so forth.

A recent survey by Accenture found that 87% of healthcare professionals said they prefer all digital, or a mix of in-person and digital interaction with sales reps once this pandemic comes to an end. How do you see the role of the sales rep evolving over time, and what steps will your organization take to begin to make some of these changes?

 The traditional pharma sales model is still relevant; it just needs some revamping for the modern age. That means investing more time and resources into all things digital. Instead of relying just on sales reps or just on digital-promoting channels, what pharma needs is a healthy mix of both.

The salesforce will need to move away from a one-size-fits-all communication approach and be able to adapt to varying customer needs. I think the industry is going to need more tech-savvy people who know how to build brands, and who can accommodate individual customer needs based on a particular physician or stakeholder. And then they will have to deliver messaging using hybrid methods and virtual tools. But at the end of the day, you can’t copy and paste a real in-person relationship into a digital environment. We all know the value of relationships and that having that one-on-one, in-person contact is the best way to sustain them.

 You are spot on, Lauren. I still believe the single biggest catalyst to affect behavior change is the sales representative who is there in the office, who is building relationships and trust by bringing valuable product information and disease education. This is not only accomplished through the one-on-one interaction with the physician but also with the entire office.

That being said, because there is a large and growing subset of physicians who are digital savvy, it can change our targeting a little bit because we’re able to accumulate digital affinity scoring for these particular physicians over a period of time. You can actually track how active they are online from their shopping, surfing, and overall engagement. As a result, you can support a hybrid approach by building a target base of physicians who would be more accessible to receiving information digitally.

There’s one example I would like to point out about technology—the speaker programs. We all talk about how much more engaging this used to be pre-COVID—the old steak dinner where everybody shows up and you fly an expert in from far away. Now you can take that top expert and have them virtually join for lunch, breakfast, or dinner. To me, that is a perfect example of something that changed that will permanently change going forward.

Sean Mcauliffe, Chief Commercial Officer, Taysha Gene Therapies:

 It is really disease-state specific. My last experience was in rare disease. We had different roles—reimbursement, field reimbursement, sales—all based on a good rationale from a compliance standpoint; but, when there was a case, we had a lot of people react. There were times it reminded me of little kids playing soccer where everyone is hovering around the ball. It was less efficient and ultimately, in some cases, we may have been able to save some time. So managing these interactions is going to be like steering the Titanic, but I think it starts with how companies leverage the data we now have access to. It will be a hybrid of some kind, and it is going to be disease-state and patient-group specific.

The beauty of being so connected as an industry right now is that we can see best practices. I think the model will continue to evolve and adapt. Do I see wholesale changes? Not really. Even with data, the key insight is that HCP customers are saying they value the interaction. They still value new engagement and education that life sciences companies bring to them. They’re just asking for different mediums to receive the information.

Todd, you mentioned earlier the need to be flexible. The last product that I launched was during COVID. One of the things we made a quick decision on right away was not to force a particular technology on a physician. There was a lot of conversation internally, especially from a legal and compliance perspective, but at the end of the day, it comes down to what the customer is comfortable using: Do they use Zoom? Do they use Teams? Are they comfortable using Veeva? If you push one platform onto a customer, it will be an uphill battle that I don’t think any of us wants to fight. It’s getting the organization aligned to say, “You know what, we’ve got to deliver our messaging using a technology that our customers are comfortable using in a compliant way, or else we’re not going to be effective.”

One trend that I have seen kind of come, go, and come again is the regional marketing manager. Are there any field or marketing roles that you believe need to evolve or simply disappear?

 In some of the therapeutic areas I’ve been in, like gastrointestinal (GI) and dermatology, we’ve started to see a consolidation of practices, whether it’s the IDNs of the world or large practices. There are some GI practices out there with close to 1,000 physicians, and they’re managing a significant patient population within a geographic area.

As therapeutic areas start to evolve, you’re seeing private equity coming in. I think there will be an increasing move toward that with consolidation. From a pharma perspective, whether you are in biotech, medium size, or large pharma, we need to recognize how the business is evolving, how therapeutic areas are going to be highly focused and concentrated in such a way that you’re going to need people looking after these large practices as key decision makers. This is because, as they grow over time, these practices may become decision-makers on treatment availability for certain geographies.

In my last role, we started to build out the capabilities of managing the business from a field perspective with individuals focused on understanding the insights of those large practices. We need to ask: What are the educational needs, what are the therapeutic needs, and what were their interests from a business perspective? What are they trying to accomplish overall from a health perspective for their constituencies? You’ve got to take an approach that looks at the needs of a group that is really going to drive behavior, inform drug selection and how patients are treated from a patient treatment pathway, and influence overall pharmaceutical approach. As a result, you may need representatives focused on various group practices, because they are all operating a little bit differently.

In transplant centers, you already have that dynamic happening. Every single transplant center is unique and they all have different protocols and HCP specialties involved in the decision-making. Decision-making regarding what’s going on formulary, and what protocols and algorithms will be embedded, is at a highly organized level. It requires a completely different sort of interaction and more of a team approach. It’s not just the commercial people, but our partners in medical, for example, also have an important role to play, whether it’s an institution or what we would more normally think of as an organized customer group. That’s an area where we’re going to continue to see the consolidation, and where we need to evolve our capabilities and these roles.

This is a really important topic. There was a rush years ago where the theme was that the commercial models were broken and needed to change. So there was a rush in commercial organizations to create highly specialized roles, and then it morphed into various therapeutic areas. We’re at the place now where we have to question that and say, “Do you really need highly specialized roles in every therapeutic area based upon your customer base? Do customers require that?” A key question that I try to ask myself and that we work on at Alkermes is: Is it really the role, or the capability, or the behavior that we’re trying to adapt to? It’s a different question, which comes up with a different answer. So yes, in certain therapeutic areas, you’re going to have to have really specific roles to address certain customers, but I don’t think you need that in every single therapeutic area.

The days of having separate people just doing market access and reimbursement probably is a little bit dated as well. You always need good professionals that engage with payers and so forth, but that’s a competency and skill that your commercial teams have to have now or you’re not going to be relevant in the marketplace to your customer. So I think about it in that domain right now as the capability, and then Kristin, you said it as well, this notion of collaboration about behavior. We all work in a great industry, good organizations as well; collaboration across our companies is really important—the right type of collaboration with the medical teams, the internal teams, and the field-facing team. That’s actually critical now, and that’s a capability we have to continue to build.

What steps do tomorrow’s chief commercial officers need to take to ensure that their companies will be ready for the future we’ve discussed here today?

 One of the first required steps is probably going back to Lauren’s point. It’s around structure both from a marketing, reimbursement, and field-facing team point of view. It is going to require, at least in some of the smaller indications, a closer collaboration and a better way of working with medical. A great deal of that starts with making sure data is managed and leveraged appropriately. Data acquisition, and management and movement of that data through an organization, will be critical.

 People are going to be key, too. What we’ve talked about is we don’t know what it’s going to look like in five or 10 years, and you’re going to have to have people that are able to adapt, overcome challenges, learn, and grow. We don’t know what the solution is, but we know we need people that are going to have a unique skillset to be able to do that as we continue forward.

 And to be flexible about how we’re looking at talent. There are lots of conversations across all of our companies right now about what it means to be in-house anymore, because in-house might be remote in a different state. We’ve learned so much about how to collaborate and work effectively during COVID, so what will the norms and expectations be going forward? How much of our workforce will we plan to be permanently remote? How are we going to develop those people and provide them with career opportunities that are on a level playing field with people who might be back in the office and closer to the managers, for example?

It’s always been important, but now more than ever, it is identifying people with the right learning agility, engaging them, and then offering them opportunities to grow new skills, whether it’s in digital or the flexibility in how to think, or learning more market access if they come from a traditional commercial field background, for example. But the investment in people is what I think every CCO really needs to have at the top of their radar screen.

 When we get talent into the organization, we’ve got to focus on building a culture of innovation. Because you can get the best people, but if we don’t enable them to learn, to make mistakes, allow them to experiment, then we are wasting all of their talents and potential. To me, innovation is a biproduct of culture. I know that culture is this big word that we all talk about as leaders, but it’s really very simple: Attract the right talent, and then provide them with an environment where they can thrive.

is a Senior Editor for Pharm Exec. He can be reached at

Features

Chief commercial officers gather for an insightful roundtable discussion anchored in the future of sales and marketing in light of new communication media brought on by the pandemic.

The Future of Sales and Marketing: How to Deliver Information in the Era of COVID-19

Diseases shape history. They shape lives. The emerging drugs in the development pipeline show promising results in treating some of the most virulent ailments in modern society. Moreover, these new drugs are the end result of accelerating innovations in next-generation pharmaceutical technologies that aim to change the face of medicine. Emerging products like phage therapies, biologics, gene therapies, monoclonal antibodies, and next-generation anti-infection/anti-microbial agents are opening up new opportunities in verticals like pain management, ophthalmology, infection control, fatty liver disease, and Alzheimer’s disease.

The drug development pipeline is filled with promising new products, and several of them are close to commercialization. These drugs are aiming to reshape the course of history; the ones that succeed will serve as best-in-class products for pharmaceutical companies that embrace innovation.

Several of these new products, like long-acting GLP-1 analogues and anti-beta amyloid monoclonal antibodies, are in Phase III trials and could be ready for commercialization shortly. Other experimental therapies, like phage-based antimicrobials and intravitreal injectable drugs for ophthalmological conditions, are winding their way through preclinical and Phase I studies.

’s 17th Annual Pipeline Report showcases the most promising and innovative new therapies under development, with data provided by Clarivate Analytics.

The search for an Alzheimer’s breakthrough

Alzheimer’s drugs have pivoted in recent years as the working theory of the disease evolves. Clarivate’s data shows that from 2000 to 2010, the most common biomarker pursued in drug studies was amyloid beta A4 protein. Over the past 11 years, though, this has shifted. There is significant new interest in microtubule-associated protein tau, with 286 clinical trials tracking that biomarker since 2010—a 13-fold increase over the previous time period.

This shift is taking place at the same time that aducanumab, a monoclonal antibody that targets amyloid beta plaque in the brain, faces ongoing controversies and challenges. FDA approved aducanumab, under the brand name Aduhelm, in June 2021 over the fierce objections of its own advisory committee members. Harvard Medical School professor and former FDA advisory committee member Aaron Kesselheim, MD, JD, called the Aduhelm approval “probably the worst drug approval decision in recent US history.”

Since its approval, Aduhelm has underperformed relative to expectations. Despite being the first Alzheimer’s drug to gain FDA approval since 2003, the drug amassed only $300,000 in sales in its first three months on the market. Analysts had expected the drug to earn $14 million in Q3-2021. Some experts have questioned whether the drug offers clinical benefits that justify its risk of side effects like cerebral hemorrhaging.

Michael Kleinrock, lead research director at IQVIA, says the problem with aducanumab and other drugs like it is that there is only a very limited correlation between amyloid plaque reduction and an observable endpoint. That’s why, he says, the bulk of the Alzheimer’s pipeline is examining other pathways. While some plaque-targeting drugs, like lecanemab,

 gantenumerab, and solanezumab, continue to undergo Phase II and Phase III trials, there is also growing interest in drugs that treat other aspects of Alzheimer’s.

Mike Ward, head of global thought leadership for Clarivate in London, says the growth of research activity in Alzheimer’s drugs is cause for optimism. He notes that a successful pharmaceutical treatment that could meaningfully prevent cognitive decline would be not only a commercial success but a social one: “The economic cost of Alzheimer’s disease is a drain on family resources. Caregivers are often close family members. There would be a tremendous benefit, both at the individual level and the society-wide level, if there were a meaningful breakthrough that could stop cognitive decline.”

The goal of stopping cognitive decline is driving diversification in the types of drugs under development, several of which are disease-modifying small-molecule drugs and biologics. Disease-modifying biologics like the PKC agonist bryostatin-1 and the peptide GV1001 are in Phase II trials; disease-modifying small-molecule drugs like GV-971 and NE3107 have progressed to Phase III studies.

 In the case of GV-971, an oligosaccharide compound derived from brown algae, a Phase III trial published in 2021 showed that treatment with GV-971 caused a statistically significant improvement in patients’ ADAS-Cog-12 scores compared to placebo, and the drug was found to be safe and well tolerated.

Meanwhile, drugs like troriluzole and tricaprilin are under investigation for their potential to work on the nervous system level to modulate disease symptoms. Troriluzole, a glutamate modulator from Biohaven Pharmaceuticals, is undergoing Phase II and Phase III studies for a variety of psychiatric and neurodegenerative disorders. While troriluzole has experienced struggles in trials,

 Biohaven is expected to continue studying the drug for use in patients with mild Alzheimer’s.

Tricaprilin, in contrast, received a boost in October 2021 when manufacturer Cerecin, formerly Accera, announced a new $40 million round of financing for pipeline development.

 Tricaprilin is a medium-chain triglyceride that promotes mitochondrial metabolism and induces ketosis. In 2013, Accera ran afoul of FDA with its branded tricaprilin product, Axona, which the company had been marketing as a medical food. FDA determined that Axona did not fit the legal definition of a medical food under the Federal Food, Drug, and Cosmetic Act. However, Cerecin has continued to develop Axona in the form of a pharmaceutical drug under the developmental name AC-1204.

One Phase III trial, completed in August 2021, showed that tricaprilin caused a 0.6-point improvement in ADAS-Cog11 scores after 26 weeks, while the placebo did not change scores from baseline. The study authors determined this difference was not statistically significant, but hypothesize that the failure to prove efficacy may have resulted from a lower-than-expected level of ketone production and the placebo group’s better-than-expected performance.

 A new formulation of the drug, code named AC-SD-03, will be undergoing further Phase III trials in 2022.

Several of the other Alzheimer’s candidates under development are symptom-reducing drugs that target specific cognitive, emotional, or behavioral symptoms of Alzheimer’s, with the aim of managing symptoms like agitation, loss of awareness, and sleep disturbances.

Nabilone, a synthetic cannabinoid and THC biosimilar currently approved for chemotherapy-induced nausea, is undergoing clinical trials to determine if it can treat agitation in Alzheimer’s patients. It has been found to be moderately effective at reducing Cohen-Mansfield Agitation Inventory (CMAI) scores. The drug also decreased overall neuropsychiatric symptoms and caregiver distress, improved cognition, and was more effective at reducing agitation than atypical antipsychotics.

 This is an important breakthrough given the recent failures of other symptom-reducing medications in trials.

The tetracyclic antidepressant mirtazapine, an alpha-1 antagonist, had previously been recommended as a treatment for Alzheimer’s-associated agitation and mood disturbances.

 However, recent research is questioning mirtazapine’s value in Alzheimer’s treatment. A 2021 trial on 204 patients with probable Alzheimer’s that did not respond to non-pharmaceutical intervention found that a 45mg-daily dose of mirtazapine did not significantly change scores on the CMAI relative to placebo after 12 weeks. There were also seven deaths in the treatment group, compared to one in the control group.

Meanwhile, a Phase III trial of azeliragon, a small-molecule RAGE inhibitor under development by vTv Therapeutics, was terminated in May 2021 after a failure to demonstrate efficacy at the 5mg dose.

 Azeliragon had previously shown efficacy in animal trials.

 Manufacturer vTv Therapeutics of High Point, N.C., has since entered into a licensing agreement with Cantex Pharmaceuticals to continue developing azeliragon as a cancer drug.

Ophthalmology development accelerates; gene therapy dominant

Drug development for ocular diseases has grown significantly in recent years. Data provided by Clarivate shows that the number of clinical trials for ophthalmology drugs and drugs targeting ocular disease has grown by a factor of four over the past 20 years. From Jan. 1, 2000, to Jan. 1, 2010, there were 2,608 clinical trials performed on ophthalmology drugs. From Jan. 1, 2010, to present, Clarivate’s data show 10,984 trials, an increase of 421%.

The major areas of development in ophthalmology drugs since 2010 have been glaucoma, ocular hypertension, and wet age-related macular degeneration (AMD), with an emphasis on modifying physiological biomarkers of disease. From 2010 to present, 4,234 trials involved small-molecule drugs, making small-molecule pharmaceuticals the dominant type of drug under development. During that same time period, there were 3,570 trials involving medical devices, 2,490 trials involving biologics, and 2,229 trials involving medical procedures. Of these more than 10,000 trials, 4,401 did not have an FDA-defined phase, while 1,833 are Phase II trials and 1,200 are Phase IV post-marketing surveillance trials. There are over 1,200 ophthalmology drugs that have entered Phase III trials since 2010, compared to 418 in the decade prior.

In September 2021, AbbVie signed a deal with Regenxbio to jointly develop and bring to market a single-treatment gene therapy for wet AMD and diabetic retinopathy called RGX-314. This gene therapy encodes an antibody fragment via the NAV AAV8 vector to inhibit the activity of vascular endothelial growth factor (VEGF). RGX-314 is designed to prevent fluid buildup in the retina by inhibiting blood vessel growth and leakage.

In October 2021, Regenxbio announced initial positive results from the Phase II ALTITUDE trial, which is investigating RGX-314 as a potential treatment for diabetic retinopathy. After three months, five of the 15 patients in the treatment group experienced at least a two-step improvement on the Early Treatment Diabetic Retinopathy Study-Diabetic Retinopathy Severity Scale (ETDRS-DRSS).

Drug companies are also looking at monoclonal antibodies for potential use in the ophthalmology market. Immunovant has been investigating its fully human FcRn monoclonal antibody IMVT-1401, formerly RVT-1401, as a potential treatment for myasthenia gravis (MG), a neuromuscular disease that can affect the muscles around the eyes, and thyroid eye disease. The company experienced a setback in February 2021 after subjects in the ASCEND GO-2 Phase IIb trial for thyroid eye disease exhibited an increase in total cholesterol and LDL levels. Later, in June 2021, Immunovant announced that it would continue developing IMVT-1401 at a lower dosage level. In July 2021, IMVT-1401 received Orphan Drug Designation for MG.

In October 2020, Novartis acquired Vedere Bio, a startup developing therapies that optimize surviving photoreceptors after vision loss. Vedere founder Cyrus D. Mozayeni, MD, launched Vedere Bio II, a sequel company developing a viral vector treatment for retinitis pigmentosa, an inherited form of vision loss. In May 2021, Vedere Bio II received $77 million in Series A funding in a financing round led by Octagon Capital.

Meanwhile, Visus Therapeutics has several candidates for multiple conditions in its pipeline. Brimochol, a combination of carbachol and brimonide tartrate administered in eye drops, is undergoing Phase II trials for treatment of presbyopia. Carbachol is a cholinergic miotic agent that constricts the pupils, while brominidine tartrate is a sympatholytic alpha-2 agonist that prevents the pupils from dilating. Five previous clinical studies have shown that Brimochol improves near-visual acuity. Visus Therapeutics’ four other candidates for the treatment of various ophthalmological conditions are currently in preclinical research. In August 2021, the company secured $20 million in financing from LSP and Sage partners. In November, it announced the appointment of David Guyer, MD, as chairman. Guyer formerly served as chair of the department of ophthalmology at the New York University School of Medicine and was the first person to develop and commercialize an anti-VEGF drug for macular degeneration.

NASH drugs navigate scientific and commercial challenges

Drugs for non-alcoholic steatohepatitis (NASH), the most severe form of fatty liver disease, have a history of struggles. In the Phase III REGENERATE study, obeticholic acid showed mixed results. FDA’s approval of obeticholic acid for treatment of primary biliary cholangitis in 2016 was regarded as a sign of hope that the drug might also be effective against NASH; the REGENERATE trial dashed those hopes when it found that the drug failed to resolve NASH after 18 months. While obeticholic acid did cause an improvement in fibrosis symptoms, this effect was only observed at the highest dose of 25mg/day.

Other clinical trials of first-generation NASH drugs have also rendered disappointing results. In the RESOLVE-IT Phase III trial, GENFIT’s drug elafibranor did not resolve NASH without exacerbating fibrosis even after 72 weeks. In the STELLAR-3 and STELLAR-4 trials, selonsertib, a NASH candidate and ASK1 inhibitor developed by Gilead, did not result in statistically significant fibrosis improvements without exacerbating NASH symptoms after 48 weeks.

“The trials have underperformed,” says Kleinrock. “It’s a challenging dynamic to create a drug that has a response without side effects. If you think about the ecosystem surrounding NASH, these patients have diabetes and high cholesterol, maybe even metabolic syndrome. There’s some comorbidity with hypothyroidism and fatty liver disease. So it’s an interesting conundrum to come up with a drug that targets NASH.”

Now, though, a new generation of NASH drugs is winding its way through the research pipeline. Several new candidates are currently in Phase II and Phase III trials. Data provided by Clarivate shows that 363 NASH drug trials have taken place since Jan. 1, 2017; from 2000 to 2010, there were only 82 trials. The majority of NASH trials have historically involved small-molecule drugs. From 2010 to present, the most common biomarker assessed in trials was alanine transaminase, followed by aspartate aminotransferase and glucose.

Kleinrock says that some of the emerging new drugs are beginning to show efficacy. Resmetirom (MGL-3196), for instance, has shown strong results in Phase II and Phase III trials. Resmetirom is a first-in-class, orally administered, small-molecule, liver-directed thyroid hormone receptor selective agonist produced by Madrigal Pharmaceuticals. The drug is undergoing two Phase III trials, the MAESTRO- NAFLD-1 trial for non-alcoholic fatty liver disease and the MAESTRO-NASH trial for NASH. The MAESTRO-NASH trial, involving 2,000 participants, is expected to end in March 2024.

 The MAESTRO-NAFLD-1 trial, involving 700 participants, will compare the effects of 80mg/day and 100mg/day of resmetirom vs. placebo among the blind participants and evaluate resmetirom’s efficacy at reducing biomarkers of NAFLD. Study completion is scheduled for December 2021.

In September 2021, Galecto announced that it had commenced a Phase Ib/IIa trial investigating its galectin-3 inhibitor, GB1211, as a potential treatment for liver cirrhosis, a complication of advanced NASH. Initial data is expected in mid- to late 2022. AngioLab, a South Korean company, is developing an angiogenesis inhibitor made from lemon balm extract called ALS-L1023. The company published the results of an animal trial on ALS-L1023 in 2017; in September 2021, AngioLab completed a Phase IIa trial on ALS-L1023 for NASH. Also in September, Oramed Pharmaceuticals announced its Phase II trial of its NASH candidate, an oral insulin capsule developmentally coded ORMD-0801, had surpassed 50% enrollment.

Some manufacturers are working on combination therapies—Allergan’s cenicriviroc and Novartis’ tropifexor—as possible NASH treatments, which have both separately received FDA fast-track status for NASH with liver fibrosis.

Cenicriviroc, which is also an experimental candidate undergoing study as a possible HIV and COVID-19 treatment, is a CCR2/CCR5 inhibitor. However, the Phase III AURORA trial on cenicriviroc was terminated early in April 2021 when the study’s initial results failed to show efficacy.

Tropifexor, a farnesoid X receptor agonist, was found to be effective at improving biomarkers of NASH like alanine aminotransferase, body weight, and hepatic fat fraction after 12 weeks, in Novartis’ Phase IIb FLIGHT-FXR study.

Now, Novartis and Allergan are co-sponsoring a new Phase II randomized, double-blind, parallel-assignment trial to determine whether a combination of tropifexor and cenicriviroc is safe and effective in improving fibrosis or resolving NASH after 48 weeks. This trial, involving 193 participants, submitted results to the National Library of Medicine in October 2021 and is undergoing quality control review.

Ionis Pharmaceuticals has two NASH candidates in its research pipeline, both of which are built on its antisense oligonucleotide technology. Ionis’ antisense therapies bind with mRNA to prevent the formation of disease-associated proteins. ION224 and ION839 are second-plus generation LICA medications designed to prevent the accumulation of liver fat by inhibiting diacylglycerol acyltransferase 2 and patatin-like phospholipase domain-containing 3 protein, respectively. Ionis is currently recruiting subjects for a Phase II trial of ION224. ION839, formerly AZD2693, was licensed to AstraZeneca in April 2018 and is currently undergoing a Phase I trial.

Another emerging NASH candidate is a repurposed drug first developed in 2012. Semaglutide is a GLP-1 analog that was originally approved as a treatment for diabetes in 2019. Earlier this year, the FDA approved Novo-Nordisk’s Wegovy (semaglutide) for chronic weight management. A 2021 Phase II trial on 320 patients with NASH and hepatic fibrosis examined whether a once-daily subcutaneous injection of 0.1mg, 0.2mg, or 0.4mg of semaglutide could resolve NASH without worsening fibrosis. Conducted by Novo Nordisk, the study found that at 72 weeks, 59% of subjects in the highest-dose semaglutide group achieved NASH resolution without worsening fibrosis, compared to 17% in the placebo group. The 0.2mg and 0.1mg doses achieved 36% and 40% NASH resolution, respectively.

Other NASH drugs in development include Vonafexor, lanifibranor, and oltipraz. In July 2021, ENYO Pharma’s Phase IIA LIFIVY trial found that Vonafexor resulted in a 6.3% absolute liver fat reduction in the 100mg group and a 5.4% reduction in the 200mg group. The differences between placebo and Vonafexor were found to be statistically significant.

 A Phase IIb trial showed that high-dose lanifibranor caused resolution of NASH without worsening fibrosis in 49% of subjects. Low-dose lanifibranor achieved the same outcome in 39% of subjects, while 22% of subjects receiving a placebo achieved NASH resolution without worsening fibrosis.

 A Phase III trial on oltipraz by South Korean company Pharmaking was estimated to be completed in October 2021.

The anti-infection/anti-microbial category is seeing significant innovation, with non-traditional products in all stages of development. While large pharmaceutical giants have mostly exited this market, a new crop of emerging biotech startups has recognized a need for more anti-infection drugs and is actively developing innovative solutions. A March 2021 research report by Pew Charitable Trusts found that over 95% of anti-infection drugs in the pipeline are being developed by startups, and over 70% of these companies are pre-revenue.

Kleinrock says that in anti-infection drugs, there has been a fundamental market failure in encouraging the amount of innovation required to solve the problems facing the niche. He notes that companies are still attempting to solve the problem of antimicrobial resistance, and in the meantime, the stopgap solution is to encourage less usage.

Despite the challenges that anti-infection drug development has historically faced, the pharma industry is beginning to take notice of antibiotic resistance. In July 2020, the World Health Organization and 20 of the world’s largest drug companies launched the AMR Action Fund, a biotechnology investment fund that intends to invest over $1 billion in biotech startups that are pursuing new antibiotics. The fund’s stated goal is to bring two to four new antibiotic drugs to market by 2030.

One of the areas of anti-infection drug development seeing significant research and investment is phage therapies. Several emerging biotech startups are developing phages that can solve the antimicrobial resistance challenge. Felix Biotechnology received a Phase I grant from the National Science Foundation in September 2021 to continue development of a machine learning platform that can match phages to the pathogens they are most effective against.

Armata Pharmaceuticals is developing multiple synthetic phage candidates. AP-PA02, a synthetic phage cocktail engineered to target P. aeruginosa, is under development for the treatment of respiratory infections in cystic fibrosis patients. AP-PA02 is the second generation of Armata’s initial phage product, AP-PA01. AP-PA01 showed favorable results in two case reports chronicled in 

 American Journal of Respiratory and Critical Care Medicine

 Armata also is developing phage therapies for cystic fibrosis, pneumonia, bacteremia, and prosthetic joint infection.

Other phage candidates are in clinical development by Adaptive Phage Therapeutics. The company’s PhageBank library of phages has already received emergency IND authorization from FDA for use in treating diabetic foot osteomyelitis, prosthetic joint infection, chronic recurrent urinary tract infection, and cystic fibrosis-related lung infection. PhageBank also has emergency use Expanded Access IND status for treatment of secondary bacteremia in COVID-19 patients.

In September 2021, Adaptive Phage Therapeutics announced the results of a case study involving a 7-year-old with chronic, antibiotic-resistant P. aeruginosa infection following a car accident injury. The infection, located in the patient’s lower leg, continued to grow after surgical debridement and hardware removal. Seven weeks after treatment completion, the patient was able to bear weight without pain for the first time since the accident. At a five-month follow-up, the patient had no additional symptoms of bacterial infection.

While phages are a compelling novel treatment, further research is required in order to make them viable. Kleinrock notes, “Phages are interesting because, conceptually, it’s a cool way to think about how we’re going to crack that nut, but I want to see more than one of them get past a certain level. Phase Ib and Phase II trials means we’re starting to figure out dosing and get a clinical effect.”

Some of the emerging anti-infection drugs that are currently undergoing Phase II and Phase III trials include omadacycline, AXC-362E, and fidaxomicin. Omadacycline is a tetracycline broad spectrum antibiotic in the aminomethylcycline subclass. Paratek Pharmaceuticals is developing omadacycline under the brand name NUZYRA as a treatment for non-tuberculous mycobacteria and pulmonary anthrax. NUZYRA is already approved for treatment of acute bacterial skin and skin structure infections, as well as community-acquired bacterial pneumonia. Paratek developed this form of tetracycline to overcome biological mechanisms that result in antibiotic resistance, and it has already shown efficacy against MRSA and drug-resistant Streptococcus pneumonia in trials.

Another unique drug is quickly making its way through the pipeline. Acurx Pharmaceuticals is developing ibezapolstat, a small-molecule bacterial DNA synthesis inhibitor. The drug is the first in a new class of Pol IIIC inhibitors for treatment of C. difficile. It has received a fast-track designation from FDA and has completed Phase IIa trials. Ibezapolstat is now in a Phase IIb trial slated for completion in March 2022. The company’s Phase IIa data showed that ibezapolstat had a 100% cure rate and eradicated colonic C. difficile within three days.

Pain management drugs to end the opioid crisis

According to the National Institute on Drug Abuse, almost 50,000 Americans died of opioid overdoses in 2019. Despite the dangers of opioid addiction, the CDC reported in 2018 that an estimated 50 million Americans suffer from some type of chronic pain. The need for safe and effective pain management drugs still exists. That’s why much of the innovation in pain management involves engineering a pain medication that can provide opioid-level relief without the risk of addiction.

Kleinrock says the majority of development activity in pain drugs is in non-opioids. IQVIA’s research indicates that in December 2020, there were five opioid-based pain management drugs in development, compared to roughly 100 to 120 non-opioid analgesics. Kleinrock says that as of December 2020, there were also roughly 300 drugs in development designed to treat underlying diseases that produce pain as a symptom. Kleinrock says that while only a small subset of those drugs will complete Phase III trials, the diversity of approaches in drug types and the number of candidates in development are both reasons for optimism.

“I am very conscious of the significant amount of failures over the pipeline,” Kleinrock says. “But I tend to reason that if there’s a lot of activity in multiple mechanisms, with multiple companies looking at the same models, it’s more likely to make it through. There are some promising antibody and RNA approaches.”

The next-generation pain management drug that is closest to commercialization is mirogabalin, a gabapentinoid and alpha-2-delta ligand developed by Daiichi Sankyo. Daiichi Sankyo sells the drug under the brand name Tarlige in Japan, where it was approved in January 2019. The drug’s Japanese approval is for the treatment of peripheral neuropathic pain, diabetic peripheral neuropathic pain, and postherpetic neuralgia. In May 2021, Daiichi Sankyo submitted a supplementary new drug application to the Japanese regulator to begin selling Tarlige for treatment of central neuropathic pain.

The US National Library of Medicine database shows 11 completed studies on mirogabalin. While the drug was previously under clinical development in the United States for the treatment of fibromyalgia pain, development was halted in the US and the European Union after mirogabalin failed to meet its primary endpoint in Phase III trials. However, new data shows that mirogabalin may be effective at treating specific types of neuropathic pain. A 2021 systematic review and meta-analysis of three randomized controlled trials with patients with diabetic peripheral neuropathic pain (DPNP) found that mirogabalin significantly reduced average daily pain scores (ADPS) relative to placebo. Mirogabalin also outperformed pregabalin after three, four, and five weeks.

Some of the most revolutionary developments in pain management medicine are still in preclinical research. In August 2021, the 

Proceedings of the National Academy of Sciences

 published a mouse model study that determined that HMGB1 is a necessary component in the process leading to the inflammatory response.

 A 2019 literature review also found that a monoclonal antibody treatment targeting HMGB1 (HMGB1-nAB) has shown promise treating various CNS and PNS diseases, including neuropathic pain, in a variety of animal models.

 A subsequent literature review published in January 2021 found that HMGB1 is responsible for the development of neuropathic pain in patients with chemotherapy-induced peripheral neuropathy, which the authors say is resistant to most conventional pain medications.

Some of the other emerging pain management candidates in development include AT-121, ML-351, RTI-5152-12, and a Nav1.7 gene therapy. All of these potential therapies are in preclinical research.

Strong science, creative thinking, and iteration fuel breakthroughs

The 2022 drug development pipeline is full of promising candidates in multiple stages of research and development, as well as some disappointing setbacks and failures. Several of these new pharmaceuticals, like phages, are first-in-class biologics that continue to undergo Phase Ib and Phase II studies. While these innovative products are likely 10 or more years away from commercialization, their creative approach holds promise in solving the challenge of antibacterial resistance. Other drugs, like resmetirom and AVP-786, have advanced to Phase III trials and are beginning to show efficacy in treating historically challenging conditions. Monoclonal antibodies are attracting interest for a variety of conditions like pain and ophthalmological conditions, with formulations at various stages of development in preclinical and clinical research. While several of these products still have many miles left to go on the long road to market approval, there are enough players in the industry ushering them along that for at least some of them, clinical and commercial success is inevitable.

Mike Straus is a freelance writer based in British Columbia, Canada. His work has appeared in Nutritional Outlook, Hoist, and Massage Therapy Canada, among other publications.

Chappell B. “3 experts have resigned from an FDA committee over Alzheimer’s drug approval.” 

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Cummings J et al. “Alzheimer’s disease drug development pipeline: 2021.” 

Alzheimer’s & Dementia Translational Research & Clinical Interventions

, vol. 7 no. 1 (2021): e12179. Published online May 25, 2021.

Xiao S et al. “A 36-week multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase 3 clinical trial of sodium oligomannate for mild-to-moderate Alzheimer’s disease.” 

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Cerecin. “Cerecin raises $40 million Series II and IIA financing to support its programs in neurological disorders.” Published online Oct. 19, 2021.

Henderson ST et al. “A placebo-controlled, parallel-group, randomized clinical trial of AC-1204 in mild-to-moderate Alzheimer’s disease.” 

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Yang L et al. “Azeliragon ameliorates Alzheimer’s disease via the Janus tyrosine kinase and signal transducer and activator of transcription signaling pathway.”

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Lavitas P. “First wave of novel drugs for NASH does not live up to expectations, Clinical Pharmacy Service’s pipeline monitoring team finds.” 

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Nature Reviews Gastroenterology & Hepatology

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US National Library of Medicine. “A phase 3 study to evaluate the safety and biomarkers of resmetirom (MGL-3196) in non alcoholic fatty liver disease (NAFLD) patients (MAESTRO-NAFLD1).” ClinicalTrials.gov Identifier: NCT04197479

Oramed. “Oramed’s Phase 2 oral insulin NASH trial reaches over 50% enrollment.” Published online Sept. 15, 2021.

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Our annual report takes a look at pharma’s current investment in drug development, which this year demonstrates a focus on five important therapeutic areas of interest—Alzheimer’s disease, ophthalmology, NASH, anti-infectives, and opioids.

2022 Pipeline Report: Capitalizing on Opportunity

After finishing medical school in Egypt, Mohamed Zaki came to the United States where he gained a PhD at the University of Pennsylvania, before becoming an assistant professor. Moving into the pharmaceutical industry in 2001, he spent the next 17 years in increasingly senior roles in hematology and oncology, first at a small company, Centocor (a Johnson & Johnson subsidiary), then at Sanofi-Aventis, and later Celgene. In September 2018, after nine years at Celgene, he joined AbbVie as global head of hematology clinical development. In July of last year, he became AbbVie’s vice president and global head of oncology clinical development.

Zaki was attracted to AbbVie because of the “amazing science being done here in hematology and oncology,” which he has helped to advance since joining. He points to venetoclax and ibrutinib as “perhaps the best evidence” of this science. Venetoclax (branded as Venclexta) was initially developed as a targeted agent for the B-cell lymphoma-2 (BCL-2) protein. In 2016, it was approved by FDA for the treatment of chronic lymphocytic leukemia (CLL) in previously treated patients with the 17p deletion genetic mutation. It has since been approved for people with CLL or small lymphocytic lymphoma (SLL), with or without 17p deletion. In combination with azacitidine or decitabine, it also was approved in 2020 for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults who are age 75 years or older.

“I used to treat patients with AML and it was almost a death sentence,” explains Zaki. “It was a horrible disease and nothing had been advanced for many years to treat those patients. The chemotherapy ended up killing them. Getting approval for a therapy that targets the disease, that can actually transform the patient’s life, was an extremely proud moment for me.”

Ibrutinib (branded Imbruvica) was originally approved by FDA in 2013 for the treatment of mantle cell lymphoma; the approval was expanded soon after to include CLL and SLL. In August 2018, ibrutinib in combination with rituximab was approved for the treatment of adults with Waldenström’s macroglobulinemia, a rare and incurable type of non-Hodgkin’s lymphoma.

Zaki also has great faith in navitoclax, AbbVie’s investigational oral BCL-XL/BCL-2 inhibitor, for the treatment of myelofibrosis, a rare blood cancer that develops when a genetic mutation occurs in blood stem cells, resulting in excessive scar tissue formation in the bone marrow.

“We are investigating if navitoclax may be an option that offers myelofibrosis patients a new level of disease management, including improved symptom control and potentially better treatment outcomes,” Zaki said in an AbbVie media release recently. These “anti-fibrosis efforts build on the company’s history of transforming standards of care in blood cancers with significant unmet needs,” he added.

, “With Venclexta, Imbruvica, and now navitoclax, we are touching almost every hematologic malignancy that you can face.” He adds, with a smile, “We’re starting to run out of hematologic indications. What is left, other than very chronic, almost cured diseases?”

He has good reason to feel confident, though, especially when contrasting these advances with the cancer landscape he knew at the start of his career. “It’s a whole different world now. What we had back then in terms of treatment was chemo, chemo, chemo. People dying from chemo, people dying from disease. Now we see people living for a very long time,” he says. “What we’re aiming for in cancer now is a functional cure, where we transform acute malignancies to chronic ones. People will die from natural causes, not from their cancer. Cancer will be like diabetes, something you can live with.”

Zaki is excited about the growth of immuno-oncology, which was the subject of his PhD back in the 1990s. “To be able to go back to your own immune system to avoid chemotherapy, that is just magic,” he says. “Do you know how many years we’ve been talking about that in cancer therapy? Now we are finally able to do it.” The rise of immuno-oncology means more precise medicine, “which means you can treat the people who actually benefit from the drug.” Zaki explains, “When you think about a 30% response rate approval, what happened to the 70%? They’re taking a drug that doesn’t work for them. With more precise treatments, that response rate would increase to 90% or 100%. It’s very hard to do, of course; you have to find the right marker. A biomarker-driven approach is the key to this precision medicine that we’re pursuing.”

Among Zaki’s priorities at AbbVie now is to maximize the pipeline to get new treatments quickly to patients in need. “It’s about providing patients with options. Options are the name of the game with cancer; people live from drug to drug.”

There is a special urgency since COVID-19, which has been particularly hard on cancer patients. Zaki talks about patients, already immuno-compromised, that were simply too afraid to go to the hospital for their therapy. But he is proud that, during the pandemic, AbbVie’s oncology team was able to provide guidance to healthcare professionals (HCPs) to help them continue looking after cancer patients. The team “worked around the clock to continue educating HCPs and patient organizations on the latest data and best practices,” he says. AbbVie’s patient access and patient support system also helped patients to obtain their drugs and offered them financial support. And the increasing availability of oral medication for these diseases now meant that patients didn’t need to make a trip to the hospital.

The experience of leading his team during the disruption of the last 21 months has been a good one for Zaki. “It was about listening,” he says. “Employees were stuck at home and they wanted to talk. You have to be able to listen to them and remind them of the vital importance of what they do and what they bring to patients.” He adds, “I’m the sort of person who wants to see people, to be physically there with them, but we adapted well to Zoom meetings and maintained a good level of interaction.”

An unexpected benefit of going virtual, says Zaki, was the boost to recruitment. “People became extremely available, and not just because of virtual communication,” he observes. “They started to want change. That’s one of the impacts of COVID. People are realizing that life is too short to put up with an unrewarding job. They want to do something more meaningful.” Where Zaki says he used to hope to get one or two CVs when trying to recruit high-caliber people, all of a sudden he was getting 15.

Zaki also credits the attraction—and, just as importantly, retention—of good candidates to AbbVie to the company’s culture of inclusiveness. “From the moment I had my interview, I felt welcomed and respected at AbbVie,” he says. “If you work here, you feel this. The company really cares about its employees. We’re reminded every day about the importance of professional development, behavior development, succession planning, etc. It is a scientific culture of inclusiveness, a culture of respect for people, their family life, and work-life balance.”

While Zaki can look back on his and AbbVie’s efforts during the pandemic with a measure of pride, he was frustrated about how COVID derailed one of his favorite pastimes—travel. Fortunately, he had his other great hobbies, reading and watching sports, to occupy him during his extra free time. Zaki is a fan of basketball—he used to play back in Egypt—and he closely follows soccer, particularly the UK’s Premier League, where his countryman Mo Salah plays for Liverpool FC. But with travel being Zaki’s great passion, he felt trapped and helpless during COVID, like many of us. He says this, however, with his recognizable cheerfulness, so it’s difficult to believe his energy and enthusiasm were dampened too much by the restrictions of this period. He is quick to add that now that the borders are being opened, he’s very happy to be traveling again. In life, as in his work, the challenges and obstacles that Zaki faces are met—and overcome—with this enduring spirit of optimism.

is Pharm Exec’s European and Online Editor. He can be reached at

Executive Profile

After two decades at the forefront of hematology and oncology drug development, AbbVie’s vice president and global head of oncology clinical development, Mohamed Zaki, MD, PhD, is more hopeful than ever about recent advances in the space.

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Pharmaceutical Executive, December 2021 Issue (PDF)