A New Transatlantic Payout for the Best in Start-Up Innovations
Collaboration. It’s the one word that rings loudest within the R&D precincts of big Pharma today – finding the right partner, with the right science, to achieve the best result: a new therapy that also does right for the patient. Making this happen seamlessly across geographies, functions and markets is arguably the most challenging aspect of collaboration, requiring the active intervention of many different players representing not only industry but academia, professional associations and government as well.
The task of building such multi-disciplinary networks is slowly gaining momentum. One fresh example is the partnership kicked off earlier this month between the Galien Foundation, which administers an annual global awards program for medicines innovation, and the French government investment and trade promotion agency, UBIFRANCE. Called UBISTART [for Synergistic Transfer of Advanced Research Technologies], it seeks to develop commercially relevant tie-ups between US and French companies active in the pharmaceutical, biotech, e-health, medical device and process technology sectors. The emphasis is on facilitating early-stage research, development and full commercialization collaborations between start-ups and the larger industry players that can provide access to financial capital and know-how. “Our collaboration with the Galien Foundation is designed to remove the silos that often slow the spread of good ideas while also creating a forum that give these ideas formal recognition by the best scientists, as a prelude to commercialization,” Yves Germani, UBIFRANCE’s Health Sector Head for North America, told Pharm Exec.
The project launched on July 10 at a networking conference hosted by the New York Academy of Sciences and featuring the participation of approximately 50 French-based biotech, specialty pharma and process technology companies along with key big Pharma players [Lilly, Pfizer, Merck, B-I, J&J and Roche], venture capital investors [Aisling Capital], and academic research centers, including Memorial Sloan Kettering and Rockefeller University. Some 600 “matchmaking” meetings took place during the course of two days. In a keynote address to the group, John Lacey, Communications and External Affairs Leader for J&J’s Innovation Center in Cambridge, MA, emphasized the role of such exchanges in bringing new ideas to the attention of his company. “We depend and thrive on partnerships,” Lacey said. “Through our global network of Innovation Centers, J&J continues to augment an aggressive early stage investment platform, with 60 separate collaborations agreed across the pharma, medical device and consumer health care fields in the last 18 months.”
And the winner is…
To add momentum beyond the one-off contacts, UBISTART is initiating a competition in which the US-French companies will, over the next three months, submit investment collaboration projects for review by a jury panel of scientific, business development and financing experts, who will then select nine nominees and grant three cash prizes – totaling $100,000 – as the first UBISTART award for excellence in start-up innovation. Criteria for selection is: advancing the state of science; meeting an unmet medical need and improving the current standard of care; larger impact on society; a commercially viable business plan; and evidencing the added value from international partnering rather than going it alone.
The deadline for nominated projects is October 31, and winners will be announced at the annual Prix Galien France drug innovation award ceremony in Monaco on December 4. In addition to the grant money, recipients will receive facilitation and counseling from UBIFRANCE to help implement their projects. The judging panel is led by former Wyeth Pharmaceuticals CEO Bernard Poussot and includes several Nobel laureates who also serve on the Board of the Galien Foundation. “The judging panel is an unusual but highly practical mix of the doers and thinkers who drive growth in this industry,” said Foundation chair Bruno Cohen.
The diversity of offerings on display by the French companies at the July 10 Academy event shows how good ideas continue to spread across geographies. As illustration, the following are three examples of some fresh new approaches to solving an unmet medical need.
Anagenesis Biotechnologies, located in Strasbourg, specializes in new stem cell-based applications for treatment of genetic and age-related muscle degenerative diseases. It is focused on three specific conditions: Duchenne muscular dystrophy [DMD], a genetic abnormality affecting mainly young boys that leads to progressive muscle loss, paralysis and death by early adulthood; sarcopenia, an aging-related degeneration of bone mass and muscle strength, and a costly driver of disability and institutionalization; and cachexia, a similar agent of physical decline and symptomatic factor for serious illnesses like cancer and AIDS. The company’s calling card is a patented cell reprogramming model that can generate healthy skeletal muscle cells from stem cells. This proprietary technology has the potential to be applied either as a screening platform to identify and differentiate diseased cell structure for targeted cell therapy, or as a biologic drug that would induce the proliferation of normal muscle cells and thus slow or halt progression of disease.
The technology comes from two decades of lab research conducted by Harvard Medical School Professor Olivier Pourquie on the paraxial mesoderm cells that are the building blocks of skeletal muscles and vertebrae in humans. Pourquie is a co-founder of Anagenesis, along with Columbia University Medical Center investigator Chris Henderson, an expert in neuroscience and regenerative medicine, and company CEO Yves Bonnefoy, a veteran operations executive who spent 30 years at GSK.
What’s next? According to Bonnefoy, the company has achieved proof of concept for its proprietary technology in animal tests for Duchenne and is now seeking financial support from investors to enter the formal clinical phase. Anagenesis has obtained funding from a key patient organization, the French Association for Muscular Dystrophy [AFM], is working with a range of other KOLs, and plans to open an office in the US to expedite the search for partners. “These are major diseases of decline and disability which currently have no real treatments despite their impact on aging societies,” Bonnefoy told Pharm Exec. “Sarcopenia alone has a global market potential of $10 billion. It’s a growth area that coincides with a huge gap in medical need, which we aim to fill.”
Affilogic is a four-year old biotech based in Nantes with a mission to maximize therapeutic drug delivery applications from the rich bacterial soup of the thermal geysers in Yellowstone National Park. Affilogic has built an artificial protein derived from the sulfolobus acidocaldarius microorganism that acts as a safer and more effective delivery vehicle to conventional monoclonal antibody drugs. Its patented Nanofitin platform improves the therapeutic effectiveness of drugs by improving a drug’s binding capacity, enhancing tissue penetration and allowing for more patient-friendly modes of administration [i.e. no injections]. The technology is especially effective in maximizing the positive pharmacokinetic effects of inflammatory drugs, anti-infectives, ophthalmology drugs, and CNS treatments. “Due to their small size and high drugability, Nanofitins ensuresthatthe process of making – and taking –medicines is more effective, safer and less costly for manufacture,” says Affilogic founder and CEO Olivier Kitten. The company is working closely with partners like Switzerland’s Ferring SA, to develop specific Nanofitins for the diagnosis and cure of diseases where the antibody approach is not successful. Fifteen Nanofitins have been identified by Affilogic researchers to date, and seven are currently being evaluated with partners for use in developing oral drugs against inflammatory disorders like psoriasis and Crohn’s disease.
Kitten, who works as both a researcher in the French public academie and private entrepreneur, points to a practical aspect of the company’s technology: for psoriasis, Nanofitins bind directly to remove lesions on the skin, in contrast to the more generalized efficacy of auto-immune drugs like Humira, which require much larger dosing over time to stay effective, thus raising the potential danger of patient side-effects. He also cites the fact that since the superior absorptive capacity allowed by Nanofitins can facilitate oral administration rather than injection, patient adherence to drug therapy is improved.
Affilogic has funding from the EU Commission’s Seventh Framework Program on Research, under the SADEL [Scaffolds for Alternative Delivery] consortium; and the company’s partnership with Ferring is slated to complete clinical work within the next 18 months. The company is seeking additional investor financing, especially from outside the EU. “Our key value proposition is the benefit big Pharma partners can obtain from the reduced risk associated with Nanofitins in the development of advanced drug therapies for medically complex conditions,” Kitten concludes.
Materials management is the mantra for another French start-up, Rondol Industrie, based in Strasbourg but reaches beyond France due to research ties to the UK and an established sales presence in Germany. The company’s business model is to apply a venerable technology in manufacturing – hot metal extrusion [HME] – to some very modern ends, namely, in offering pharma customers new, more efficient machinery that keeps pace with pharma materials science and changing regulatory requirements. Starting in 2008, Rondol began introducing a new line of extruder machines specifically adapted to health care sector requirements, including equipment for small batch testing and production, precision dosing, content uniformity and enhanced materials stability, at variable Ph and moisture levels.
In 2012, the company initiated further extruder design improvements that meet or exceed upgraded requirements for GMP compliance from the FDA and EMA. “A new generation of APIs and biologics derived from living organisms has made manufacturing a strategically important capability that gives an edge to producers who can do it at lower cost. There is a compelling business proposition for drug manufacturing equipment that can do many things simultaneously or in sequence, according to the highest safety and quality standards,” says Rondol’s Chairwoman Victoire de Margerie. “What we are offering through our next-gen extrusion technologies is a high level of customization that allows companies to run hundreds of trials, produce small batches of product precisely according to spec, with easy cleaning and sterilization, at a fraction of what it used to cost. It is highly scalable, at the level of what we might call ‘a lab or factory in a box’.”
Persuading big Pharma to purchase this technology has proven a bit tougher than expected, particularly in the US. Some of this may be due to a misalignment of incentives – for example, do drug companies want to risk alienating US oncologists with manufacturing so efficient that it creates chemotherapy drugs with the stability to be infused in patients once a week rather than daily, resulting in a potential loss of their income? To raise interest in its technology, Rondel has formed collaborative research ties to leading process engineering schools like the University of Texas/Austin and the University of Mississippi, while using its brands’ quality reputation in Germany as a reference point to attract more US customers. And now that manufacturing is firmly on the radar screen of CEOs, Margerie sees real benefit from extending its sales pitch directly to the c-suite. Contends Margerie, “the continued restructuring and downsizing of industry physical plant is not a crisis, but an opportunity for innovation. CEOs readily understand that.”
Message to regulators
One perspective all three French entrepreneurs share is a sensitivity to the broader regulatory environment that enfolds not only them, but the larger players in pharmaceuticals as well. Uncertainty about risk exposure around new technology is compounded by the absence of clarity in the way regulators classify it. Says Kitten, “our innovations are often simpler and less risky to apply in real world settings, but because it is novel technology regulators still tend to classify it with the most complex products when deciding on a pathway to approval. This can slow the process for us but more important it gives our potential big Pharma partners pause.” As an alternative, regulators should have more discretion in considering registrations on the basis of overall benefit and reduced level of risk – “technology is changing so fast that the ‘one size fits all approach’ is an anomaly.”
Another “ask” relates to the perennial pitfalls of the so-called “valley of death,” where after proof of concept is completed, and companies are poised to launch human trials, financing disappears. Regulators could help address this by moving toward a more common set of standards that eases the complexities of initiating those trials, thus reducing direct costs to the developer. The three start-ups noted that making this happen on a Trans Atlantic basis would be a real step forward, though progress is necessary within the EU as well.