Rare skin disease, novel drug target discovery, neglected tropical disease immunotherapy, and rare bone disease treatment all drew fresh investment this week, as companies moved to lock down late-stage and early-stage assets through a mix of outright acquisitions and licensing agreements.

LEO Pharma acquires dersimelagon

LEO Pharma has entered into an agreement to acquire worldwide rights to dersimelagon from Tanabe Pharma, with LEO Pharma committing up to $435 million in upfront and near-term milestone payments, plus potential downstream milestones and tiered royalties on net sales.¹ The transaction remains subject to customary closing conditions, including applicable regulatory approvals.

Dersimelagon is an oral, once-daily small molecule MC1R agonist being developed for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), rare and severe genetic diseases that cause severe sunlight-induced skin damage and can significantly limit patients' ability to spend time outdoors.¹ Symptoms may include skin rash, swelling, redness, burning sensations and, in some cases, liver damage. Dersimelagon is designed to increase skin melanin, helping reduce sunlight penetration and protect against phototoxic reactions.¹

Dersimelagon has been granted both FDA Fast Track Designation and Orphan Drug Designation, and an NDA for the treatment of EPP and XLP was submitted to FDA for regulatory review in June 2026, though it has not yet been approved by FDA or any other regulatory agency and its safety and efficacy have not been established by any regulatory authority.¹

"Patients living with EPP or XLP face the devastating lifelong burden of severe reactions to sunlight, and there is a clear need for treatment options that can make a meaningful difference in their everyday lives," said Christophe Bourdon, chief executive officer of LEO Pharma. "By addressing a clear unmet need in a rare skin disease, dersimelagon represents a compelling opportunity to expand our rare dermatology pipeline with a late-stage oral therapy candidate."

The deal is expected to increase LEO Pharma's investments in 2026 for pre-launch activities and in 2027 for a potential launch, with an updated financial outlook to be provided alongside the company's H1 2026 Interim Report on August 18, 2026.¹ J.P. Morgan Securities plc acted as financial advisor to LEO Pharma, and Latham & Watkins LLP acted as legal advisor.

OmniAb signs global ion channel collaboration with Eli Lilly

OmniAb, Inc. has announced a global collaboration and license agreement with Eli Lilly and Company for a new ion channel program, leveraging OmniAb's technology platform and expertise in ion channel discovery and screening.² Under the terms of the agreement, OmniAb will receive an upfront payment and is eligible to receive up to $370 million in research, development and commercial milestone payments, as well as tiered royalties on global net sales. The therapeutic target and modality remain undisclosed.²

"We are pleased to enter into this important new relationship with Lilly," said Matt Foehr, chief executive officer of OmniAb. "Lilly is focused on delivering medicines to address major unmet medical needs and our differentiated expertise and established technologies that relate to ion channels are well positioned to help drive this new discovery program."

Chugai grants GSK exclusive license to AID351

Chugai Pharmaceutical has signed an exclusive license agreement with GSK for the anti-dengue virus antibody AID351 following the collaboration agreement for AID351 the two companies signed on January 30, 2025.³ Under the new agreement, GSK is set to obtain an exclusive, worldwide license from Chugai to develop, manufacture and, if successful, commercialize AID351, which is expected to be used to manage flavivirus infections, including dengue fever.³

GSK will be responsible for clinical development, including Phase I trials, as well as manufacturing of the asset, while Chugai is eligible to receive royalties based on future product sales by GSK if successful, along with a percentage of any sublicensing income GSK receives from third parties.³

"This agreement reflects GSK's ambition to change the trajectory of high-burden infectious diseases in lower-income countries. With between 100 and 400 million people globally estimated to be infected by dengue each year and a gap in solutions, AID351 offers a potential solution for vulnerable communities," said Dr. Harmony P. Garges, chief global health officer of GSK.

BioMarin acquires Alesta Therapeutics

BioMarin Pharmaceutical Inc. has entered into a definitive agreement to acquire Alesta Therapeutics to gain Alesta's lead clinical-stage asset, ALE1, with Alesta shareholders set to receive an upfront payment of $275 million plus up to $215 million in subsequent payments upon achievement of certain development and regulatory milestones.⁴

Alesta plans to spin out all non-ALE1 assets prior to the close of the transaction, with former Alesta employees transferring to the spinout entity so that no Alesta employees become BioMarin employees.⁴ The deal is approved by the boards of directors of both companies and is expected to close this quarter, subject to customary closing conditions. BioMarin intends to fund the transaction with cash on hand and expects to provide updated full-year 2026 guidance reflecting the acquisition following closing.⁴

ALE1 is an orally active, small molecule candidate for the potential treatment of hypophosphatasia, a rare genetic bone disease caused by mutations in the ALPL gene that can affect bone and tooth mineralization, resulting in easy or frequent bone breaks, early tooth loss and, in adults, clinically significant muscle weakness, fatigue and pain.⁴ ALE1 is currently being evaluated in an ongoing Phase I/IIA clinical trial assessing safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with hypophosphatasia. If approved, ALE1 is expected to be the first oral therapy approach that targets the central disease metabolite PPi, or inorganic pyrophosphate, with the potential to impact both skeletal and broader manifestations of the disease through systemic correction of disease biology.⁴

The program will become part of BioMarin's Skeletal Conditions Business Unit following close.

"ALE1 is a strong strategic fit for BioMarin, bringing a potential oral alternative to the injectable therapies available today for people living with hypophosphatasia around the world while meaningfully strengthening our early-stage clinical pipeline," said Alexander Hardy, president and chief executive officer of BioMarin. "This is exactly the kind of opportunity to address a significant unmet need that lets us compete in larger rare disease markets."

Sources

1. LEO Pharma Further Strengthens Late-Stage Pipeline With the Acquisition of Dersimelagon LEO Pharma August 18, 2026, https://www.businesswire.com/news/home/20260817946702/en/LEO-Pharma-Further-Strengthens-Late-Stage-Pipeline-With-the-Acquisition-of-Dersimelagon
2. OmniAb Announces Global Collaboration and License Agreement for Ion Channel Program with Eli Lilly & Company OmniAb Inc. August 17, 2026,https://www.businesswire.com/news/home/20260812350809/en/OmniAb-Announces-Global-Collaboration-and-License-Agreement-for-Ion-Channel-Program-with-Eli-Lilly-Company
3. Chugai and GSK Sign Exclusive License Agreement for Anti-Dengue Virus Antibody AID351 Chugai Pharmaceutical August 18, 2026, https://www.chugai-pharm.co.jp/english/news/detail/20260818113000_1263.html?year=2026&category=
4. BioMarin to Acquire Alesta Therapeutics to Gain ALE1, a Potential First Oral Therapy for Hypophosphatasia, Adding an Important Clinical Program to BioMarin's Pipeline BioMarin Pharmaceutical August 18, 2026, https://www.prnewswire.com/news-releases/biomarin-to-acquire-alesta-therapeutics-to-gain-ale1-a-potential-first-oral-therapy-for-hypophosphatasia-adding-an-important-clinical-program-to-biomarins-pipeline-302854068.html