Celldex Therapeutics Inc. unveiled top-line data from the ongoing phase II study testing barzolvolimab (barzo) in eosinophilic esophagitis (EoE), a chronic inflammatory disease of the esophagus. The study met its primary endpoint, proving barzo’s oomph in depleting mast cells in the gastrointestinal tract. But this didn’t result in betterment of EoE symptoms, which means they are not a primary driver, Hampton, N.J.-based Celldex said. Barzo turned up a favorable safety profile, but the company will not pursue the compound in EoE. Shares of Celldex (NASDAQ:CLDX) fell 11% to $21.38 in early trading Aug. 20.

Rocket’s RP-A501 back on track as FDA lifts hold on Danon trial

With a few tweaks to the protocol, Rocket Pharmaceuticals Inc.’s phase II trial testing RP-A501 in Danon disease is expected to resume following the lifting of the clinical hold, issued by the U.S. FDA in May following the death of a patient in the pivotal gene therapy study. The agency authorized the study to proceed with a lower dose to be administered to the next three patients, treated sequentially with a minimum of four weeks between each treatment, after which another review will be conducted. The study will also continue with a slightly revised prophylactic regimen, eliminating use of a C3 complement inhibitor, while maintaining sirolimus, rituximab and steroids. RP-A501, which is designed to work by delivering a LAMP2B gene to heart cells, is the only clinical-stage candidate targeting Danon disease, a rare genetic disorder that causes heart muscle thickening and weakening.

Puretech on hunt for phase III funding for newco Celea

Quoted technology commercialization company Puretech Health plc is scouting for third parties to fund phase III development of deupirfenidone, after spinning the respiratory drug into a new startup, Celea Therapeutics. This follows the publication of positive phase IIb data for the reformulated version of the marketed antifibrotic Esbriet (pirfenidone) in December 2024. “I believe that deupirfenidone has the potential to be a true turning point in the treatment of idiopathic pulmonary fibrosis,” said Sven Dethlefs, CEO of Celea. “Our phase IIb data demonstrated the potential for best-in-class efficacy with a favorable safety and tolerability profile, addressing two of the most critical limitations of current therapies.”

Technique for aging brain organoids makes better Alzheimer's model

A new method for accelerating the maturation of neuronal cell models and brain organoids is poised to make it possible to track the etiology of neurodegenerative diseases that develop over decades. The non-invasive technique uses graphene to convert light into electrical cues that prompt neurons to connect and communicate in vitro. That avoids the need for genetic engineering of cells to make them express light-sensitive proteins, as is the case in the traditional optogenetics approach to controlling cellular activity. It also sidesteps the damage to neurons by the use of external actuators. The graphene-mediated optical stimulation (GMOS) technology is a “game changer” for brain research, said Alysson Muotri, professor of pediatrics at the Stem Cell Institute, University of California San Diego.

Vantai, in a $1B+ deal, joins up with Halda

Generative AI drug discovery company Vantai Inc. could bring in more than $1 billion in a new collaboration with Halda Therapeutics Inc. Vantai, which already has deals with other companies including Bristol Myers Squibb Co., Janssen Pharmaceutica and Blueprint Medicines Corp., will use its foundation model and platform to identify and validate context-specific target effector pairs that will then be fed into Halda’s development pipeline. Halda is developing what it calls RIPTAC (regulated induced proximity targeting chimera) therapeutics to selectively kill cancer cells. Its most advanced candidates, oral small-molecule androgen receptor modulators, are for treating solid tumors.

Remegen licenses VEGF/FGF inhibitor to Santen for ¥1.3B

Remegen Co. Ltd. is out-licensing its VEGF/FGF dual-target fusion protein drug, RC28-E, to Santen Pharmaceutical (China) Co. Ltd. in a carve-out deal worth more than ¥1.3 billion (US$180 million). Under the terms, Santen China gains exclusive rights to develop, manufacture, and commercialize RC28-E in mainland China, Hong Kong, Macau, and Taiwan, as well as in South Korea, Thailand, Vietnam, Singapore, the Philippines, Indonesia, and Malaysia. Remegen retains global exclusive rights to RC28-E outside these regions. In exchange, Santen will pay Remegen an up-front payment of ¥250 million, plus development and regulatory milestone payments of up to ¥520 million, and sales milestone payments of up to ¥525 million. Remegen is also entitled to tiered sales-based royalties.

Opthea avoids bankruptcy under new investor settlement

After announcing in April that it would discontinue development of its lead candidate, sozinibercept (OPT-302), in wet age-related macular degeneration (AMD), Opthea Ltd. has come to a settlement agreement that will allow it to avoid bankruptcy and continue operations. Opthea announced in April that it was shuttering the phase III Shore trial after sozinibercept failed the phase III Coast trial in wet AMD. Following the trial failures, Opthea said it was assessing its rights and obligations under its Development Funding Agreement (DFA). Certain termination events under the DFA could have triggered payments by the company of up to $680 million to investors. After extensive negotiations, Opthea has reached a binding agreement with the DFA investors to terminate the DFA and enter into a settlement arrangement under which investors will receive a one-time payment of $20 million, allowing Opthea to avoid bankruptcy.

Also in the news

Adaptive, Ajax Therapeutics, Aldeyera, Altimmune, Antengene, āshibio, Astazeneca, Axelyf, Biogene, Biomx, BMS, Cantargia, Cytomed, Genentech, Henlius, Imbria, Inhibikase, Invivyd, Iovance, Kelun-Biotech, Kriya, Memo Therapeutics, Opthea, Oric, Pfizer, Propanc, PTC Therapeutics, Remegen, Rengexbio, Sanofi, Santhera, Silo Pharma, Spyglass