In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value. Shape is also eligible to receive tiered royalties on future sales of products resulting from the collaboration. Shape will do the preclinical research to identify and then deliver candidates while Roche will be responsible for the development and worldwide commercialization. Shape’s platforms, enabling RNA targeting, editing and replacement for treating genetic disorders, include RNAfix, which enables direct in vivo targeting and modification of RNA by leveraging proteins such as ADARs, suppressor transfer RNAs and engineered adeno-associated viruses.

Vertex taps Arbor CRISPR tech in potential $1.2B cell therapy development deal

Facing inevitable challenges ahead beyond peak performance for its wildly successful cystic fibrosis franchise, Vertex Pharmaceuticals Inc. is continuing to make moves, announcing Aug. 24 it licensed rights to CRISPR gene-editing technology from Arbor Biotechnologies Inc. Co-founded by Feng Zhang and David Walt, Arbor's tech could figure into new Vertex cell therapies for diabetes, hemoglobinopathies and other diseases. Under deal terms, it's eligible to receive up to $1.2 billion from Vertex in milestone payments across up to seven programs, plus royalties. Vertex is further backing Arbor via a convertible note.

FDA approves Cara’s Korsuva for itching in hemodialysis patients

The FDA has approved Korsuva (difelikefalin) from Cara Therapeutics Inc. for pruritis in patients undergoing hemodialysis, following a fast review. Korsuva is the first therapy approved by the FDA in the indication and is the first ever approval for the Stamford, Conn.-based company. Korsuva is the injected form of the drug, which acts as an agonist by selectively targeting kappa opioid receptors, which are associated with addictive behaviors. The drug is the first approved in its class and has shown statistically significant reductions in itch intensity, while also improving quality of life measures in the Kalm-1 and Kalm-2 phase III trials, building on supportive phase II results.

Theravance deflates on phase II primary, secondary endpoint misses in UC

Shares of Theravance Biopharma Inc. (NASDAQ:TBPH) were trading midday at $9.86, down $4.31, or 30%, after the firm disclosed top-line results from its phase IIb dose-finding induction study of izencitinib for ulcerative colitis (UC). The oral, gut-selective pan-Janus kinase inhibitor also known as TD-1473 missed its primary endpoint of change in the total Mayo score and the key secondary endpoint of clinical remission at the eighth week relative to placebo. There was a small dose-dependent increase in clinical response measured by the adapted Mayo score, Theravance said, which was driven by a reduction in rectal bleeding. At all doses, the drug was well-tolerated when given once daily for the eight-week period, with adverse event rates similar among patients on izencitinib vs. placebo. Yet to come are results from the phase II study with izencitinib in Crohn’s disease, expected late this year or early next. Based on the UC data, Theravance said it will “seek to minimize future expenses associated with the izencitinib program.”

Former Peloton CEO Josey takes reins at Atavistik Bio, backed with $60M in series A funding

Atavistik Bio Inc. has raised $60 million in a series A financing round to advance its preclinical molecules targeting genetically validated targets in metabolic diseases and cancer. In charge of the company is acting CEO John Josey, who was formerly CEO at Peloton Therapeutics Inc. before its acquisition by U.S.-based Merck & Co. Inc. in 2019 for $1.05 billion. The financing, which was led by The Column Group and joined by Lux Capital and Nextech Invest Ltd., will support further development of Atavistik’s drug discovery platform and future identification of drug candidates.

Bluewillow posts positive data for its intranasal anthrax vaccine

Bluewillow Biologics Inc. is developing an intranasal anthrax vaccine and just posted positive interim data from BW-1010’s phase I trial. When administered four weeks apart in two doses each, the anthrax vaccine was found to be safe and well-tolerated. Systemic and mucosal immunity was induced as the study found levels of neutralizing systemic antibodies, nasal IgA/IgG and Th17 response in peripheral blood cells. The Ann Arbor, Mich.-based company, along with Medigen Vaccine Biologics Corp., of Taipei, Taiwan, also is developing a SARS-CoV-2 intranasal vaccine, S-2P-NE-01, and has preclinical data for it showing positive serum and mucosal immune response. Bluewillow is in a crowded field of 44 COVID-19 intranasal therapeutics and vaccine programs, according to BioWorld records.  

Astellas and Fibrogen bag European approval for first-in-class HIF-PH inhibitor

Less than two weeks after Fibrogen Inc. received an FDA complete response letter for its hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, Evrenzo (roxadustat), Astellas Pharma Inc. and Fibrogen have won European Commission approval for the drug to treat symptomatic anemia associated with chronic kidney disease in adults. The approval triggered a $120 million milestone payment from Astellas to Fibrogen, which will also receive royalties on European net sales of the drug.

FTC attorneys invoke COVID-19 vaccine in case against Illumina/Grail deal

The U.S. Federal Trade Commission is hardly alone in its antipathy toward Illumina’s acquisition of Grail, and FTC attorney Susan Musser said Aug. 24 that Illumina’s dominance of the market for next-generation sequencing (NGS) is perhaps the key aspect of the FTC’s case. However, Musser invoked the wide number of companies that jumped into the fray to develop a vaccine for the COVID-19 pandemic as an illustration of the need to maintain competition in the multi-cancer testing space, adding that “it would be crazy to put all our eggs in one basket, which is exactly what Illumina is asking us to do.”

Even as manufacturing ramps up, liability hinders vaccine distribution

COVID-19 vaccine makers continue to ramp up their manufacturing capacity to meet the EU demand for vaccines, even as many countries around the world clamor for doses. Meanwhile, potential liability issues are hampering distribution of donated vaccines in some of those countries. For instance, U.S. “vaccine donations to India reportedly remain in a Delhi warehouse because the Indian government has not agreed to indemnify vaccine manufacturers for vaccine-related injuries,” according to a recent U.S. Congressional Research Service report. Subsequently, only a small percentage of the population in India, which has seen a surge in COVID-19 cases, is fully vaccinated.

PDUFA VII sets sites on rare diseases, more patient involvement

Advancing efforts from PDUFA VI, the seventh iteration of the user fee agreement negotiated between the U.S. FDA and drugmakers is intended to take patient involvement in drug development to the next level, support the next wave of biologics, and provide new tools for developing therapies and cures for rare and ultra-rare diseases. PDUFA VII is “an opportunity to advance innovative research and development and review approaches to prepare for the future of drug development, including leveraging applicable lessons learned from the COVID-19 pandemic,” said Stephen Ubl, president and CEO of the Pharmaceutical Research and Manufacturers of America.

Slipping behind last year, nonprofit deals and grants still driven by COVID-19

Biopharma company deals with nonprofit entities, as well as grants, are not keeping the same pace as last year, but the proportion of money flowing into COVID-19 efforts continue to account for the majority of those recorded overall. Through the last week of August, BioWorld has tracked 237 grants valued at $1.87 billion, and 568 bio/nonprofit deals worth $8.03 billion.

Also in the news

180 Life Sciences, AB Science, Abbvie, Abilita, Abveris, Adalta, Adamis, Adjuvance Technologies, Advanced Proteome, Alimera Sciences, Alphamab Oncology, Antengene, Arbor, Atea, Avidity, Awakn Life Sciences, Azurity, Biogenes, Bio-Path, Cancer Advances, Cara, Cardiol, Carina, Cartesian, Cassava Sciences, Celltrion Healthcare Australia, Cytrx, Develco, Dice Molecules, Eom, Forian, Fyonibio, HCW Biologics, Humanetics, Immunocore, Index, Janssen, Lexicon, Meiji, Microquin, Mindmed, Nouscom, Novartis, Nrx, Oncotelic, Oramed, Pharmacyte, Pieris, Polarityte, Privo Technologies, PTC, RDIF, Regeneron, Reistone, Roche Products, Sorrento, Stealth, Tarsier, Theravance, Timber, Tonix, Tryp, Vifor, Xalud