More than two years after the U.S. FDA shocked Intercept Pharmaceuticals Inc. with its rejection of obeticholic acid (OCA) to treat fibrosis due to nonalcoholic steatohepatitis (NASH), the company has announced data it said will form the basis of an NDA refile in the disease for which there is no approved therapy. Morristown, N.J.-based Intercept has positive top-line results from a new interim analysis of its ongoing phase III Regenerate trial of OCA in patients with liver fibrosis due to NASH, otherwise known as fatty liver disease. In June 2020, when the FDA served Intercept with a complete response letter, it asked for additional efficacy and safety data after deeming that the information in a first interim analysis of Regenerate did not show a convincing benefit-risk balance.

Left holding the bag: Diamedica’s phase II/III halted by FDA after adverse events

Three serious adverse events have led the U.S. FDA to place a clinical hold on Diamedica Therapeutics Inc.’s phase II/III study of DM-199, a synthetic form of human tissue kallikrein-1 for treating acute ischemic stroke. The blood pressure in three participants dropped to a significantly low level shortly after receiving an I.V. dose of the therapy. All three bounced back to normal within minutes of stopping the I.V. Diamedica said it believes the cause was switching to I.V. bags formulated from different materials than the ones used in the prior phase II study. Supply issues led to the original bag not being available in many U.S. hospitals, the company said. The Minneapolis-based company’s stock (NASDAQ:DMAC) had taken a steep nosedive at midday, with shares trading 36% lower.

ADC program on hold after Cytomx unveils phase II breast results

Shares of Cytomx Therapeutics Inc. (NASDAQ:CTMX) were trading midday at $1.40, down 58 cents, or 29%, on data from a phase II study with CD166-directed Probody-drug conjugate CX-2009 (praluzatamab ravtansine) in patients with hormone receptor-positive/HER2-non-amplified breast cancer. Arm A met its primary efficacy endpoint of confirmed objective response rate of greater than 10% by central radiology review. Wall Street was concerned about toxicities, and Cowen analyst Boris Peaker called the results “disappointing” from an efficacy standpoint as well. He highlighted a clinical benefit at 24 weeks of 40% with a median progression-free survival of 2.6 months. Cytomx said it’s putting the program on hold while seeking a partner.

Sumitovant’s Urovant inks $75M licensing deal with Pierre Fabre for OAB treatment vibegron 

Sumitovant Biopharma Ltd. subsidiary Urovant Sciences GmbH is out-licensing overactive bladder (OAB) treatment vibegron to Pierre Fabre Médicament to register and commercialize the drug in the EU, U.K. and Switzerland. Under the terms of the deal, Basel, Switzerland-based Urovant will receive up to $75 million in up-front payments, regulatory and sales milestones and royalties, while Urovant will retain full commercialization rights to vibegron in the U.S. and certain other markets. As part of the deal, Pierre Fabre has the option to commercialize the drug in other territories that mostly include French-speaking countries in Sub-Saharan Africa, as well as Turkey, and certain Eastern European countries.

Beigene collaborates with Innorna for mRNA therapies; new phase III data out

Beigene Ltd. has formed a partnership with Innorna Co. Ltd. to jointly develop messenger RNA (mRNA)-based therapeutics. Shenzhen, China-based Innorna will get an undisclosed up-front payment and is also eligible to receive additional payments based on development, regulatory and commercial milestones, as well as tiered royalties. Beigene will maintain exclusive global rights to the mRNA candidates co-developed in the deal. In addition, Beigene acquired nonexclusive rights to Innorna’s lipid nanoparticles (LNP) technology platform for its internal research and development.

US Rx price negotiations edge closer to reality

A few weeks ago, the odds of the deeply divided U.S. Congress passing drug pricing reforms that would allow direct Medicare negotiation seemed pretty slim. But those odds improved significantly yesterday when Senate Democrats reached a compromise on a pared-down version of the Biden administration’s Build Back Better agenda. While the Senate’s draft bill is more lenient on Medicare negotiation than the House version, its pricing policies “would have a tangible – although not catastrophic – long-term negative impact on large cap companies . . . and could potentially affect long-term patent strategies, R&D, and M&A across biopharma,” said Brian Abrahams, an equity analyst with RBC Capital Markets. On the positive side, he said passage of such a bill may “finally clear a lingering overhang to enable clearer [long-term] projections of revenues for therapeutics companies.”

Also in the news

Agile, Albireo, Altamont, Applied Molecular Transport, Aqualung, Arch, Ascletis, Bioarctic, Cend, Chinook, Clairvoyant, Coherus, Eisai, Enlivex, Genentech, Gnubiotics, Homology, Huili, Humanigen, Hutchmed, Immunome, Inflarx, Inmagene, Innocoll, Intravacc, Junshi, JW, Kalvista, Kamada, Kazia, Kernal, Kiora, Mnemo, Nordic Nanovector, OBI, Olix, Pierre Fabre, Pfizer, Plakous, Pyramid, Quoin, Ring, Roivant, Sarepta, Sensorion, Sollievo, Takeda, Titan, Touchlight, Turnstone, Urovant, Vast, Vera, Veru, Walden, Zenith, Zhiyi