Lexicon Pharmaceuticals Inc. won U.S. FDA approval of Inpefa (sotagliflozin) for heart failure (HF). Regulators gave their go-ahead for the inhibitor of renal sodium-glucose co-transporter 2 (SGLT2) and intestinal SGLT1, given a broad label across the full range of left ventricular ejection fraction, including HFpEF and HFrEF, and for HF patients with or without diabetes. Shares of Lexicon (NASDAQ:LXRX), which had risen after hours on news of the green light in late afternoon May 26, were trading midday at $2.96, down 22 cents.

Pfizer posts positive phase III data for its hemophilia A/B treatment

Pfizer Inc. has positive phase III data for marstacimab in treating hemophilia that could lead to the first once-weekly subcutaneous treatment for hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B. Marstacimab (PF-06741086), an anti-tissue factor pathway inhibitor for severe hemophilia A or B, without inhibitors for those without inhibitors to factor VIII or factor IX, produced a statistically significant and clinically relevant reduction in annualized bleeding rate compared to prophylaxis and on-demand intravenous regimens. It’s a crowded field, with Sanofi SA receiving U.S. FDA approval in February for Altuviiio (efanesoctocog alfa), a recombinant factor VIII therapy for hemophilia A and Novo Nordisk A/S receiving a complete response letter in May from the FDA for concizumab to treat hemophilia A and B.

In $392M deal, C4 and Betta partner in China on preclinical NSCLC drug

In a deal potentially worth $392 million, C4 Therapeutics Inc. signed with Betta Pharmaceuticals Co. Ltd. to develop and market an orally bioavailable BiDAC degrader for non-small-cell lung cancer (NSCLC). Terms of the agreement call for C4 to receive $10 million up front, as well as $25 million through an equity investment and up to $357 million for development and commercial milestones. The deal also includes low to mid double-digit royalties on net sales in greater China, which includes Hong Kong, Macau and Taiwan. Betta will develop, manufacture and commercialize CFT-8919 in greater China. It is eligible for low single-digit royalties on net sales outside of greater China, where C4 retains all rights. C4 intends to submit an IND to begin clinical trials of CFT-8919 with the U.S. FDA within the next month.

Lilly out of suit alleging spread between list, net insulin pricing is fraud

Eli Lilly and Co. is paying $13.5 million to bow out of a class action lawsuit that claimed the list price for some of the company’s insulin products was fraudulent. Lilly also committed to capping the monthly patient out-of-pocket cost at $35 for at least four years, bringing the total value of the settlement, announced May 26, to more than $500 million, according to the attorneys who filed the class-action on behalf of patients in 2017. The attorneys at Hagens Berman alleged that as the list prices of insulin products made by Lilly, Novo Nordisk A/S and Sanofi SA “moved further and further away from the real prices offered to pharmacy benefit managers [PBMs], these list prices become so misrepresentative, they are fraudulent.” While it detailed the role PBMs played and the profits they were realizing, the suit only named the three drug companies as defendants.

Reprocell reports mixed results for phase II Stemchymal trial in spinocerebellar ataxia

Regenerative medicine product Stemchymal, an allogeneic adipose-derived mesenchymal stem cell treatment, missed the primary efficacy endpoints in two phase II spinocerebellar ataxia trials conducted in Japan and Korea, but a subpopulation analysis showed efficacy signals in patients with more severe conditions, Reprocell Inc. reported. The phase II trial conducted in Japan was a placebo-controlled, multicenter, randomized, double-blinded, parallel-group study to evaluate the safety and efficacy of the drug. Independently, another phase II trial with a similar design was conducted in Taiwan by the originator, Steminent Biotherapeutics Inc. In the Japan study conducted by Reprocell, of Yokohama, Japan, 31 patients received Stemchymal and 28 patients received placebo.

Holiday notice

BioWorld's offices were closed in observance of Memorial Day in the U.S. No issue was published Monday, May 29.

Also in the news

Abiogen, Acurex, Adcendo, Alpha Cognition, Amplo, Astrazeneca, Astrivax, Atea, Biodexa, Bionanosim, Biophytis, Biosenic, Bristol Myers Squibb, Catalyst, CNS, Concentra, Curium, Defence, Dermata, Duality Biologics, Eli Lilly, Ferring, Genelux, Gilead, Greenlight, Hutchmed, Idorsia, Ipsen, Johnson & Johnson, Marinus, Mydecine Innovations, Nascent, Nexcella, Novavax, Novo Nordisk, Oculis, Oncopeptide, Oxurion, Pharmanovia, Phebra, Philogen, Profoundbio, Rafarm, Rain Oncology, Rakovina, Roche, Scisparc, Sino, Stealth, Structure, Sun, Theseus, TME, Xilio, Xtalpi