Based on positive phase III study data, Applied Therapeutics Inc. plans to take its CNS-penetrant aldose reductase inhibitor to the FDA to talk about an NDA for treating the rare disease sorbitol dehydrogenase (SORD) deficiency. Interim data showed govorestat (AT-007) hit the study’s primary endpoints along with several key secondary endpoints. SORD affects about 3,300 people in the U.S., according to Applied Therapeutics. A mutation in the SORD gene was found in a subset of patients already diagnosed with Charcot-Marie-Tooth disease or distal hereditary motor neuropathy. The company’s stock (NASDAQ:APLT) had bounced 20% higher at midday, with shares going for $3.19 each.

Firefly Bio embarks on new degrader-antibody conjugate frontier

Raising $94 million in a series A round, South San Francisco-based Firefly Bio Inc. has emerged from stealth to advance its Firelink linker platform technology to develop degrader-antibody conjugates (DACs) to treat cancer. A combination of antibody-drug conjugates (ADCs) and targeted protein degradation therapies, DACs are a new class of medicines that have recently gained attention for their ability to replace toxic ADC payloads and to eliminate cancer-driven proteins. Incubated through founding investor Versant Ventures’ Ridgeline Discovery Engine, located in Basel, Switzerland, Firefly was able to establish proof of concept with its lead asset. The series A was co-led by Versant, MPM Bioimpact and Decheng Capital, with Eli Lilly and Co. participating.

Ono inks deals with Shattuck, Numab in oncology, autoimmune disease

Ono Pharmaceutical Co. Ltd. inked deals with Shattuck Labs and Numab Therapeutics AG aimed at bolstering its pipeline in oncology and autoimmune and inflammatory diseases. Ono, of Osaka, Japan, struck a drug discovery collaboration and option agreement with Shattuck Labs Inc. to generate bifunctional fusion proteins for pathways involved in autoimmune and inflammatory diseases. It also signed a global research, development and commercialization deal with Numab for its NM-49, a multispecific antibody designed to activate tumor-associated macrophage phagocytosis for treating cancers.

US agencies to explore role of middlemen in drug shortages

Seeking answers to the chronic drug shortages plaguing the U.S. health care system, the FTC and Health and Human Services jointly issued a request for information (RFI) regarding how the practices of group purchasing organizations and drug wholesalers may be contributing to the shortages of generic drugs. “For years Americans have faced acute shortages of critical drugs, from chemotherapy to antibiotics, endangering patients,” FTC Chair Lina Khan said in issuing the RFI. The agencies are specifically looking for comments on the market concentration among the middlemen and details of their contracting practices.

US lawmakers warn of IP, national security threats posed by Wuxi

Citing national security concerns and alleging the support of human rights abuses, a bipartisan group of U.S. lawmakers is calling on three federal departments to investigate China’s Wuxi Apptec’s ties, and that of its subsidiary Wuxi Biologics, to the Chinese Communist Party (CCP) and the country’s People’s Liberation Army (PLA). “Wuxi Apptec and Wuxi Biologics have obscured their ties to the CCP and PLA and, as a result, are rapidly integrating themselves into U.S. supply chains by signing agreements with prominent U.S. biotech entities. . . . We are deeply concerned that Wuxi Apptec’s growing role in the global [contract research organization] market represents a backdoor means of transferring sensitive U.S. biotech IP to China,” the lawmakers said in a letter this week to the heads of the Commerce, Treasury and Defense Departments.

Also in the news

ABVC, Aim Immunotech, Amo, Annovis, Ascentage, Atara, Aurinia, Avicanna, Bristol Myers Squibb, Carrick, Celularity, Citius, Compugen, Dermavant, Firefly, Galderma, Greenwich, Grifols, Ipsen, Kalvista, Kiromic, Lexaria, Merrimack, Mindbio, Mink, Ocular, Oncolytics, Opthea, Precision, Prime Medicine, Projenx, Replicate, Santhera, Sunshine, Tevogen, Unravel, Xortx