Verve Therapeutics Inc.’s heart disease candidate, VERVE-101, is the latest gene editing-based therapy to hit a snag at the FDA, which issued a clinical hold, delaying the start of phase I testing in the U.S. News of the hold, which followed preclinical presentations over the weekend at the American Heart Association 2022 meeting, sent shares of Verve (NASDAQ:VERV) falling 30.5% to close Nov. 7 at $21.75.
Heading into the readout of the phase III Mont Blanc study testing Nicox SA’s NCX-470, a nitric oxide-donating version of prostaglandin analogue bimatoprost, analysts were looking for a superior intraocular pressure-lowering profile vs. latanoprost, predicting such data could position the drug as a new standard of care in a crowded market.
The U.S. FDA declined to approve Gilead Sciences Inc.’s Hepcludex (bulevirtide), issuing a complete response letter (CRL) citing manufacturing and delivery concerns for the antiviral aimed at treating confirmed chronic hepatitis delta virus (HDV) infection in adults with compensated liver disease. Acquired in Gilead’s 2021 buyout of Myr GmbH, Hepcludex received conditional approval from the European Commission in 2020 and would have been the first drug cleared for HDV in the U.S. Its delay, however, could give a boost to Eiger Biopharmaceuticals Inc., which is expected to report phase III data for lonafarnib by the end of 2022.
The U.S. FDA cleared teclistamab from Janssen Pharmaceutical Cos. as the first bispecific antibody for treating patients with relapsed or refractory multiple myeloma (MM), joining other BCMA-targeted drugs, including an antibody-drug conjugate and CAR T therapies.
It’s been a long road, but Astrazeneca plc’s anti-CTLA4 antibody, tremelimumab, finally earned its first U.S. FDA nod, cleared for use in combination with anti-PD-L1 drug Imfinzi (durvalumab) to treat patients with unresectable hepatocellular carcinoma (HCC). The commercial impact of the dual checkpoint therapy, however, remains to be seen, as it goes up against Roche Holding AG’s combination of Avastin (bevacizumab) and Tecentriq (atezolizumab), which gained standard-of-care status in first-line HCC in 2021.
Mirum Pharmaceuticals Inc. plans to “move rapidly toward regulatory filings” to expand use of IBAT inhibitor Livmarli (maralixibat) into progressive familial intrahepatic cholestasis (PFIC), President and CEO Chris Peetz told investors during a conference call Oct. 24 to discuss the positive top-line results from the phase III March study, which not only hit its primary endpoint in patients with PFIC2 but showed statistical improvements in pruritis across a range of PFIC subtypes.
With roughly a decade of work behind it, Valitor Inc. is ready to put its Multivalent Polymer (MVP) platform to the test, having recently announced its presence with a $28 million series B round and appointment of biopharma veteran Steven Lo as CEO. The next step is IND-enabling studies for its lead ophthalmology candidate, with clinical testing slated to start in 2024.
Over the next two weeks, the team at NGM Biopharmaceuticals Inc. will be digging through the data from its phase II trial of complement 3 inhibitor NGM-621 in patients with geographic atrophy (GA) secondary to age-related macular degeneration, following reports of a top-line miss Oct. 17 and a severe reaction from investors, who sent shares (NASDAQ:NGM) plummeting 70.5% to close at $3.41.
Biocryst Pharmaceuticals Inc. caught some industry observers by surprise early in 2022 when it upped its peak sales estimate for Orladeyo (berotralstat), its oral therapy for prevention of hereditary angioedema attacks, from $500 million to $1 billion. But blockbuster status appears more attainable following the latest setback from a potential competitor, as safety concerns prompted Kalvista Pharmaceuticals Inc. management to ax its phase II study testing KVD-824 in prevention of HAE attacks.
At first glance, Cellarity Inc. might appear as one more company harnessing the computational power of AI and machine learning to boost drug discovery efforts. A closer look, however, reveals a different approach, one that looks at cells and cellular behavior to address disease rather than the traditional method of seeking out molecular targets.
