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.
Abpro Corp. and Celltrion Healthcare Inc. entered a global partnership valued at up to $1.75 billion involving ABP-102, a bispecific antibody targeting HER2-positive cancers, a move Abpro CEO Ian Chan said would help “accelerate this type of therapy for patients in need.”
Shares of Lava Therapeutics NV rocketed by more than 90% Sept. 26 as the company disclosed a licensing deal with Seagen Inc. to develop and commercialize preclinical-stage EGFR-targeting bispecific candidate LAVA-1223, which comes with $50 million in up-front funding and up to a potential $650 million in milestones. It also adds further validation for harnessing gamma-delta T cells to treat cancer, an approach that is growing increasingly popular.
Abpro Corp. and Celltrion Healthcare Inc. entered a global partnership valued at up to $1.75 billion involving ABP-102, a bispecific antibody targeting HER2-positive cancers, a move Abpro CEO Ian Chan said would help “accelerate this type of therapy for patients in need.” With ABP-102 in preclinical development, Abpro decided to start looking for a partner. HER2 is “one of the biggest targets in biotech, mostly relevant for breast, colorectal and gastric cancers,” Chan told BioWorld. “Celltrion happened to have a lot of experience in the space.”
Bluebird Bio Inc.’s elivaldogene autotemcel (eli-cel) gained U.S. approval late Sept. 16 for use in early active cerebral adrenoleukodystrophy (CALD), making it the firm’s second gene therapy to clear the FDA in as many months. Branded Skysona, eli-cel is expected to be available commercially by the end of 2022 and its launch will require only “incremental” company resources on top of those required for the ongoing launch of beta-thalassemia gene therapy Zynteglo (betibeglogene autotemcel), Bluebird said.
Bearish investors dwelling on a single grade 4 liver enzyme elevation seemed to be the cause for Intellia Therapeutics Inc.’s sinking stock Sept. 16, despite the company reporting impressive, though early stage, data for its leading systemically administered CRISPR candidates targeting hereditary angioedema (HAE) and amyloid transthyretin (ATTR) amyloidosis.
