The RNAi therapeutic zilebesiran, which was the focus of a 2023 $2.8 billion deal between Alnylam Pharmaceuticals Inc. and Roche Holding AG, demonstrated significant systolic blood pressure reductions in patients with mild to moderate hypertension who were part of the Kardia-2 phase II study.
Despite what appears to be a recovering public financing market for the biopharma industry, with about $20 billion raised so far through follow-on offerings in the first two months of 2024, emerging companies remain in that precarious position between dwindling cash and their next milestone inflection points.
San Diego-based Kenai Therapeutics Inc. raised $82 million in a series A round to move its disease-modifying cell therapy for Parkinson’s disease into the clinic. The company, which leverages induced pluripotent stem cell (iPSC) technology, will advance its next-generation allogeneic neuron replacement cell therapies for neurological diseases, specifically completing a clinical proof-of-concept trial for its lead candidate, RNDP-001. The series A was co-led by Alaska Permanent Fund Corp., Cure Ventures and The Column Group, with participation from Euclidean Capital and Saisei Ventures. Proceeds will enable Kenai to submit an IND for RNDP-001 and bring it through the completion of phase I trials, which are expected to begin sometime in 2024.
San Diego-based Kenai Therapeutics Inc. raised $82 million in a series A round to move its disease-modifying cell therapy for Parkinson’s disease into the clinic. The company, which leverages induced pluripotent stem cell (iPSC) technology, will advance its next-generation allogeneic neuron replacement cell therapies for neurological diseases, specifically completing a clinical proof-of-concept trial for its lead candidate, RNDP-001.
Strong early data of Janux Therapeutics Inc.’s tumor-activated T-cell engager compounds JANX-007 and JANX-008 sent the San Diego-based company’s shares surging by 229%, indicating enthusiasm for their best-in-class potential and the possibility of tapping into multibillion-dollar prostate cancer and solid tumor markets.
Blueprint Medicines Corp. found a new U.S. commercialization home for Gavreto (pralsetinib) through an agreement with Rigel Pharmaceuticals Inc. potentially worth $117.5 million, now that Roche Holding AG has relinquished all rights. The product, a once-daily oral small-molecule kinase inhibitor of wild-type RET (rearranged during transfection) and oncogenic RET fusions, received accelerated approval in 2020 by the U.S. FDA, under priority review and with orphan drug designation, to treat adults with metastatic RET fusion-positive non-small-cell lung cancer.
Alnylam Pharmaceuticals Inc.’s decision to change its analysis plan for the phase III Helios-B trial of RNAi therapeutic Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy, pushing top-line results back by three months, pressured its share price (NASDAQ:ALNY) down by 10% on Feb. 15, while also boosting shares of competitor Bridgebio Pharma Inc. (NASDAQ:BBIO) by 14%.
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.
Weeks after Roche Holding AG paid a $40 million milestone payment to partner Repare Therapeutics Inc. when the first cancer patient in the phase II Tapistry trial was dosed with camonsertib, the Basel, Switzerland-based pharma backed out of the 20-month-old deal.
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
