Technology of genomically recoded organisms borne out of Yale and Stanford university laboratories and housed at Khosla Ventures-backed Pearl Bio received validation on March 12 through a $1 billion deal signed with Merck & Co. Inc. Cambridge, Mass.-based Pearl, which emerged from stealth last year, is eligible for the funds through up-front, option and milestone payments, plus potential royalties on sales of deal-related products that gain approval. The synthetic biology company aims to create a new class of multi-functionalized therapeutics with tunable properties. The deal with Rahway, N.J.-based Merck will focus on new cancer biologics.
Top-line phase II results of privately held Ocuterra Therapeutics Inc.’s novel selective small-molecule RGD integrin inhibitor, nesvategrast (OTT-166), showed it failed to meet primary and key secondary endpoints in patients with diabetic retinopathy, prompting the Boston-based company to seek strategic alternatives.
Phase I data of oral amycretin, a co-agonist of GLP-1 and amylin receptors from Novo Nordisk A/S, showed a 13.1% reduction in body weight at 12 weeks vs. 1.1% for placebo, pushing shares of the Bagsvaerd, Denmark-based company to their highest levels to date.
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%.