Morphic Therapeutic, Inc.

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )--SiteOne Therapeutics, Inc., a biopharmaceutical company developing selective ion channel modulators for the treatment of pain, cough, and other conditions, today announced a $100 million Series C financing. Led by Novo Holdings, with participation from OrbiMed, Wellington Management, Mission BioCapital, BSQUARED Capital, and existing investors, the financing will support the advancement of SiteOne's portfolio of selective, small molecule ion channel modulators for the treatment of sensory hyperexcitability disorders through human clinical proof of concept. In conjunction with the financing, SiteOne Therapeutics appointed to its Board of Directors: Ken Harrison, Ph.D., Senior Partner at Novo Holdings; Mona Ashiya, Ph.D., Partner at OrbiMed; and Zach Collins, Ph.D., Partner at Mission BioCapital. In addition, Praveen Tipirneni, M.D., most recently CEO of Morphic Therapeutic, has joined the Board as Executive Chairperson, and Martin Edwards, M.D., an experienced life science advisor, has joined as an independent Board member. “SiteOne Therapeutics is committed to developing non-opioid treatments for conditions that involve hyperexcitability of the peripheral nervous system such as pain and cough. Our strategy is focused on owning the action potential, the fundamental unit of electrical signaling in the peripheral nervous system. This funding will enable human proof of concept studies on our isoform-selective sodium channel inhibitors and development of other ion channel modulators,” commented John Mulcahy, Ph.D., President and Chief Executive Officer of SiteOne Therapeutics. “SiteOne welcomes Ken Harrison, Mona Ashiya, Zach Collins, Martin Edwards, and Praveen Tipirneni as members of our Board of Directors. Their collective expertise in drug development, commercial strategy, capital markets and business development will be pivotal as we build out our team and advance our small molecule candidates in the clinic.” SiteOne’s approach is grounded in the development of selective best-in-class sodium channel inhibitors, including Na V 1.7 and Na V 1.8. The company’s lead program for pain targets the Na V 1.8 channel, which is primarily located in the peripheral nervous system, and has the potential to provide meaningful analgesia with a reduced side effect profile and risk of abuse compared to current, central nervous system-focused standard-of-care. Beyond pain, SiteOne is exploring the potential of sodium channels and other genetically and clinically validated targets to treat pain and sensory hyperexcitability disorders. Ken Harrison, Senior Partner, Novo Holdings, stated, “SiteOne Therapeutics’ portfolio of selective ion channel modulators holds immense promise for the non-opioid treatment of pain. SiteOne has built upon years of research on the contribution of ion channels to peripheral nerve excitability, and we are very encouraged by the company’s underlying science and its mission to transform pain management for patients living with chronic pain in the US and around the globe.” “While many companies are focused on drug discovery for the central nervous system, SiteOne is instead pursuing a pipeline of therapeutics that specifically target the peripheral nervous system,” added Praveen Tipirneni. “By targeting peripheral sensory neurons directly, SiteOne is positioned to develop complementary, robust treatments for pain and other sensory hyperexcitability disorders without the centrally mediated side effects of existing therapeutics. I am excited to join SiteOne as they continue to develop these important therapeutics for the potential benefit of millions of patients.” About Novo Holdings A/S Novo Holdings is a holding and investment company that is responsible for managing the assets and the wealth of the Novo Nordisk Foundation. The purpose of Novo Holdings is to improve people’s health and the sustainability of society and the planet by generating attractive long-term returns on the assets of the Novo Nordisk Foundation. Wholly owned by the Novo Nordisk Foundation, Novo Holdings is the controlling shareholder of Novo Nordisk A/S and Novonesis A/S (Novozymes A/S) and manages an investment portfolio with a long-term return perspective. In addition to managing a broad portfolio of equities, bonds, real estate, infrastructure and private equity assets, Novo Holdings is a world-leading life sciences investor. Through its Seed, Venture, Growth, Asia, Planetary Health and Principal Investments teams, Novo Holdings invests in life science companies at all stages of development. As of year-end 2023, Novo Holdings had total assets of EUR 149 billion. www.novoholdings.dk About SiteOne Therapeutics SiteOne Therapeutics is a biopharmaceutical company advancing a novel class of highly selective small molecule inhibitors targeting Na V 1.7, Na V 1.8, and other voltage-gated ion channels to treat sensory hyperexcitability disorders such as chronic cough, itch, and pain. Since its inception, SiteOne has been dedicated to the development of safe and effective pain therapeutics without the significant addiction potential and side effects of opioids. The company is also advancing additional novel drug candidates that exhibit precise selectivity for individual ion channel subtypes to treat other sensory hyperexcitability disorders such as chronic cough and chronic ocular surface pain. For more information, visit www.siteonetherapeutics.com .

Kronos Bio is letting go nearly all its employees after flashing red lights earlier this month. As part of an 83% workforce downsizing, CEO Norbert Bischofberger is heading out the door and handing the keys to recently hired finance and operating chief Deborah Knobelman. The biotech had eliminated a trio of executives in January and conducted a couple of rounds of layoffs this year and last year . The latest round of layoffs will “substantially” wrap up by Dec. 31, the company said. Bischofberger was the fifth employee at Kronos, and he’s been with it since nearly the beginning, having taken part in the startup’s seed round back in 2018 after a three-decade R&D career at Gilead. “Norbert’s steadfast leadership was instrumental in the founding and building of Kronos Bio,” chair Arie Belldegrun said in a release. Bischofberger’s resignation takes effect Dec. 3, and he’ll continue on the board, according to an SEC filing. His decades of expertise will continue elsewhere: He recently joined the board of Vir Biotechnology and is also a scientific advisor to a new VC firm trying to build up the biotech ecosystem in his native Austria. He was also on the board of Morphic until Eli Lilly bought it for $3.2 billion this summer. Kronos said earlier this month it would seek “strategic alternatives” after looking at data for its CDK9 inhibitor. It determined additional development wasn’t warranted. The biotech had inked a $20 million upfront discovery deal with Genentech last year. At the end of September, the biotech had $124.9 million in cash, equivalents and investments, meaning it could be a target for a reverse merger. There were 10 biotech reverse mergers through the first nine months of 2024, according to a William Blair third-quarter report. At least five more have been disclosed since early October. Kronos went public at $19 per share $KRON in a $250 million IPO in October 2020. It trades below $1 today.

Novartis will pay $150 million upfront to Schrödinger, in a pact announced Tuesday to work together on several preclinical drug programs. Founded in 1990, Schrödinger has been developing and selling its physics-based R&D software to drugmakers like Novartis for decades, outlasting multiple waves of excitement over AI that have come in and out of its space. This latest deal is centered on multiple drug targets that both companies were researching. Schrödinger is not disclosing the targets beyond saying they are highly validated with compelling genetics or human biological evidence and fall outside oncology. “Schrödinger and Novartis are going to combine our efforts on these targets to finish discovery, to identify development candidates, and then Novartis will take on the further development of these programs,” Karen Akinsanya, Schrödinger’s president of R&D, therapeutics, said in an interview. Beyond $150 million upfront, which Schrödinger expects to recognize as revenue over the course of the research, the deal outlines potential payments of up to $892 million for R&D and regulatory achievements and up to $1.38 billion more for possible commercial milestones. Novartis is also ramping up its broader usage of Schrödinger software through a second agreement that includes an undisclosed increase in how much Novartis pays for the software. That fits a trend Schrödinger CEO Ramy Farid sees in pharma giants shifting from using its software as a way to explore or understand experiments to using it at a greater scale, where calculations are relied on more than traditional methods. He pointed to the success of biotech startups like Nimbus Therapeutics , Morphic Therapeutic , Ajax Therapeutics and Structure Therapeutics — all heavy users of Schrödinger software from the start. “I don’t think that can be ignored, and it looks like the industry is not ignoring it anymore,” Farid said. “So now you have companies like Lilly and BMS and Novartis scaling up their usage in a really meaningful way, but we’re in the early days of it. There are many, many more companies that will, I think, transition in the way that Novartis just did.” Schrödinger’s challenge will be turning that vision into a reality that impacts the top line. The company’s software revenue for the third quarter, also announced Tuesday morning, came in below expectations at $31.9 million, up 10% from a year ago. The $1.4 billion biotech has seen its stock price fall 44% this year, as investors await clinical readouts from its own drug pipeline expected in 2025.