Ambros' $125m for pain disorder drug, and other financings

Our roundup of financings in the biotech arena this week is led by a $125 million Series A for Ambros Therapeutics, a startup formed to develop a drug for a rare pain disorder that is already on the market in Italy.

Irvine, California-based Ambros hopes to bring the first medicine to market in the US for complex regional pain syndrome type 1 (CRPS-1), a rare and debilitating condition that can follow an injury or trauma to the bone and results in intense pain that can be continuous in the affected limb. There are no approved medicines for CRPS-1 outside of the Italian market, and there are around 65,000 new cases of the disorder each year in the US.

It has licensed rights to neridronate, a bisphosphonate drug marketed in Italy by Abiogen Pharma for CRPS-1 as well as osteogenesis imperfecta and Paget's disease. The candidate already has breakthrough therapy, fast track, and orphan drug designations for the treatment of CRPS from the FDA.

The first-round financing was co-led by RA Capital Management and Patient Square Capital's platform Enavate Sciences, and will fund a phase 3 trial (CRPS-RISE) of neridronate that is due to start enrolling subjects in early 2026. The drug is administered in a single cycle of four intravenous infusions over a 10-day period.

Also this week

In other recent financing announcements, Cambridge, Massachusetts-based Atavistik Bio raised $120 million in a Series B that will be spent in part on clinical trials of its lead drugs for inherited blood disorder hereditary haemorrhagic telangiectasia (HHT) and myeloproliferative neoplasms (MPNs). The round was led by Nextech Invest and The Column Group, with existing investor Lux Capital and new investor Regeneron Ventures also taking part.

Atavistik said that trials of an oral allosteric AKT1-selective inhibitor in HHT will start in the first half of next year, and will be followed by proof-of-concept testing for its MPN candidate, a JAK2 V617F mutant-selective inhibitor. There are no approved therapies for HHT, which affects 1.6 million people worldwide, while current therapies for MPNs – a group of rare blood cancers – are limited.

Orum Therapeutics said it raised KRW 145 billion – around $100 million – to advance a pipeline of degrader-antibody conjugates (DACs) for cancer and other diseases led by CD123-directed ORM-1153, a potential treatment for acute myeloid leukaemia (AML). The round was led by KB Investment along with existing and new investors, including Weiss Asset Management and Korea Investment Partners.

Boston, US and Daejeon, Korea-based Orum said the new funding will support the next stage of the company's growth and help it to advance multiple DACs into clinical development. Its pipeline also includes a DAC for small cell lung cancer (SCLC) and neuroendocrine tumours, against an undisclosed target, and Bristol Myers Squibb-partnered CD33-targeting BMS-986497 for AML and myelodysplastic syndrome (MDS).

A spinout from the University of California, Addition Therapeutics, has emerged from the shadows with $100 million bankrolled by SR One, Pivotal Life Sciences, Abingworth, Osage University Partners, the Gates Foundation, and BEVC, along with a mission to develop genomic medicines for chronic and rare diseases.

The company has been set up around an all-RNA, non-viral, lipid nanoparticle (LNP) technology platform – called PRINT (Precise RNA Mediated Insertion of Transgenes) – that promises to deliver "safer, durable, one-time therapies" that overcome some of the limitations of current genetic medicines. It has grown out of the lab of the University of California, Berkeley, scientists Prof Kathleen Collins, specifically her work on retrotransposon machinery to achieve precise transgene insertion at target genomic sites.

Link Cell Therapies – another California spinout, this time from Stanford University – formally launched this week with $60 million earmarked for the development of safer CAR-T therapies for both blood cancers and solid tumours. Its discovery platform draws on 'logic-gating technologies' to identify target antigens on cancer cells that are not expressed on healthy cells, or only found in minimal amounts.

Link's lead programme, LNK001 for the treatment of renal cell carcinoma, is being prepared for phase 1 clinical trials next year, with a follow-up candidate for colorectal cancer due to start human testing in 2027. The round was led by Johnson & Johnson's venture capital arm, with participation from Link Cell's founding investors – Samsara BioCapital and Sheatree Capital – as well as Wing Venture Capital and other new investors.

Finally, Berkeley, California startup Aeovian Pharma added $55 million to its cash reserves in a second round co-led by new investors Luma Group and CTI Life Sciences Fund. It will finance the completion of a phase 2 trial of AV078, Aeovian's mTORC1 inhibitor, in refractory epilepsy resulting from the rare genetic disorder tuberous sclerosis complex (TSC). The disease affects around 50,000 people in the US.

Other investors in the Series B included new investors Foresite Capital, SymBiosis, the TSC Alliance Endowment Fund, and Wilson Sonsini, along with previous backers Apollo Health Ventures, Hevolution, Sofinnova Investments and venBio.

Photo by Imani Bahati on Unsplash