Roche Holding AG’s investigational monoclonal antibody, crovalimab, which inhibits part of the innate immune system in patients with the rare blood condition paroxysmal nocturnal hemoglobinuria (PNH), met its co-primary efficacy endpoints in a phase III trial that will form the basis of its approval submissions worldwide. Crovalimab binds to C5, the last step of the complement cascade, so preventing the destruction of red blood cells, which in PNH are destroyed by the complement system, resulting in symptoms such as anemia, fatigue, blood clots and kidney disease.

Fly like an eagle: Garuda brings in a $62M series B

Garuda Therapeutics Inc. has raised a $62 million series B financing as it drives toward treatments that replace cell donation with the ability to generate off-the-shelf, self-renewing blood stem cells. The company, which said it could potentially treat and possibly more than 120 diseases, has now raised a total of $134 million. The financing was led by Northpond Ventures, Orbimed Advisors, Cormorant Asset Management and Aisling Capital, with participation from Sectoral Asset Management, Mass General Brigham Ventures and Alexandria Venture Investments. The company was named after Garuda, the mythological eagle that was the steed of Vishnu and who was considered the king of birds.

BIO CEO 2023: Mitigating the impacts of the US Inflation Reduction Act

With the passage of the Inflation Reduction Act (IRA) in the U.S. in August 2022, biopharma company leaders have re-evaluated pipelines, sought legal advice, and discussed ways to mitigate the potential impacts the legislation will have on pricing therapies and extending their reach to new indications. A panel of executives seemed to agree on Feb. 7 at the BIO CEO 2023 meeting in New York that the IRA – although it includes some positive aspects such as a focus on clean energy and caps on out-of-pocket patient costs – forces companies to justify the value of their products and ultimately dis-incentivizes innovation. “This is litigation directly hampering science,” said Peter Thompson, a private equity partner with Orbimed Advisors LLC.

Tilt Biotherapeutics adds $13M as clinical readouts loom

With readouts from three clinical studies of lead oncolytic virus TILT-123 due later this year, Tilt Biotherapeutics Ltd. secured €12 million (US$12.9 million) in new funding, to add to the €10 million it raised last June. The European Commission supplied the bulk of the additional cash, €5.9 million of it as equity investment from the European Innovation Council (EIC) fund and another €2.1 million in grant funding from the EIC’s accelerator program. That will enable the company to complete the ongoing studies, to prepare for a phase II in ovarian cancer, its lead indication, and to take forward several preclinical programs that are nearing the clinic. Helsinki, Finland-based Tilt is among a large cluster of contenders seeking to make the case for oncolytic virus therapy, a modality that, up to now at least, has flattered to deceive. Despite its patchy record so far, there is still wide open territory for any therapeutic modality that can make credible inroads in solid tumor indications.

Australia to allow psychiatrists to prescribe psilocybin and MDMA for depression, PTSD 

Beginning July 1, Australia’s Therapeutic Goods Administration (TGA) will allow medicines containing the psychedelic substances psilocybin and MDMA (3,4-methylenedioxy-methamphetamine) to be prescribed by authorized psychiatrists for certain mental health conditions. Specifically, the TGA will permit prescribing MDMA for treating post-traumatic stress disorder (PTSD) and psilocybin for treatment-resistant depression. “These are the only conditions where there is currently sufficient evidence for potential benefits in certain patients,” the TGA said.

Nodus adds funding to advance PARG inhibitor, other DDR programs

Nodus Oncology Ltd. closed on the first £2.4 million (US$2.9 million) of £12 million it expects to raise in a number of tranches over the next 12 months, to source and take forward third-generation DNA damage response (DDR) programs. The new funding will be applied to accelerate the lead poly (ADP-ribose) glycohydrolase (PARG) inhibitor, around which Nodus was formed with seed funding in September 2022. At the same time, Nodus will continue to bring in other recently discovered third wave DDR targets that are thought to play an important role in response to DNA damage.

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Aceragen, Accutar, Addex, Ashvattha, Atyr, Biogen, Bioxytran, Certa, Context, Curevac, Daewoong, Defence, Egret, Endogena, Evonetix, Gilead, Gracell, Hinova, Idorsia, Immutep, Inmagene, Janssen, Lyra, Neogap, Neurosense, Novartis, Pulmatrix, Sage, Sandoz, Seelos, Spyglass, Takeda, Tonix, Vera