VX-150

Lamenting the dearth of financing available for antibiotics research despite a growing demand for new products to fight drug resistance, little Waltham, MA-based X-Biotix has suspended its own R&D efforts in the field while continuing to try to hunt up fresh backing. Funded with a small A round in 2018, X-Biotix says it will now look to back up its IP and publish manuscripts as research work lapses. Antibiotics players have described a dire lack of funding and interest in the field, as payers continue to rely on old, cheap generics to fill most of the demand. X-Biotix CEO Ramani Varanasi has been working with chairman Steve Isaacs, who ran Aduro for years before tossing in the towel after a steady drumbeat of failures. “Policy makers must enact market-based reforms, including reimbursement reform and commercial ‘pull’ incentives, in order to revitalize the antibiotics market and drive sustainable investments in antibiotics R&D,” Isaacs noted in a statement. — John Carroll Vertex picks a pain drug for mid-stage studies Over a year after dropping their second attempt at a drug for pain, Vertex is back with a third. The big Cambridge biotech announced today that it’s moving a new molecule, VX-548, into Phase II studies for visceral and non-visceral acute pain. The molecule replaces an older candidate, VX-150, that yielded proof-of-concept data for reducing pain but which Vertex said lacked key features — such as safety and optimal dosing regimen — they wanted to see before bringing a molecule into mid or late-stage development. They scrapped the second attempt, VX-961, in January of 2020 after disappointing pharmacokinetic results in Phase I. VX-548 showed promising PK results in a Phase I study, Vertex said. The series of molecules are all designed to go after Nav1.8, a receptor that drug developers began pursuing in the mid-200s after a study showing that people with a rare mutation on the receptor experienced no pain. Those attempts, though, largely fell short as big pharma’s best attempts proved too weak or too toxic. Vertex’s VX-548 will be one of the first to reach mid-stage development and, if it bears out, could provide a potent, non-addictive alternative to opioids. — Jason Mast With clinical hold lifted, Voyager Therapeutics preps its gene therapy for a swift PhI/II launch About six months after putting a clinical hold on Voyager’s gene therapy for Huntington’s disease, the FDA is giving the biotech a green light to enter the clinic. The agency had requested more information about chemistry, manufacturing and controls (CMC), including “drug device compatibility and drug substance and product characterization,” according to Voyager’s 2020 financial report. With that cleared away, VY-HTT01 is expected to enter a Phase I/II study later this year. “Our investigational gene therapy has been designed to achieve broad knockdown of HTT mRNA throughout the brain via a one-time MRI-guided neurosurgical delivery,” CMO Omar Khwaja said in a statement. Back in 2019, Sanofi Genzyme deserted programs in an up to $845 million partnership with Voyager, walking away from an option to acquire US co-commercialization rights and ex-US development and commercialization rights to multiple candidates including VY-HTT01. The Phase I/II dose escalation study, dubbed VYTAL, will evaluate the safety and tolerability of VY-HTT01 in patients with early manifest Huntington’s disease. Secondary endpoints include disease biomarkers and clinical outcome measures. — Nicole DeFeudis I-Mab reads offers first look at how IL-6 inhibitor performed in PhII ulcerative colitis patients Ulcerative colitis patients who received I-Mab’s IL-6 inhibitor olamkicept saw significantly higher response rates than those who received a placebo, the company said on Monday. The 12-week results come from a Phase II study, which hit both primary and key secondary endpoints, according to I-Mab. While it hasn’t released any hard data, the biotech also said that more patients in the 600 mg group achieved clinical remission and mucosal healing than patients in the placebo group, with a p-value of less than 0.001. “This is the first demonstration that IL-6 blockade through the trans-signaling pathway plays a significant therapeutic role in UC,” principal investigator Minhu Chen said in a statement. A more detailed readout is coming during Digestive Disease Week and at the European Crohn’s and Colitis Organisation (ECCO) meeting in July 2021. — Nicole DeFeudis Carisma Therapeutics strikes CAR-M deal with University of Minnesota A month after dosing the first patients with its CAR-M therapy for HER2-overexpressing solid tumors, Carisma Therapeutics is joining forces with professor Bruze Blazar at the University of Minnesota to take its macrophage-based cell therapy research beyond oncology. “The collaboration with Dr. Blazar marks the initiation of the development of allogeneic, universal donor derived monocyte and macrophage cell therapies at CARISMA,” Michael Klichinsky, Carisma’s scientific co-founder and senior VP of research, said in a statement. “The focus of this multi-year collaboration will be optimizing and developing iPSC derived allogeneic chimeric antigen receptor macrophages, further expanding the potential of macrophage-based cell therapy for cancer and other diseases,” he said. The company is keeping the financial terms of the deal under wraps. — Nicole DeFeudis Bristol Myers Squibb expands on protein degradation deal with Evotec Bristol Myers Squibb is wading deeper into the protein degradation field by expanding a partnership with Evotec to identify new candidates for treating solid tumors. By extending the partnership, struck back in 2018, BMS triggered an undisclosed payment to Evotec to expand its screening efforts. The collaboration makes use of Evotec’s PanOmics platform, which combines enhanced throughput proteomics, high throughput transcriptomics and cell imaging with the company’s data analysis platform PanHunter. So far, two candidates generated through the partnership have transitioned to the lead optimization phase, according to Evotec. — Nicole DeFeudis Deerfield joins forces with accelerator to support academic research Through a partnership with a nonprofit accelerator, Deerfield Management is injecting more cash into academic research. The company announced on Monday that it’s joining forces with the Empire Discovery Institute (EDI), a nonprofit that works in partnership with research labs at the University of Rochester, the University at Buffalo, and Roswell Park Comprehensive Cancer Center. Deerfield says it plans on investing $65 million over the next five years. A newly launched company called Empire Deerfield Discovery & Development (ED3), will provide funding for projects in high-need areas, including hard-to-treat and rare diseases, according to a statement. “EDI was founded to help overcome the key challenges often faced by life science researchers in academia, namely a lack of external funding to advance pre-clinical development and access to pharmaceutical industry expertise to advance programs in an efficient manner to the clinic,” EDI CEO Martin Graham said in a statement. Deerfield has struck multiple academic partnerships over the years, including with the Broad Institute of MIT, Harvard, and the University of North Carolina, Chapel Hill. — Nicole DeFeudis *This article previously stated more patients in the treatment arm I-Mab’s Phase II trial achieved clinical remission and mucosal healing than patients in the placebo group, with a p-value of less than 0.01. The p-value is less than 0.001.