Cassava being investigated by unnamed 'government agencies'; Rocket Pharma gives updated data on Danon disease
Cassava Sciences likes to fight fire with fire, with CEO Remi Barbier calling a citizen petition to halt development on its lead drug a “short attack” in September. But the noise surrounding the saga has apparently drawn the attention of the US government.
The biotech reported in an SEC filing Monday that “certain government agencies” have asked Cassava to provide them with “corporate information and documents.” It’s not clear which agencies are involved in probing Cassava, or if multiple agencies are doing so, but the company says it’s cooperating.
Though Cassava reported that the government said it had not informed the biotech that any wrongdoing had occurred, the company left the door open to potential penalties.
“We cannot predict the outcome or impact of any [of] these ongoing matters, including whether a government agency may pursue an enforcement action against us or others,” Cassava wrote in the filing.
Cassava was originally accused by the law firm Labaton Sucharow in August of data manipulation, with the shareholder rights activists petitioning the FDA to stop development of the Alzheimer’s drug simufilam. The biotech fought back with Barbier’s comments as well as an unusual Q-and-A style press release put out around that time. — Max Gelman
Rocket Pharma reports updated PhI data for Danon disease
New Jersey biotech Rocket Pharmaceuticals has been working on an investigational gene therapy for gene disorder Danon disease — and the pharma announced updated data from the Phase I trial today.
The trial was looking at a single IV infusion of gene therapy RP-A501 at two different dosages in five patients — “low-dose” with three patients (6.7e13 vg/kg) and “high-dose” with the other two (1.1e14 vg/kg). While the gene therapy was well-tolerated at the low dose and conferred sustained clinical benefit, the same can’t be said for the high dose.
As previously made public, dose-dependent toxicity was seen in one of the two patients on the high dose regimen — and developed thrombotic microangiopathy that fully resolved with treatment, which included transient hemodialysis.
As such, the biotech will be cutting off the higher dosage and sticking with the lower amount moving forward.
“We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Rocket CEO Gaurav Shah. “We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as rapidly as possible toward Phase II.” — Paul Schloesser
Motric Bio launches from ex-Novartis CEO’s investment shop
After Novartis’ former CEO Joe Jimenez and NIBR’s former president Mark Fishman co-founded Aditum Bio in 2019, they are launching another offshoot — for the seventh time — with the launch of Motric Bio in a partnership with Motorpharma.
In a joint statement, the companies announced the formation of Motric Bio, a portfolio company developing a new therapy for spasticity associated with neurological injuries and disease. Motric Bio successfully in-licensed a myosin-2 inhibitor from Motorpharma — and that inhibitor has shown selective inhibition of fast skeletal muscle.
“This molecule has shown selectivity to fast skeletal myosin-2, with practically no inhibition of cardiac and smooth muscle myosins,” Fishman said in a company statement. “We believe this could lead to greater efficacy and fewer side effects than the current standard of care. We look forward to advancing this important therapy into the clinic to help these patients.”
“This molecule is the result of over 20 years of research to develop selective myosin-2 inhibitors,” said Motorpharma CEO Anna Rauscher. “We are pleased to partner with Aditum Bio to form Motric Bio, enabling this drug to enter the clinic to help patients in need.” — Paul Schloesser
InFlaRx’s mAb for autoimmune disease shows positive results
German biopharma InFlaRx announced positive results from its Phase II study of vilobelimab, a anti-C5a antibody in patients with ANCA-associated vasculitis. Patients went into remission at similar rates to standard of care, while using much less glucocorticoids — translating to an efficacy and safety profile investigators said they were encouraged by.
The primary efficacy evaluation was defined by a 50% reduction on the Birmingham Vasculitis Activity Score (BVAS) and no worsening in any body system from baseline. Clinical remission was the secondary endpoint.
“We are pleased to report that vilobelimab monotherapy has shown similar efficacy compared to a standard dose of glucocorticoids in patients with ANCA-associated vasculitis in this trial,” chief development officer Korinna Pilz said in the press release. “Vilobelimab treatment led to a considerably reduced use of glucocorticoids, resulting in remarkably less treatment emergent adverse events and a reduction in the glucocorticoid toxicity index.”
ANCA-associated vasculitis is an autoimmune disease that affects small blood vessels in the body. Vilobelimab is believed to be the first monoclonal anti-C5a antibody introduced into clinical development. Over 300 people have been treated with vilobelimab in completed clinical trials, the company says. — Josh Sullivan
Shooting for clinic in 2022, Athos raises $15M in Series A
Athos Therapeutics closed its Series A on Monday.
The biotech raised $15 million in the oversubscribed round, aiming to launch a Phase I trial of its lead compound in 2022. That candidate, ATH-63, is being developed for “inflammatory bowel disease, lupus, other autoimmune disorders, and select cancer indications,” Athos said.
“I am delighted at the progress Athos has made since our Seed round of $4.25MM in March of 2020,” CEO Dimitrios Iliopoulos said in a statement. “Those seed funds enabled us to hire a stellar team, develop a powerful drug discovery engine, perform multi-omics analysis of annotated patient samples from key IBD centers around the world, file 8 patents, and create a medicinal chemistry platform that produced 100’s of highly selective and safe compounds with excellent pharmacologic profiles.” — Max Gelman