Vertex and CRISPR send exa-cell for FDA approval; Aridis Therapeutics axes 20% of its workforce

03 Apr 2023
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Vertex and CRISPR Therapeutics have finished the BLA for its treatment exagamglogene autotemcel, or exa-cel, and will be going toward the FDA for approval. Exa-cell is intended to treat sickle cell disease and transfusion-dependent beta-thalassemia. The BLA also has a priority review attached and, if given, could bring down the FDAs review of the application to eight months instead of 12 months. Vertex and CRISPR are backing the submission with data from two Phase III trials and a follow-up study. “Within a decade, we have progressed from the discovery of the CRISPR platform to the first regulatory filings for a CRISPR-based therapy, which speaks to the transformative nature of CRISPR technology,” said Phuong Khanh Morrow, CRISPR Therapeutics CMO in a release . — Tyler Patchen Aridis Pharmaceuticals laid off seven of 33 employees after AstraZeneca terminated a license for its drug candidate being studied to prevent ventilator-acquired pneumonia. “While we seek a remedy to the license dispute, we want to assure our shareholders that we remain steadfastly committed to developing our pipeline products as potential breakthrough therapies to fight antimicrobial resistance,” Aridis CEO Vu Truong said in a statement Friday night. With AstraZeneca scrapping the agreement, Aridis’ European-funded Phase III went back onto the shelf. The monoclonal antibody, known as AR-320, entered the late-stage clinical trial last fall. — Kyle LaHucik Arrowhead Pharmaceuticals has roped in a further $30 million in a milestone payment from GSK. What triggered the payment was the pharma kicking off the Phase IIb trial of the GSK4532990, formerly known as ARO-HSD, an RNAi therapeutic to treat NASH. The drug is being developed between GSK and Arrowhead since both signed an exclusive license to develop and commercialize the candidate in all territories except Greater China, where Arrowhead retains the rights. GSK is responsible for the development and commercialization of areas outside of China. The trial itself will look at the safety and efficacy of the drug in adults and plans to enroll up to 246 participants. — Tyler Patchen A Boston biotech recently joined the growing list of shuttered startups in drug development. Samus Therapeutics closed shop, per updates to two clinical studies on the US federal trials database. Based on work out of Memorial Sloan Kettering Cancer Center, Rockefeller University and Weill Cornell Medicine, Samus was trying to go after the epichaperome. The protein complex is thought to be involved in cancers, neurological disorders and chronic traumatic encephalopathy, or CTE, the brain condition that became a focus of attention after many former NFL players were found to have it. Samus axed a Phase II study of its oral drug candidate zelavespib, or PU-H71, for adults with myeloproliferative neoplasm. The company also terminated a Phase I testing icapamespib, or PU-AD, in patients with recurrent brain lesions. The private startup had also set out to make diagnostics to go along with its investigational therapeutics. CEO Dick Bagley declined to comment. The company was previously led by Jonathan Lewis. Both executives formerly held leadership roles at Ziopharm Oncology, now known as Alaunos Therapeutics . — Kyle LaHucik The Swiss biotech Covis Finco Sarl has entered an agreement to recapitalize itself and reduce its debt by an estimated $450 million, with 95% of its first lien lenders and 100% of its second lien lenders. The release stated that the move will boost Covis’s balance sheet and its liquidity. Once the recapitalization is finished, the funds will be managed by an affiliate of Apollo Global Management, who will remain the majority owners, and the first lien lenders will be handed a minority stake. According to Covis, this will put the biotech on a path to scale up its business, work on its existing portfolio, and grow physically. It also said it was pursuing “inorganic growth opportunities” but did not specify what those options could be. “At Covis, our paramount focus continues to be patient outcomes and improving the health of people with serious medical conditions,” said Michael Porter, CEO of Covis, in a release . — Tyler Patchen The Massachusetts-based liquid biopsy biotech Mercy BioAnalytics has closed a $41 million Series A round. The investment will be going towards development and commercialization efforts for its Mercy Halo testing platform, which plans to look for cancer-related biomarkers on the surface of cancer-derived extracellular vesicles. The platform specifically will be used for high-risk lung cancer screening. The cash will also be going towards moving along other clinical programs in other indications, such as ovarian cancer. “A simple, inexpensive blood-based screening test could be a vital new tool to significantly increase patient engagement in lung cancer screening, creating the opportunity to save lives through early detection,” said Mercy BioAnalytics CEO Dawn Mattoon in a release . The round was led by Novalis LifeSciences and had participation from the American Cancer Society BridgtEdge, Sozo Ventures, Hatteras Venture Partners, iSelect Fund and Broadway Angels. There were also investments from Bruker and Labcorp. — Tyler Patchen
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