Akashi Therapeutics Inc., of Cambridge, Mass., said the FDA notified the company that a review was completed and Akashi may continue clinical development of HT-100 (delayed-release halofuginone) for Duchenne muscular dystrophy (DMD). The research involves studying those who have genetic mutations that cause DMD, a rare muscle disease that affects boys. Akashi said it will start a new study, HALO-DMD-04, as soon as possible.
Aprea Therapeutics AB, of Boston and Stockholm, said it has cemented a collaboration with Memorial Sloan Kettering Cancer Center in New York to study the effects of reactivation of tumor suppressor protein p53 by APR-246. The aim of the collaboration is to determine preclinical efficacy of APR-246 when combined with various other anticancer agents and against multiple tumor types. Financial terms were not disclosed.
Avrobio Inc., of Cambridge, Mass., said it added a Gaucher disease program to its pipeline. The late-stage clinical program was licensed from Lund University in Sweden, which had shown preclinical proof of concept. The gene therapy is Avrobio's second therapy for lysosomal storage disorders. The company said it is now preparing for phase I/II trials. Financial terms were not disclosed.
Chemocentryx Inc., of Mountain View, Calif., said avacopan, its treatment for C3 glomerulopathy (C3G), has been designated an orphan drug by the FDA. Avacopan targets the complement C5a receptor, or C5aR, which drives the destructive inflammatory cells in C3G and other kidney diseases.
Cue Biopharma Inc., of Cambridge, Mass., reported that it has selected its lead candidate, Cue-101, targeting human papillomavirus (HPV)-associated cancers. Cue anticipates the drug to begin clinical trials in the first half of 2018. The company said that Cue-101 showed antitumor activity in HPV-driven cancer models in preclinical research.
Cytokinetics Inc., of South San Francisco, reported preclinical data showing that CK-2127107, a fast skeletal troponin activator that it's developing with Astellas Pharma Inc., of Tokyo, improves muscle function in mouse models of spinal muscular atrophy. "The increased muscle force at sub-maximal nerve stimulation frequencies in mice inform the potential for CK-2127107 to increase muscle function in patients living with motor neuron dysfunction," said Fady Malik, Cytokinetics' research and development chief. The study outcome was presented at the MDA Scientific Conference in Arlington, Va.
Hemostemix Inc., a Blackfalds, Alberta-based cell therapy company, said that it is continuing to have trouble securing investment in the company, an effort complicated by unresolved active litigation and the status of its phase II trial of an experimental therapy for patients with critical limb ischemia. "Serious concerns have been raised as to the integrity of the data collected to date, as well as the continuing usefulness (or lack thereof) of samples and data relating to patients being treated" when Criterium Inc. terminated a clinical services agreement with the company, it said. Company shares (TSX:HEM), which traded at a 52-week high of C45 cents (US34 cents) closed Wednesday at C$0.04, down 60 percent.
Innoviva Inc., of Brisbane, Calif., appealed to shareholders to reject a bid by Sarissa Capital Domestic Fund LP to gain three of the seven seats on its board during the company's upcoming annual meeting, April 20. In a proxy filing with the Securities and Exchange Commission, Sarissa alleged that given the nature of Innoviva's business, it "has a bloated cost structure and grossly overpays its officers and directors," a claim that Innoviva countered at length in its own proxy materials and a press release, issued Wednesday.
Moleculin Biotech Inc., of Houston, gained FDA orphan status for its lead candidate, annamycin, for the treatment of acute myeloid leukemia (AML). The candidate is a liposome formulated anthracycline that has been designed to produce little to no cardiotoxicity and avoid the multidrug-resistance mechanisms that often defeat current anthracyclines, the company said. It is working with the FDA on an investigative new drug application for a phase I/II trial for second-line treatment of relapsed or refractory AML.
Nemus Bioscience Inc., of Costa Mesa, Calif., and its discovery and research partner, the University of Mississippi, said that, during in vitro studies, they achieved "bactericidal synergy" against multiple species of methicillin-resistant Staphylococcus aureus (MRSA) using a proprietary cannabinoid-based therapeutic platform. MRSA species tested included community acquired, healthcare-acquired, and mupirocin-resistant strains.
Opko Health Inc., of Miami, gained FDA orphan status for oligonucleotide-based Antagonat (CUR-1916) for the treatment of Dravet syndrome. The Antagonat technology, part of Curna Pharmaceuticals, acquired by Opko in 2011 for $10 million. It was further developed in Opko's Miami research laboratories. (See BioWorld Today, Feb. 2, 2011.)
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., filed a lawsuit on Monday in U.S. District Court District of Massachusetts against Amgen Inc. and Immunex Corp. regarding Sanofi's and Regeneron's Dupixent (dupilumab), an eczema treatment. The two companies are seeking a court order that declares Dupixent does not infringe Amgen's U.S. Patent No. 8,679,487 for AMG-317, a failed asthma treatment. The filing Monday follows a string of previous legal actions for those companies. Dupixent is also being studied in asthma and other indications. (See BioWorld Today, Feb. 10, 2017.)
Shire plc, of Dublin, gained an FDA fast track designation for SHP655 (formerly BAX930) for treatment of acute episodes of hereditary thrombotic thrombocytopenic purpura in patients with a constitutional deficiency of the von Willebrand factor-cleaving protease ADAMTS13. The company said will initiate a phase III trial with SHP655 as a randomized, open-label, two-period crossover study with a single arm continuation to evaluate its safety and efficacy of SHP655 in the treatment and prevention of acute TTP events in patients with severe hereditary ADAMTS13 deficiency.
Sunesis Pharmaceuticals Inc., of South San Francisco, submitted its responses to the EMA Day 180 List of Outstanding Issues, issued by the Committee for Medicinal Products for Human Use (CHMP) as part of the centralized review process of the marketing authorization application (MAA) for vosaroxin as a treatment for relapsed/refractory acute myeloid leukemia in patients age 60 and older. It anticipates a CHMP decision on the MAA mid-year. To assist with the CHMP decision, it is also preparing to go before the Scientific Advisory Group's Oncology Division in April, President and CEO Daniel Swisher said.
Xoma Corp., of Berkeley, Calif., said that it has fully prepaid the outstanding balance of about $6.5 million under the company's term loan with Hercules Technology Growth Capital Inc. CEO Jim Neal called it "another important step in our strategy to reduce debt and strengthen our balance sheet, as we continue to pursue our goal of becoming a profitable company," adding that the company has decreased its debt by about $16.7 million, or 39 percent, in the past three months. Xoma funded the payment to Hercules in part with proceeds from a near-$25 million equity offering to Biotechnology Value Fund, L.P., completed in February 2017.
XTL Biopharmaceuticals Ltd., of Raanana, Israel, closed a private offering of securities for proceeds of $2.8 million. The company issued and sold 1.4 million American depository shares (ADSs) at a purchase price of $2 per ADS. It is developing its candidate, hCDR1, for the treatment of autoimmune diseases including systemic lupus erythematosus and Sjögren's syndrome.