Applied Genetic Technologies Corp., of Gainesville, Fla., and Cambridge, Mass., published preclinical data providing the foundation for the vector and starting dose used in its ongoing phase I/II trial in patients with X-linked retinitis pigmentosa (XLRP) due to mutations in the RPGR gene. The data, showing the company’s AAV vector and engineered RPGR constructs were well-tolerated and had beneficial effects on markers of disease in a canine model of XLRP, were published online in Human Gene Therapy.
Airway Therapeutics LLC, of Cincinnati, and Basel, Switzerland’s Celonic Group will collaborate to produce the human recombinant protein AT-100 (rhSP-D) as a COVID-19 therapy. Celonic will be responsible for process optimization and GMP manufacturing of AT-100 for clinical study. Production of AT-100 is expected to begin in June. AT-100 is an engineered version of an endogenous protein that has been shown in preclinical studies to reduce inflammation and infection in a range of bacterial and viral respiratory diseases by modulating the body's immune response, Airway said. Airway has previously filed a pre-IND submission with the FDA for AT-100 as a preventive treatment of the serious respiratory disease bronchopulmonary dysplasia in very preterm infants, with an IND filing expected in the third quarter of 2020 and plans to enter the clinic in late 2020.
Assertio Therapeutics Inc., of Lake Forest, Ill., completed its merger with Wayne, Pa.-based Zyla Life Sciences Inc. and will operate under the Assertio name. The company now has a differentiated portfolio of nonsteroidal anti-inflammatory drugs commonly used by neurologists, orthopedic surgeons, internists, women's health providers, podiatrists, pain care specialists and urologists. All eight products are branded and FDA approved.
Cansino Biologics Inc., of Tianjin, China, and Precision Nanosystems Inc., of Vancouver, British Columbia, agreed to co-develop an mRNA lipid nanoparticle vaccine against COVID-19. Precision will develop the mRNA-LNP vaccine, and Cansino is responsible for preclinical testing, clinical trials, regulatory approval and commercialization. Cansino has rights to commercialize the vaccine in Asia (except Japan), with Precision retaining rights for the rest of the world. The agreement includes undisclosed payments and royalties.
Coda Biotherapeutics Inc., of South San Francisco, formed a research collaboration with the Facial Pain Research Foundation for using Coda's chemogenetic gene therapy platform to identify and develop therapies and cures for trigeminal neuralgia and related neuropathic pain. The foundation provided an undisclosed grant to an academic-based research institution supporting preclinical research of Coda's chemogenetically controlled inhibitory ion channels in neuropathic pain. Coda will work with the foundation to establish a research continuum to identify mechanisms underlying neuropathic facial pain.
Faron Pharmaceuticals Oy, of Turku, Finland, said results of its detailed analyses into the effects of glucocorticoids on intravenous (I.V.) interferon beta-1a activity, which arose following the trial called Interest in 2018, have been published in Intensive Care Medicine. The results showed that the desired mechanism of action of I.V. interferon beta-1a in the lung vasculature – the up-regulation of CD73 – is blocked by the administration of glucocorticoids. In addition, the administration of glucocorticoids with the drug increases mortality in patients with acute respiratory distress syndrome compared to patients administered the drug alone. Analysis from the Interest trial showed day 28 mortality for patients receiving concomitant glucocorticoids with I.V. interferon beta-1a was 39.7% compared to 10.6% for patients receiving the drug alone (p < 0.001).
Hoth Therapeutics Inc., of New York, licensed technology and intellectual property exclusively from Virginia Commonwealth University for a novel peptide therapeutic to prevent spike protein binding, which may be a leading cause of COVID-19, and slow its transmission. The research is being led by inventor Michael Peters, and is being aided, in part, by supercomputers as part of the COVID-19 High Performance Computing Consortium through a virtual system that scientists can use to interactively share computing resources known as the Extreme Science and Engineering Discovery Environment, Hoth said.
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa., disclosed the publication of the preclinical study data for IN0-4800, its COVID-19 DNA vaccine, demonstrating robust neutralizing antibody and T-cell immune responses against coronavirus SARS-CoV-2. The study was published in Nature Communications. The data build on previously reported phase I/IIa data on the firm’s vaccine against the coronavirus that causes MERS, which demonstrated near-100% seroconversion and neutralization from similarly designed vaccine INO-4700.
Iqvia Inc., of Danbury, Conn., said the Walter and Eliza Hall Institute of Medical Research, a leading biomedical research organization in Australia, has selected the firm to manage COVID Shield, a phase II/III randomized, double-blinded study of preventive care for front-line health care workers fighting the COVID-19 pandemic. The study will test an expanded indication for generic hydroxychloroquine as a prophylactic treatment to prevent high-risk health care workers from contracting COVID-19. Local hospitals across Australia will administer the drug or a placebo to about 2,250 health care professionals who are currently looking after COVID-19 patients.
Nanoviricides Inc., of Shelton, Conn., said strong effectiveness against infection by an ACE2-utilizing coronavirus in an animal model has been observed for the drug candidates it is developing against SARS-CoV-2 to treat the COVID-19 spectrum of diseases. The firm is developing an animal model for coronavirus infection using hCoV-NL63 as a surrogate for SARS-CoV-2. HCoV-NL63 is a circulating human coronavirus that causes a disease that is similar to SARS-CoV-2, but much milder. Both viruses utilize the same cell receptor, namely ACE2, to enter the cell.
Surface Oncology Inc., of Cambridge, Mass., signed a clinical trial collaboration with Merck & Co. Inc., of Kenilworth, N.J., to evaluate the safety and efficacy of combining Surface’s SRF-617, an antibody therapy targeting CD39, with Merck’s Keytruda (pembrolizumab), an anti-PD-1 therapy. The combination will be studied as a component of the first-in-human phase I/Ib study of SRF-617 and will be evaluated in patients with solid tumors, with a focus on patients with gastric cancer and those who have developed resistance to checkpoint inhibition. Shares of Surface (NASDAQ:SURF) closed May 20 at $3.88, up $1.22 cents or 45.9%.
Vaxart Inc., of South San Francisco, has selected its lead COVID-19 vaccine candidate. The company has started production of the oral vaccine to be used in a phase I study, scheduled to start in the second half of 2020. Vaxart selected San Francisco-based Kindred Biosciences Inc. as a second contract manufacturer of the vaccine. Terms of the deal weren't disclosed.