Acceleron ACE-1334 nabs U.S. Orphan drug tag for connective tissue disorder

 

• The FDA has designated Orphan Drug status to Acceleron Pharma's (NASDAQ:XLRN) ACE-1334 for the treatment of systemic sclerosis, an autoimmune rheumatic disease characterized by excessive production and accumulation of collagen, called fibrosis, in the skin and internal organs.
• The Company intends to initiate a Phase 1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD) in 2021, an autoimmune connective tissue disorder characterized by immune dysregulation.
• The Orphan Drug tag may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the U.S. following marketing approval.
• ACE-1334 is a TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2, which are believed to be key signaling factors in the pathogenesis of fibrotic disease.
• https://seekingalpha.com/news/3640270-accelerons-aceminus-1334-nabs-u-s-orphan-drug-tag-for-connective-tissue-disorder

Regenxbio starts study of gene therapy for rare metabolic disorder

 

• The first participant has been dosed in a 5-subject Phase 1/2 clinical trial evaluating REGENXBIO's (NASDAQ:RGNX) RGX-111 for the treatment of mucopolysaccharidosis type 1 (MPS I), a rare inherited disorder in which the body cannot metabolized large sugar molecules called glycosaminoglycans due to a deficiency in an enzyme called IDUA which leads to organ and tissue damage.
• The estimated primary completion date is November 2021.
• Orphan Drug-, Rare Pediatric Disease- and Fast Track-tagged RGX-111 is a one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS).
• https://seekingalpha.com/news/3640278-regenxbio-commences-study-of-gene-therapy-for-rare-metabolic-disorder

FDA accepts BridgeBio/QED's infigratinib application for bile duct cancer

 

• The FDA accepts for review BridgeBio Pharma (NASDAQ:BBIO) and affiliate QED Therapeutics' New Drug Application (NDA) for infigratinib for the first-line treatment of advanced/metastatic cholangiocarcinoma (bile duct cancer) with FGFR2 gene fusions or translocations.
• Infigratinib a lead drug candidate is an orally administered, FGFR1-3 selective tyrosine kinase inhibitor.
• The NDA has been granted priority review designation and is being reviewed under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA’s Oncology Center of Excellence designed to expedite the delivery of safe and effective cancer treatments to patients.
• Additionally, BridgeBio will submit for review in Australia and Canada under Project Orbis, an initiative of the FDA’s Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies.
• BBIO's NDA for fosdenopterin in molybdenum cofactor deficiency (MoCD) Type A was accepted in September 2020.
• https://seekingalpha.com/news/3640300-fda-accepts-bridgebio-pharma-qeds-infigratinib-application-for-bile-duct-cancer

Amicus rolling U.S. AT-GAA marketing application for Pompe disease

 

• Amicus Therapeutics (NASDAQ:FOLD) has initiated rolling marketing application to the FDA for AT-GAA for the treatment of late onset Pompe disease (LOPD), an inherited metabolic disorder caused by the buildup of glycogen in the body's cells due to mutations in the gene that codes for an enzyme called acid alpha-glucosidase, leading to organ dysfunction.
• The rolling submission allows submission of portions of the regulatory application as they are completed, rather than waiting until every section of the application is complete.
• Earlier this year, the FDA authorized Amicus to proceed with a rolling BLA submission.
• AT-GAA consists of cipaglucosidase alfa (ATB200), an enzyme replacement therapy with optimized carbohydrate structures, administered in conjunction with miglustat (AT2221), an orally administered stabilizer of cipaglucosidase alfa.
• Amicus has submitted the nonclinical component of the cipaglucosidase alfa application and is on-track to submit the chemistry, manufacturing and controls component, and the final clinical module in 1H of 2021.
• The company will separately submit miglustat application at the same time as the remainder of the cipaglucosidase alfa BLA.
• AT-GAA received FDA Breakthrough status for LOPD. Top-line data from ongoing Phase 3 PROPEL study of AT-GAA in LOPD is on-track for Q1 of 2021.
• Recently, under the priority review status, the FDA accepted  Sanofi's application for long-term enzyme replacement therapy for Pompe disease. Agency's action date is May 18, 2021.
• https://seekingalpha.com/news/3640312-amicus-launches-rolling-u-s-gaa-marketing-application-for-pompe-disease

Agios' mitapivat succeeds in late-stage study in rare blood disorder

 

• A Phase 3 clinical trial, ACTIVATE, evaluating Agios Pharmaceuticals' (NASDAQ:AGIO) mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions met the primary endpoint.
• Specifically, 40% of treated patients achieved a hemoglobin response at weeks 16, 20 and 24 compared to 0% in the control group (p<0.0001).
• Key secondary endpoints were also met.
• No new safety signals were observed.
• Complete results will be submitted for presentation at the European Hematology Association Virtual Congress in June 2021.
• The company plans to file marketing applications in the U.S. and Europe in 2021.
• Mitapivat is an orally available activator of wild-type and mutated pyruvate kinase-R enzymes.
• https://seekingalpha.com/news/3640320-agios-mitapivat-successful-in-late-stage-study-in-rare-blood-disorder

TG Therapeutics rolling U.S. application for leukemia doublet therapy

 

• TG Therapeutics (NASDAQ:TGTX) initiates its rolling U.S. marketing application seeking approval of ublituximab, combined with umbralisib, for the treatment of chronic lymphocytic leukemia (CLL), a Fast Track- and Orphan Drug-tagged indication. The company expects to complete the filing in H1 2021.
• Ublituximab is an anti-CD20 monoclonal antibody. Umbralisib is a dual inhibitor of enzymes PI3K-delta and CK1-epsilon. The company calls the doublet therapy "U2."
• Its U.S. marketing applications for umbralisib for marginal zone lymphoma and follicular lymphoma are currently under FDA review with action dates of February 14, 2021 and June 15, 2021, respectively.
• https://seekingalpha.com/news/3640326-tg-therapeutics-commences-rolling-u-s-application-for-doublet-therapy-for-cll

Dr. Reddy's launches new study of Russian COVID-19 vaccine in India

 

• Dr. Reddy's Laboratories (NYSE:RDY) announces the start of an adaptive Phase 2/3 clinical trial in India evaluating Russia's Sputnik V COVID-19 vaccine. The study will be conducted by JSS Medical Research.
• An adaptive study allows for modifications to the trial and/or statistical models while the study is in process.
• The company is partnering with the Biotechnology Industry Research Assistance Council (BIRAC), Department of Biotechnology (DBT) for advisory support and to use BIRAC’s clinical trial centers for the vaccine.
• The Russian Direct Investment Fund (RDIF) recently announced the results from the second interim analysis of a large-scale Phase 3 study that showed 91.4% efficacy at day 28 after the first dose and over 95% 42 days after the first dose. Over 40K volunteers are participating.
• In September, RDY and RDIF entered into a partnership to conduct clinical trials of the vaccine and the rights for distribution of the first 100 million doses in India.
• https://seekingalpha.com/news/3640378-dr-reddys-launches-new-study-of-russian-covidminus-19-vaccine-in-india