FDA grants Orphan Drug Designation for BCX9930 in PNH

The review of unblinded data from part 1 of an ongoing phase 1 trial (NCT03891420) to assess the safety (primary endpoint), clinical impact and antiviral effects of galidesivir in patients with *redacted*, and a decision to choose a dose and advance into part 2 of the trial, are expected to occur in 4Q-2020.

Galidesivir is an investigational broad-spectrum antiviral drug that was safe and well tolerated in previous phase 1 trials in healthy subjects, and has demonstrated broad-spectrum activity in vitro against more than 20 RNA viruses in nine different families, including the viruses that cause MERS and SARS.

Press release about ODD

Press release about NIAID funds

CorMedix NDA for Defencath accepted for priority review by FDA

The Defencath NDA submission was based on positive results from the 795 patient LOCK-IT-100 clinical trial, which demonstrated a good safety profile and a highly statistically significant reduction of 71% (p=0.0006) in CRBSI in patients undergoing hemodialysis compared to heparin, the current standard of care. 

Defencath is a novel, antibacterial and antifungal solution designed to prevent costly and life-threatening bloodstream infections associated with the use of central venous catheters in patients undergoing chronic hemodialysis. Defencath had been granted Fast Track and Qualified Infectious Disease Product (QIDP) designations by the FDA, which provided eligibility to request priority review of the NDA. It also provides an additional five years of marketing exclusivity, which will be added to the five years granted to a New Chemical Entity upon approval of the NDA. CorMedix also intends to develop Defencath as a catheter lock solution for use in oncology and total parenteral nutrition patients.

Compugen gets composition of matter patent for TIGIT inhibitor COM902

U.S. Patent No. 10,751,415,titled “Anti-TIGIT Antibodies, Anti-PVRIG Antibodies and Combinations Thereof,” relates to the composition of matter of COM902, alone or in combination with a second antibody targeting an immune checkpoint, including PD-1 and PVRIG (specifically COM701). This patent is expected to expire no earlier than August 2037 in the United States. 

This patent expands intellectual property protection for COM902 in the United States,for which a patent was previously issued in November 2018, relating to the method of use of COM902 for activating T cells in cancer patients, in addition to claims covering the combination of COM902 and COM701 for activating T cells in cancer patients. Similar to this new U.S. patent,  in November 2019, Compugen was also granted a European patent relating to the composition of matter of COM902, alone or in combination with a second antibody targeting an immune checkpoint, including PD-1 and PVRIG (specifically COM701), as well as for use in treating cancer by activating T cells.

Liquidia gets patent allowance covering methods of treatment of PAH

The patent, which is expected to be issued in the fourth quarter of 2020, should have a term that expires no earlier than 2037. After issuance, Liquidia plans to list the U.S. patent in the FDA’s Approved Drug Products with Therapeutic Equivalence Evaluations, commonly referred to as the “Orange Book”, for LIQ861, if approved.

Liquidia uses its proprietary PRINT technology that enables precise production of uniform drug particles designed to improve the safety, efficacy and performance of a wide range of therapies. Currently, Liquidia is focused on the development of two product candidates for which it holds worldwide commercial rights: LIQ861 for the treatment of pulmonary arterial hypertension (PAH) and LIQ865 for the treatment of local post-operative pain. 

Rocket’s fifth gene therapy program receives Fast Track Designation

RP-L401 was in-licensed from Lund University, under the research leadership of Dr. Johan Richter, M.D., Ph.D. and Dr. Ilana Moscatelli, Ph.D. The vector was in-licensed through a collaboration with Dr. Axel Schambach, M.D., Ph.D. of the Medizinische Hochschule Hannover. 

Rocket’s non-randomized, open-label Phase 1 clinical trial of RP-L401 for the treatment of IMO will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status and endocrine abnormalities.

UCLA will serve as the lead trial site under principal investigator Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the UCLA. 

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.