Biotech Companies Announce Big Drug Wins with FDA

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

Several biotech and biopharmaceutical companies have announced new wins with the U.S. Food and Drug Administration (FDA), with these wins ranging from Investigational New Drug (IND) application approvals for cancer treatment candidates to Fast Track Designations for investigational agents aimed toward opioid use disorder (OUD).

Kronos Bio announced Tuesday the FDA cleared its IND for next-generation spleen tyrosine kinase (SYK) inhibitor lanraplenib (LANRA) for relapsed/refractory and newly diagnosed acute myeloid leukemia (AML). The drug is specifically targeting FLT3-mutated and/or NPM1-mutated AML.

“This LANRA IND caps off an outstanding year for our SYK portfolio, which we acquired just over a year ago,” said Kronos Bio’s chief medical officer and executive vice president of clinical development, Jorge DiMartino, M.D., Ph.D., in a statement

“Since that time, we have nearly completed the integration of the ENTO and LANRA programs with our systems, built out the requisite clinical, translational, regulatory and manufacturing infrastructure for LANRA and ENTO and had successful interactions with the FDA, both for the ENTO Phase 3 clinical trial, as well as for LANRA in relapsed/refractory FLT3-mutated AML patients.” 

Clearance of the IND allows Kronos Bio to proceed with a Phase I/II trial of LANRA with gilteritinib, an anti-cancer agent which inhibits FLT3, in patients with relapsed/refractory FLT3-mutated AML. The study is expected to launch in the fourth quarter of 2021. Currently, this is the first IND for the company’s therapy in a cancer indication. The agent previously demonstrated acceptable safety in trials of over 250 healthy people and patients with autoimmune conditions.

Dr. DiMartino explained that ENTO (entospletinib), Kronos Bio’s other SYK inhibitor, is also entering a registrational Phase III clinical trial. Findings from this trial may lend support for accelerated approval of the agent, in combination with chemotherapy, for the indication of newly diagnosed NPM1-mutated AML. 

Ultragenyx has also scored an FDA win after the agency recently granted Orphan Drug Designation for the company’s investigational messenger RNA (mRNA) therapy UX053 for the treatment of glycogen storage disease. The European Commission concurrently granted Orphan Drug Designation to the therapy for the same indication. 

Ultragenyx is currently enrolling patients with glycogen storage disease type III in a Phase I/II study to test the safety, tolerability and efficacy of UX053. The company is also developing UX053, in addition to other mRNA therapies, for indications currently undisclosed to the public. The development of the therapy is under a collaboration agreement with clinical-stage mRNA drug company Arcturus Therapeutics.

The FDA has also recently granted Fast Track Designation to Lyndra Therapeutics’ investigational OUD therapy LYN-014. The oral, weekly therapy is an ultra-long-acting, extended-release capsule of levomethadone, a therapy not available in the U.S.

In a statement, Lyndra’s CMO, Dr. Richard Scranton, said there is currently “a great need for new and improved, patient friendly treatments for OUD, as deaths from opioid misuse, already high, have surged during the COVID pandemic.” He added that the weekly dosage of LYN-014, if approved, could “free people from daily trips to methadone clinics and support consistency of treatment, recovery and well-being.”


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