Axial — New Medicines #29. Get these analyses to your inbox —…
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New medicines #29 — November 21, 2020 — November 27, 2020

A weekly overview of the development of new medicines.

- ADC Therapeutics announced that the FDA has accepted its Biologics License Application (BLA) for loncastuximab tesirine (Lonca) for the treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and granted a PDUFA date under priority review of May 21, 2021.

- Alnylam Pharmaceuticals announced the FDA approved Oxlumo (lumasiran) injection for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

- Arcutis Biotherapeutics announced positive top line data from its phase 2b clinical trial evaluating ARQ-154 (topical roflumilast foam) as a potential treatment for scalp psoriasis:

The trial met the primary endpoint of Scalp Investigator Global Assessment (S-IGA) success assessed at week 8, achieving a rate of 59.1% compared to a vehicle rate of 11.4% (p<0.0001)

S-IGA success is defined as the achievement of an S-IGA score of clear or almost clear on a 5-grade scale plus at least a two-point change from baseline

- AstraZeneca announced interim data from its phase 3 trial of its COVID-19 vaccine, AZD1222, in patients in the UK and Brazil:

The trial met its primary endpoint, showing to be highly effective in preventing COVID-19 and no hospitalizations or severe cases of the disease were reported in participants receiving the vaccine

The data showed an average efficacy of 70% across two dosing regimens. The company hopes to produce 3 billion doses in 2021

One dosing regimen (n=2,741) showed vaccine efficacy of 90% when AZD1222 was given as a half dose, followed by a full dose at least one month apart, and another dosing regimen (n=8,895) showed 62% efficacy when given as two full doses at least one month apart

- Bellerophon Therapeutics announced that its phase 3 trial of INOpulse for the treatment of COVID-19 has been placed on clinical hold following interim analysis from the independent Data Monitoring Committee (DMC).

- BioLineRx released interim analysis from its phase 2b BLAST study in consolidation Acute myelogenous leukemia (AML) did not meet the primary endpoint.

- Eiger BioPharmaceuticals received approval from the FDA for Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older..

- Liquidia Technologies announced that the FDA issued a Complete Response Letter (CRL) for its New Drug Application (NDA) for LIQ861 (treprostinil) inhalation powder for the treatment of pulmonary arterial hypertension (PAH):

The CRL identified the need for additional information and clarification on chemistry, manufacturing and controls (CMC) data pertaining to the drug product and device biocompatibility

The FDA also reconfirmed the need to conduct on-site pre-approval inspections (PAIs) of two of Liquidia’s U.S. manufacturing facilities before the application can be approved

The FDA noted it had been unable to conduct these inspections during the initial review cycle due to COVID-19 related travel restrictions

- Reata Pharmaceuticals announced after hours the FDA has completed its review of the Baseline-Controlled Study results of omaveloxolone for the treatment of patients with Friedreich’s ataxia (FA) and concluded that the results do not strengthen the results of Part 2 of the MOXIe study:

The company had previously guided for a NDA filing mid-2021 assuming the FDA viewed the crossover study data as sufficiently positive to provide confirmatory evidence for filing

The company will now consider the next steps for the development program, including whether to conduct a second pivotal study in patients with FA

- Y-mAbs Therapeutics announced the FDA approved its Biologics License Application (BLA) for Danyelza (naxitamab) for the treatment of patients with relapsed/refractory high-risk neuroblastoma.

Source: BPC (reformatted), SY, Bioworld