Update for July 13, 2022

Regulatory Update

The FDA accepted the BLA for mosunetuzumab to treat relapsed or refractory follicular lymphoma in patients who have received at least two prior systemic therapies. A PDUFA date is set for 12/29/2022.

The FDA accepted the NDA for lecanemab to treat Alzheimer’s disease, and set a PDUFA date for 1/6/2023.

Two months after rejecting toripalimab due to a manufacturing quality process deficiency, the FDA accepted the resubmitted NDA and set a PDUFA date for 12/23/2022. An onsite inspection of the manufacturing plant will be required.

The FDA placed a hold on the Phase II/III ReMEDy2 trial (NCT05065216), due to the incidence of hypotension in patients treated with DM199.

Announced Research Updates

Nordic Nanovector discontinued the Phase IIb Paradigm trial, evaluating lutetium (177lu) lilotomab satetraxetan to treat refractory follicular lymphoma, after an interim analysis revealed low efficacy

Sarepta announced one-year functional results from 20 patients enrolled in cohort 1 of the open-label, Phase I, ENDEAVOR trial, where treatment with delandistrogene moxeparvovec improved the NSAA score compared to a historical control group of patients with DMD. There was one case of myocarditis identified in the Phase I ENDEAVOR trial, which appeared to resolve four months after treatment with IV methyl-prednisolone. Sarepta also announced four-year results from a four patient, Phase I/II trial, where delandistrogene moxeparvovec maintained a 7 point improvement in the North Star Ambulatory Assessment (NSAA) over baseline and a 9.4 point improvement compared to a historical control group in patients with Duchenne muscular dystrophy. 

Lysogene announced 24 month data from 16 patients enrolled in the 20 patient, Phase II/III, AAVance trial, where treatment with LYS-SAF302 resulted in a 22% decrease in heparan sulfate (HS) in the cerebrospinal fluid (CSF), a 20% decrease in HS-derived oligosaccharides in the CSF, 41% decrease in serum neurofilament light (NFL) levels and a 33% decrease in CSF NFL levels in patients with mucopolysaccharidosis Type IIIA (MPS IIIA).

MacroGenics discontinued a Phase 2 trial evaluating enoblituzumab in combination with retifanlimab or tebotelimab, in the treatment of recurrent or metastatic squamous cell carcinoma of the head and neck, after seven fatalities potentially associated with hemorrhagic events were reported.

Novo Nordisk announced that in the 32-week, 133 patient, Phase III, explorer7 trial, prophylaxis with concizumab resulted in an annual bleeding rate of 1.7 compared to 11.8 with no prophylaxis in patients with hemophilia A or B with inhibitors.

Sanofi announced that in the 13-month, 80 patient, Phase III, ATLAS-PPX trial, prophylaxis with fitusiran resulted in an annual bleeding rate of 0 compared to 61.1% with prior factor or bypassing agent prophylaxis in patients with hemophilia A or B with or without inhibitors.

Aldeyra announced that in a two-day, 50 patient, Phase II crossover trial, treatment with reproxalap decreased eye redness and increased tear production as measure with a Schirmer test in patients with dry eye disease.

Published Research Updates

In the 104-week, 206 patient, open-label, Phase II, BE ACTIVE 2 trial, a long-term extension of the BE ACTIVE trial, continued treatment with bimekizumab resulted in sustained improvement in pain, fatigue and function.

16-week results from the 52-week, 503 patient, Phase III, OASIS-1 trial, found that 69.3%of patients treated with mirikizumab achieved at least a 2-point improvement in their sPGA score and 64.3% achieved PASI90 compared to 6.5% and 6.5%with placebo in patients with moderate to severe plaque psoriasis. At 52-weeks, PASI90/PASI100/sPGA response was achieved with mirikizumab 250Q4W/placeboQ8W in 18.7%/9.9%/17.6%, mirikizumab 250Q4W/125Q8W in 85.6%/58.9%/85.6%, and mirikizumab 250Q4W/250Q8W in 84.6%/60.4%/82.4%. 

In a 90 patient cohort of an open-label, Phase I/Ib trial, treatment with mosunetuzumab resulted in a complete response (CR) rate of 60% compared to 14% in a historical control group that received copanlisib in patients with relapsed or refractory follicular lymphoma.

In the 94-patient, Phase II, open-label, POD1UM-202 trial, treatment with retifanlimab resulted in an objective response rate of 13.8% in patients with locally advanced or metastatic squamous cell carcinoma of the anal canal, who progressed on, or are intolerant of, platinum-based chemotherapy.

In a 33 patient, Phase II trial, treatment with eprenetapopt plus azacitidine resulted in relapse free survival of 12.5 months in post-transplant patients with TP53 mutant MDS and AML.