Product Development

TG Therapeutics' umbralisib and ublituximab in R/R and treatment-naïve lymphomas face pushback on market penetration; FDA approval highly expected, say experts

TG Therapeutics’ (NASDAQ:TGTX) umbralisib monotherapy in relapsed/refractory (R/R) lymphomas is unlikely to have extensive market traction, experts said. The same sentiment holds for its combination with ublituximab (U2) in relapsed/refractory (R/R) and treatment-naïve chronic lymphocytic leukemia (CLL) despite both showing an improved safety profile to competitors, they added. However, FDA approval is still highly expected based on positive registrational trial data, they said.

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Arrowhead’s interim Phase II data in A1AD liver disease lacks correlation with potential fibrosis impact, investigators say

Arrowhead Pharmaceuticals’ (NASDAQ:ARWR) interim Phase II ARO-AAT data falls short of informing of a highly desired impact on alpha-1 antitrypsin deficiency (A1AD) liver fibrosis, investigators said. This is despite results showing reductions in mutant proteins emblematic of the disease, they added.

Interim data released on 13 November led to a 40.3% Arrowhead stock boost. Additional results were released over the weekend at the AASLD Digital Liver Meeting, which led to a 2.5% stock increase. Arrowhead has a USD 7.05bn market cap.

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Aurinia’s voclosporin has possible future nephrotoxicity concerns that could minimize first-line potential for lupus nephritis patients, experts say; FDA approval still likely

Aurinia Pharmaceutical’s (NASDAQ:AUPH) voclosporin has potential long-term nephrotoxicity concerns that could preclude widespread first-line use in lupus nephritis (LN) until extended safety data becomes available, most experts said. However, a Phase III investigator countered this view, noting the mechanism of action (MOA) means there will be no significant nephrotoxicity concerns.

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Pfizer/BioNTech’s late-phase COVID-19 vaccine trial speedier than Moderna, yet utility conclusions dubious from either trial’s interim analysis, experts say

Although Pfizer (NYSE:PFE)/BioNTech’s (NASDAQ:BNTX) late-phase COVID-19 vaccine trial is designed to report interim data earlier and has the potential for a broader label than Moderna’s (NASDAQ:MRNA) vaccine, the former’s results could leave vaccinators unclear about which patient group would be best suited to use the vaccine. Uncertainty about how to best use both vaccines would be an issue if FDA approval comes via an Emergency Use Authorization (EUA), as premature results at such an early stage would lack detail.

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vTv’s azeliragon study for Alzheimer’s disease and type 2 diabetes has posthoc analysis, potential biological support but some skepticism remains, experts say

Despite failing a Phase III trial for Alzheimer’s disease (AD), vTv Therapeutics’ (NASDAQ:VTVT) azeliragon could have an improved chance of success in an ongoing Phase II and planned Phase III for patients with AD and type 2 diabetes, some experts said. A Phase III posthoc analysis based on a potential biological connection between AD and diabetes could prove promising, though there has been a long history of unsuccessful therapeutic attempts to target mild-to-severe AD, experts said.

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BCG vaccines best repositioned for COVID-19 prophylaxis than therapeutic investigation, yet may only offer reduced disease severity, not full protection, investigators say

The Bacillus Calmette-Guérin (BCG) vaccines being investigated for COVID-19 are likely to have a better shot in the prophylactic setting than in patients positive for SARS-CoV-2, investigators said. These vaccines are more inclined to reduce the risk of severe disease rather than offering complete protection due to their nonspecific mechanism, they noted.

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Sarepta’s Phase II SRP-9001 for DMD has most experts touting 3% microdystrophin increase benchmark for FDA accelerated approval

Sarepta Therapeutics’ (NASDAQ:SRPT) SRP-9001 gene therapy for Duchenne Muscular Dystrophy (DMD) has most experts touting a minimum 3% increase in the Phase II microdystrophin primary endpoint necessary to attain clinical significance. If this benchmark is hit, as expected, the company should secure a regulatory nod if it files for FDA accelerated approval.

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Arena’s Phase IIb etrasimod faces uphill climb to match Regeneron’s Dupixent in atopic dermatitis, dermatologists say

Arena Pharmaceuticals’ (NASDAQ:ARNA) etrasimod drew dermatologist skepticism about its potential to report positive results in its Phase IIb trial in moderate-to-severe atopic dermatitis (AD) due to its broad mechanism and current data lacking relevance in AD. Regeneron Pharmaceuticals’ (NASDAQ:REGN) Dupixent (dupilumab) is well established in AD treatment, further raising the bar for clinical relevance, they added.

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Repurposing efforts for Fibrogen's, Blade's, Boehringer's and Roche's IPF drugs in COVID-19 holds water despite lung pathology differences

There is rationale for evaluating antifibrotics in COVID-19 given the SARS-CoV-2 virus’ impact on the lungs, but several unknowns persist on the overlap between idiopathic pulmonary fibrosis (IPF) and COVID-19 related fibrosis, making the therapeutic impact of antifibrotics unclear, experts noted. While Fibrogen’s (NASDAQ:FGEN) idiopathic pulmonary fibrosis (IPF) data is strong enough for pamrevlumab to be considered in Phase II, the potential of Blade Therapeutics’ BLD-2660 is tougher to determine given the existence of only in vitro data for the product, they noted.

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Sinopharm’s, Sinovac’s positive COVID-19 vaccine immunogenicity data undercut by durability, manufacturing challenges ahead, experts say

Despite positive early immunogenicity data for Sinopharm’s and Sinovac’s (NASDAQ:SVA) inactivated virus vaccines, experts noted the path to approval is filled with obstacles in proving durable protection and ironing out manufacturing.

In contrast with live-attenuated vaccines, inactivated vaccine efficacy hinges on multiple shots as well as an adjuvant, making the choice of adjuvant key, some noted. Sinopharm and Sinovac have so far only reported seroconversion rates.

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