FDA ad­comm votes 11-2 to ex­pand Roche's Po­livy la­bel in DL­B­CL

An FDA com­mit­tee of out­side can­cer ex­perts vot­ed 11-2 Thurs­day that the FDA should ex­pand Roche’s Po­livy la­bel to in­clude it as a treat­ment in com­bo with rit­ux­imab, cy­clophos­phamide, dox­oru­bicin, and pred­nisone (R-CHP) for the treat­ment of adult pa­tients with pre­vi­ous­ly un­treat­ed dif­fuse large B-cell lym­phoma (DL­B­CL).

The pos­i­tive On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee (ODAC) vote for the an­ti­body-drug con­ju­gate fol­lowed a four hour-plus meet­ing where the ad­comm hashed out con­cerns high­light­ed in brief­ing doc­u­ments re­leased by FDA. Those con­cerns in­clud­ed is­sues like a mod­est ben­e­fit in pro­gres­sion-free sur­vival, over­all sur­vival rates, and lim­i­ta­tions in ef­fi­ca­cy end­points.

A rep­re­sen­ta­tive for the FDA high­light­ed the over­all sur­vival haz­ard ra­tio in the DL­B­CL NOS sub­group, which was 1.02, with an up­per bound of 1.49.

“The ap­pli­cant seeks a front­line in­di­ca­tion on the ba­sis of a sin­gle large ran­dom­ized tri­al. How­ev­er, these find­ings cre­ate un­cer­tain­ty about the ben­e­fit risk pro­file of Po­livy plus R-CHOP in pa­tients with pre­vi­ous­ly un­treat­ed large B cell lym­phoma, a set­ting where again, treat­ment is de­liv­ered with cu­ra­tive in­tent,” FDA Of­fice of On­co­log­ic Dis­eases clin­i­cal team leader Yvette Kasa­mon said be­fore the com­mit­tee.

The drug was first ap­proved back in 2019 un­der the ac­cel­er­at­ed ap­proval path­way for pa­tients with re­lapsed or re­frac­to­ry DL­B­CL, not oth­er­wise spec­i­fied (NOS), af­ter at least two pri­or ther­a­pies.

Com­mit­tee mem­ber Mikkael Sek­eres from the Uni­ver­si­ty of Flori­da vot­ed against ap­proval and said, “I’m not sure I trust who pro­gressed, and who didn’t, and what their base dis­ease was,” adding that from his view, the tri­al did not meet “the ba­sics of a large clin­i­cal tri­al in hema­to­log­ic ma­lig­nan­cies.”

Mark Conaway of the Uni­ver­si­ty of Vir­ginia was the oth­er no vote, say­ing, “Even though I agree that Po­livy did show ben­e­fits in this tri­al, for me at present here was too much un­cer­tain­ty about the mag­ni­tude and the ro­bust­ness of the treat­ment ef­fects.”

An­tho­ny Sung out of Duke Uni­ver­si­ty School of Med­i­cine vot­ed yes.

“This is a ran­dom­ized clin­i­cal tri­al that met its pri­ma­ry end­point of im­prove­ment in pro­gres­sion free sur­vival,” Sung said, say­ing that he thought PFS is a “clin­i­cal­ly sig­nif­i­cant end­point, and I feel that the dif­fer­ence, even though it is small, is sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly sig­nif­i­cant as well.”

Com­mit­tee chair Jorge Gar­cia, al­so chief of the sol­id tu­mor on­col­o­gy di­vi­sion at Case West­ern Re­serve Uni­ver­si­ty, vot­ed yes too.

“I think that the tri­al met pri­ma­ry end­point… And al­though I con­tin­ue to wres­tle with that lack of dif­fer­ence in com­plete re­spons­es and the ex­ist­ing sur­vival da­ta, it ap­pears to be im­prac­ti­cal for us to wait for that fi­nal OS — it may nev­er ar­rive based up­on the in­abil­i­ty to get there,” Gar­cia added.

The FDA is ex­pect­ed to make a de­ci­sion on ap­proval some­time in ear­ly April.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.