Roche boosts its gene ther­a­py game, ex­pand­ing part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics

Join­ing the Big Phar­ma rush to pick up po­ten­tial­ly valu­able gene ther­a­py tech, Roche has ex­pand­ed its part­ner­ship with 4D Mol­e­c­u­lar Ther­a­peu­tics to work on a drug for night blind­ness, among oth­er oph­thal­mol­o­gy pro­grams.

David Kirn

Roche had first part­nered with the Emeryville, Cal­i­for­nia-based biotech back in 2016, when the duo was re­search­ing a small hand­ful of rare reti­nal dis­ease in­di­ca­tions. Now, it’s ex­pand­ed that com­mit­ment to take in oph­thal­mol­o­gy pro­grams that in­clude prospects for much larg­er mar­kets.

The progress of Spark and its gene ther­a­py hit Lux­tur­na — along with new part­ner­ships forged by its phar­ma brethren Pfiz­er and No­var­tis — like­ly laid the ground­work for Roche to re­con­sid­er its part­ner­ship with 4DMT.

Roche and 4DMT’s first col­lab­o­ra­tion pro­gram is for 4D-110, a clin­i­cal can­di­date to treat choroi­deremia (an in­her­it­ed form of night blind­ness that can show up in ear­ly child­hood). 4DMT is just now do­ing IND-en­abling stud­ies on the pro­gram. 4D-110 al­ready has some ad­vanced com­pe­ti­tion in the field, as Lon­don’s Night­star $NITE is al­so tack­ling this dis­ease with a Phase III gene ther­a­py drug.

4DMT’s edge would be its ade­no-as­so­ci­at­ed virus plat­form, which the com­pa­ny says is su­pe­ri­or to its ri­vals.

The part­ner­ship al­so in­cludes ad­di­tion­al clin­i­cal can­di­date pro­grams that are un­der­way to treat reti­nal dis­eases with high un­met need, 4DMT says. The com­pa­ny’s CEO David Kirn said this in a state­ment:

To­geth­er we elect­ed to ex­pand our part­ner­ship af­ter 4DMT com­plet­ed pro­pri­etary in­trav­it­re­al vec­tor dis­cov­ery and char­ac­ter­i­za­tion. We have cre­at­ed clear syn­er­gies be­tween 4D’s vec­tor dis­cov­ery, gene ther­a­py de­vel­op­ment and man­u­fac­tur­ing ca­pa­bil­i­ties and Roche’s ex­per­tise in late-stage clin­i­cal de­vel­op­ment and glob­al bi­o­log­ics com­mer­cial­iza­tion. The de­ci­sion to ex­pand our part­ner­ship rep­re­sents val­i­da­tion of our oph­thal­mol­o­gy plat­form, clin­i­cal can­di­dates and team.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Amit Munshi, Arena

One of Are­na's top drugs flops in a PhI­Ib study for IBS pain. But re­searchers tease out a pos­si­ble path for­ward as CEO ex­plores 's­trate­gic op­tion­s'

Four years ago, when Arena CEO Amit Munshi cut its ties to a troubled weight drug and doubled down on the pipeline, a cannabinoid receptor 2 agonist figured prominently in the biotech’s future. On Tuesday evening, however, Munshi’s high hopes for the drug took a nasty hit after it failed a Phase IIb study for patients with irritable bowel syndrome pain.

Put through a randomized pace with 273 patients, researchers said it flat failed the primary endpoint among the large group with abdominal pain. But they quickly went on to highlight subgroup data, always a tricky and controversial ploy, where they spotlighted a positive p value for patients with moderate to severe pain who received the high dose of the drug — one of 3 provided in the study.

Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Marco Taglietti (Scynexis)

'N­ev­er been more ur­gent:' Scynex­is looks to tack­le su­per­bug cri­sis with late-stage read­out for an­ti­fun­gal hope­ful

As the superbug crisis heats up around the world, Scynexis says it has new data from two interim analyses that prove its antifungal has the potential to treat a broad range of infections.

“The need for new anti-infectives capable of fighting the most resistant pathogens has never been more urgent as we confront the ongoing COVID-19 global pandemic,” CEO Marco Taglietti said in a statement.

A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Takeda spun out a pipeline of experimental psychiatry drugs to Neurocrine in a $2 billion deal amid a post-merger shakeout, R&D chief Andy Plump described the therapies as “very interesting but still difficult.”

On Tuesday, we got some idea of how difficult.

San Diego-based Neurocrine revealed that one of the three spotlight clinical programs they’d acquired failed the primary endpoint in a Phase II trial for schizophrenia, registering a negative outcome on the change from baseline in the positive and negative syndrome scale/negative symptom factor score (PANSS NSFS).

Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,400+ biopharma pros reading Endpoints daily — and it's free.