Roche, PTC will now get an FDA de­ci­sion on their SMA drug in 6 months — fac­ing off with Bio­gen, No­var­tis

A ma­jor com­peti­tor to Bio­gen’s Spin­raza fran­chise could ar­rive as ear­ly as this com­ing May, as Roche and its biotech part­ner ob­tained a pri­or­i­ty re­view for ris­diplam.

FDA reg­u­la­tors will make a de­ci­sion on the spinal mus­cu­lar at­ro­phy drug by May 24, 2020, ac­cord­ing to PTC Ther­a­peu­tics, who is bag­ging $15 mil­lion in mile­stone pay­ment for the NDA fil­ing ac­cep­tance.

The move comes on the heels of topline da­ta from the Phase III SUN­FISH tri­al, in which ris­diplam met the pri­ma­ry end­point on change from base­line in the Mo­tor Func­tion Mea­sure 32 (MFM-32) scale af­ter one year of treat­ment, com­pared to place­bo. The re­sults con­firmed ear­li­er find­ings from part one of the study.

Levi Gar­raway

SUN­FISH en­rolled 180 pa­tients with type 2 or 3 SMA be­tween 2 and 25 years old. An ear­li­er, open la­bel tri­al dubbed FIRE­FISH cov­ered those with the type 1 vari­a­tion of the neu­ro­mus­cu­lar dis­ease.

“The FIRE­FISH and SUN­FISH tri­als were de­signed to rep­re­sent the re­al world spec­trum of peo­ple liv­ing with SMA and in­clude many peo­ple pre­vi­ous­ly un­der­rep­re­sent­ed in clin­i­cal tri­als,” not­ed Levi Gar­raway, Roche’s new­ly mint­ed head of glob­al prod­uct de­vel­op­ment, in a state­ment.

In PTC’s re­lease, CEO Stu­art Peltz added that the NDA sub­mis­sion in­cludes all three types of SMA pa­tients “demon­strat­ing im­prove­ments in mo­tor func­tions and de­vel­op­men­tal mile­stones, and a com­pelling safe­ty pro­file.”

Un­like Spin­raza, an an­ti­sense oligonu­cleotide ad­min­is­tered in­trathe­cal­ly every four months fol­low­ing ini­tial load­ing dos­es, ris­diplam is an oral med­ica­tion to be tak­en dai­ly. By mod­i­fy­ing how the SMN2 gene is spliced, the drug sup­pos­ed­ly in­creas­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

Stu­art Peltz

The drug is al­ready dec­o­rat­ed with the FDA’s or­phan and fast track des­ig­na­tions.

The fil­ing could lead to a broad­er la­bel than ex­pect­ed, ac­cord­ing to a note from Cred­it Su­isse an­a­lyst Evan Seiger­man, who’s pre­vi­ous­ly warned that ris­diplam spells an even big­ger threat to Bio­gen’s Spin­raza fran­chise than No­var­tis’ gene ther­a­py. Since gain­ing ap­proval in May, Zol­gens­ma has been plagued by re­im­burse­ment is­sues as pay­ers push back against its $2.1 mil­lion price tag (al­though No­var­tis stress­es that the pay­ment is de­liv­ered in a 5-year in­stall­ment plan for a sup­posed once-and-done cure). It’s al­so on­ly ap­proved for in­fants un­der the age of 2.

Al­though Spin­raza has earned Bio­gen more than $1.5 bil­lion in the first three quar­ters of 2019, Seiger­man said he is “in­creas­ing­ly con­cerned” about its dura­bil­i­ty. Hav­ing launched late 2016 (and ac­crued more than $4.1 bil­lion in record­ed sales so far), the fran­chise may be­gin to wane come H2 2020.

And Roche is go­ing all-in for the three-ri­val ri­val­ry. To­geth­er with PTC and the SMA Foun­da­tion, the phar­ma gi­ant is run­ning more clin­i­cal tri­als with pa­tients rang­ing from new­borns to 60 years old, in­clud­ing those pre­vi­ous­ly treat­ed with oth­er SMA ther­a­pies.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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FDA re­view­ers head back to White Oak in 2023, with lead­er­ship look­ing to ap­pease a new Con­gress

Republicans have taken a stand against the pandemic era habit of lax work-from-home schedules. Now that they’ve wrestled control of the House majority, the FDA’s leadership is playing ball, sending many of the agency’s more than 18,000 employees back to their desks early next year.

Whether this exodus back to White Oak in Silver Spring, MD (many staff will still be allowed to work from home for multiple days per week) will mean more defections to industry and elsewhere remains to be seen.

Jakob Lindberg, Oncopeptides CEO

Scoop: On­copep­tides CEO ‘lean­ing to­ward’ giv­ing in to FDA with­draw­al re­quest amid ‘piss­ing con­test’ with reg­u­la­tors

Oncopeptides CEO Jakob Lindberg is “leaning toward” complying with the FDA’s request to formally and completely pull its cancer drug Pepaxto, he told Endpoints News on Thursday, a day after the biotech announced the agency’s ask. But he also accused US regulators of a double standard and claimed it would be unethical to do so, potentially leaving patients in the wind.

In a phone conversation with Endpoints early Thursday afternoon, Lindberg stressed Oncopeptides hasn’t made any decision yet and that one would come within the “next few weeks.” Lindberg and his team are still deliberating whether contesting the FDA’s decision and starting a potentially lengthy legal process would be worth it.

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