Years af­ter a ma­jor tri­al set­back, No­var­tis switch­es gears with SMA drug. This time they're try­ing it for Hunt­ing­ton's

Four years af­ter a Phase I/II set­back in spinal mus­cu­lar at­ro­phy (SMA), No­var­tis is hop­ing its drug branaplam will find more suc­cess in a new neu­ro­log­i­cal in­di­ca­tion: Hunt­ing­ton’s dis­ease.

Jay Brad­ner No­var­tis

The de­ci­sion was an­nounced a year af­ter the head of re­search, Jay Brad­ner, said he did not see a “big op­por­tu­ni­ty” in SMA, ac­cord­ing to Reuters. No­var­tis says it has pre­clin­i­cal da­ta show­ing that branaplam re­duces lev­els of mu­tant hunt­ingtin pro­tein, and SMA da­ta show­ing pa­tients on the drug had re­duc­tions in hunt­ingtin mR­NA. The FDA gave branaplam their or­phan drug des­ig­na­tion, and No­var­tis plans to move forth with a Phase IIb tri­al next year.

“No­var­tis in­tends to start a de­vel­op­ment pro­gram for branaplam to de­ter­mine if it has the po­ten­tial to be a trans­for­ma­tive treat­ment for peo­ple liv­ing with this dev­as­tat­ing con­di­tion,” the com­pa­ny said in a state­ment.

No­var­tis isn’t drop­ping the SMA in­di­ca­tion al­to­geth­er — ac­cord­ing to a com­pa­ny state­ment, branaplam is still “un­der in­ves­ti­ga­tion” for SMA. But a 2-year test­ing hia­tus set No­var­tis be­hind its ri­val Roche, which won ap­proval for its SMA ther­a­py Evrys­di last year.

In 2016, No­var­tis halt­ed en­roll­ment in a Phase I/II study in SMA af­ter “signs of nerve in­jury ap­peared in some an­i­mal tests,” ac­cord­ing to a let­ter it re­leased. The tri­al was re­sumed about 2 years lat­er in Bel­gium, Ger­many, Den­mark and Italy, with ad­di­tion­al nerve tests added to the pro­to­col.

“It is still a long road, but we are pleased to be back on track,” the let­ter read.

Evry­si­di is priced by weight, be­gin­ning at less than $100,000 for in­fants un­der 15 pounds and cap­ping at $340,000 per year. An­a­lysts pre­dict­ed that the drug would threat­en Bio­gen’s SMA drug Spin­raza, which comes in at $382,500 on the low-end. But Mani Foroohar of SVB Leerink pre­dict­ed No­var­tis’ gene ther­a­py Zol­gens­ma would take the lead in young SMA pa­tients. “Giv­en strong da­ta, one-time ther­a­py, and sim­ple IV ad­min­is­tra­tion, we see Zol­gens­ma as re­main­ing the treat­ment of choice for very young SMA pa­tients,” he wrote, ac­cord­ing to a Fierce Phar­ma re­port.

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.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

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Doug Williams, departing Codiak BioSciences CEO

Co­di­ak files for Chap­ter 11 bank­rupt­cy as most ex­ec­u­tives head for the ex­it

Codiak BioSciences has filed for Chapter 11 bankruptcy, spelling an end to the employment of most executives, including founder Doug Williams, as the biotech says it “expects to consummate a sale.”

The eight-year journey at Codiak is nearing an end with Williams; CFO Linda Bain; medical chief David Mauro; scientific head Sriram Sathyanarayanan; legal and compliance chief Yalonda Howze; and SVP of HR Nicole Barna all packing up their bags in the first few days of April. Chief technology officer Konstantin Konstantinov will stay.

Chaim Lebovits, BrainStorm Cell Therapeutics CEO

Brain­Storm gets FDA ad­comm for ALS drug af­ter failed tri­al and RTF

BrainStorm Cell Therapeutics has had a rocky road trying to get its ALS treatment through to the FDA, including a particularly difficult setback after the FDA slapped the company with a refusal to file (RTF) letter in November after it submitted its original BLA in September last year.

But there’s a glimmer of hope for BrainStorm’s ALS treatment, NurOwn – the FDA will hold an advisory committee meeting to discuss the company’s BLA for the treatment, the company announced today.

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Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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Max Colao, OncoVerity CEO

Tiny mul­ti­omics biotech se­cures for­mer J&J drug, new ex­ecs and new fi­nanc­ing

A new spinout from Belgium’s argenx seeks to give new life to a candidate once in-licensed to J&J.

OncoVerity announced Monday afternoon that it had in-licensed cusatuzumab from argenx. The biotech also appointed two new C-suite executives and secured a $30 million Series A round.

CEO Max Colao joins OncoVerity after working as Aurinia’s commercial chief, and stints at Alexion and Amgen. New CMO Clay Smith has been associate chief of the University of Colorado’s hematology division.

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