No­var­tis an­nounces pos­i­tive da­ta from halt­ed Zol­gens­ma tri­al

The tri­al may be on hold, but No­var­tis says the drug works.

No­var­tis an­nounced to­day new da­ta on a de­liv­ery route to make the gene ther­a­py Zol­gens­ma ap­plic­a­ble and ac­ces­si­ble to old­er kids with spinal mus­cu­lar at­ro­phy, even as the Phase I/II tri­al on that route re­mains on hold. The FDA paused the tri­al in Oc­to­ber af­ter pre­clin­i­cal stud­ies showed loss of neur­al func­tion in some mice. The hold came af­ter an in­dus­try-rock­ing scan­dal over how No­var­tis han­dled da­ta-tam­per­ing in a dif­fer­ent set of pre­clin­i­cal Zol­gens­ma stud­ies.

No­var­tis sub­sidiary AveX­is, which de­vel­oped and mar­kets Zol­gens­ma, said that in the me­di­an dos­ing arm of their STRONG tri­al, the 2-year-old to 5-year-old pa­tients met the pri­ma­ry end­point: They im­proved an av­er­age of 6.0 points on the Ham­mer­smith Func­tion­al Mo­tor Scale-Ex­pand­ed, a met­ric used to mea­sure mus­cle func­tion in kids with spinal mus­cu­lar at­ro­phy. AveX­is said that was twice the im­prove­ment re­quired to be clin­i­cal­ly mean­ing­ful and re­flect­ed ad­vances in three to six mo­tor skills.

David Lennon No­var­tis

In a press re­lease, AveX­is CEO David Lennon praised the re­sults and hint­ed he hoped they would con­vince reg­u­la­tors to re­sume the tri­al. He used the ex­per­i­men­tal com­pa­ny name of the drug.

“STRONG da­ta demon­strat­ed po­ten­tial best-in-cat­e­go­ry pro­file with re­mark­able mo­tor func­tion im­prove­ment fol­low­ing a sin­gle, one-time in­trathe­cal dose,” Lennon said. “We look for­ward to shar­ing these da­ta with reg­u­la­tors to fur­ther our dis­cus­sions to­ward reg­is­tra­tion of in­trathe­cal AVXS-101.”

Ap­proved last year, Zol­gens­ma was the sec­ond gene ther­a­py to reach the mar­ket in the US and just the sec­ond-ever treat­ment for spinal mus­cu­lar at­ro­phy af­ter the 2016 ap­proval of Io­n­is’s and Bio­gen’s Spin­raza. The ther­a­py was lim­it­ed, though, to pa­tients un­der the age of 2. The swift­ness of the ge­net­ic dis­or­ders re­duced the odds of suc­cess the old­er pa­tients got. Most kids with SMA type 1 — the first ap­proved in­di­ca­tion — do not live past the age of 2.

Peo­ple with SMA type 2, how­ev­er, can live in­to ear­ly adult­hood, and No­var­tis has been try­ing to test if an in­trathe­cal in­jec­tion of  Zol­gens­ma — as op­posed to an in­tra­venous one — can im­prove their prog­no­sis.

The hold on the tri­al af­fect­ed the high­est dos­ing arm of the study. Since Oc­to­ber, the Swiss gi­ant has tried to get the FDA to lift the halt, point­ing to the fact that the neur­al dam­age shown in the pre­clin­i­cal study has nev­er ap­peared across the 300-plus pa­tients who have re­ceived the gene ther­a­py. The com­pa­ny said they ex­pect a de­ci­sion from the FDA next quar­ter.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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