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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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