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.

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.

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.

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.

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.