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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

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UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Am­gen sev­ers 14-year Cy­to­ki­net­ics part­ner­ship, bail­ing on ome­cam­tiv af­ter mixed PhI­II re­sults

Amgen is shrugging off a 14-year development alliance and the tens of millions of dollars spent to develop a new heart drug at Cytokinetics after a Phase III trial turned up weak data — leaving Cytokinetics to soldier on alone.

Omecamtiv mecarbil technically worked, meeting the primary composite endpoint in the Phase III GALACTIC-HF study. But it missed a key secondary endpoint, which analysts had been following as a key marker for success — reduction of cardiovascular (CV) death. While Cytokinetics celebrated the results, its stock tanked 43% upon the news, and analysts warned of an uncertain path ahead. Now, Amgen wants out.

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Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

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