Sarep­ta read­ies FDA pitch af­ter a small study spot­lights 1% dy­s­trophin add for golodirsen in Duchenne MD

Af­ter win­ning an FDA ap­proval for Ex­ondys51 as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy with­out hav­ing to ac­tu­al­ly pro­duce da­ta to prove it worked, Sarep­ta $SRPT fol­lowed up this new morn­ing with a fresh set of da­ta from a small study un­der­scor­ing the abil­i­ty of its ex­per­i­men­tal fol­lowup ex­on-skip­ping drug golodirsen to trig­ger added pro­duc­tion of dy­s­trophin.

Doug In­gram, Sarep­ta CEO

The num­bers it cites, though, are tiny. De­spite that, Sarep­ta is telling an­a­lysts to­day that it plans to see if the FDA could be will­ing to give it an ac­cel­er­at­ed green light.

Prin­ci­pal in­ves­ti­ga­tor Francesco Muntoni not­ed:

All (25) treat­ed boys showed the an­tic­i­pat­ed ex­on skip­ping af­ter treat­ment and this re­sult­ed in a mean in­crease of dy­s­trophin pro­tein, as mea­sured by West­ern blot, from 0.095 per­cent at base­line to 1.019 per­cent of nor­mal af­ter at least one-year of treat­ment with golodirsen.

To un­der­score, that’s a claimed win with slight­ly more than 1% of nor­mal dy­s­trophin. And it will ar­rive at the FDA af­ter an in­ter­nal war was fought over Ex­ondys51, which Janet Wood­cock even­tu­al­ly won by shov­ing aside a line­up of reg­u­la­tors that tried to get in her way.

Some of the an­a­lysts found the out­come to be more than a lit­tle lean. Not­ed RBC’s Matthew Eck­ler:

To­day’s re­lease con­firms the abil­i­ty of Sar­pe­ta’s ex­on-skip­ping ap­proach to in­crease dy­s­trophin in the mus­cles of DMD pa­tients, in our view. How­ev­er, re­call that dur­ing FDA re­view of Ex­ondys 51’s NDA, the agency raised con­cerns around the clin­i­cal ben­e­fit of pro­duc­ing small amounts of dy­s­trophin (cit­ing 10% as the thresh­old for clin­i­cal ben­e­fit). In­deed, one of the bear cas­es we hear for SRPT is that the small amount of dy­s­trophin pro­duc­tion and lack of a con­firmed clin­i­cal ben­e­fit leave the door open to pay­or push­backs and de­nials. We’d still ex­pect some push­back around the head­line “1% dyst. How­ev­er re­gard­less of the ul­ti­mate clin­i­cal ben­e­fit, we think that with the cur­rent da­ta re­lease (and pos­si­bil­i­ty of ac­cel­er­at­ed ap­proval), in­vestors will start as­crib­ing val­ue to Sarep­ta’s pipeline, and that shares will bet­ter re­flect the in­trin­sic val­ue of the com­pa­ny be­yond Ex­ondys 51.

Bri­an Sko­r­ney at Baird likes it. His rea­sons in­clude:

Re­call Sarep­ta sub­mit­ted base­line and 48-week treat­ment dy­s­trophin analy­ses to the FDA last year from the PRO­MOVI study, which showed eteplirsen in­creased dy­s­trophin from 0.16% of nor­mal to 0.44% at week 48, a 0.28% ab­solute in­crease. To­day’s SRP-4053 re­sults are clear­ly more im­pres­sive, with an in­crease from 0.095% to 1.019% (p<0.001), al­most 3.5x the dy­s­trophin pro­duc­tion seen in the PRO­MOVI pa­tients. Get­ting across the 1% of nor­mal thresh­old ap­pears to be an im­por­tant psy­cho­log­i­cal thresh­old, as crit­ics have of­ten cit­ed the “frac­tion of a per­cent” is­sue for eteplirsen.

The com­pa­ny is out to prove that its ex­on-skip­ping tech does work ef­fec­tive­ly, but the biotech still has a very long way to go on that score. Nev­er­the­less, with some help­ful cheer­lead­ing on the side­lines, its stock jumped 15% on the re­lease.

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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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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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.

Eu­ro­pean Union aims to es­tab­lish patent workaround in case of emer­gen­cies while try­ing to strength­en its own IP

The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.

Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.

Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

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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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.