Ver­tex and Covid-19 spell the end for Savara's CF pro­gram

It’s a tough mar­ket for cys­tic fi­bro­sis these days. Just ask Savara.

Two years ago, the Austin-based biotech de­signed and launched a tri­al to test whether their lead GM-CSF drug mol­gradex can re­duce lung in­fec­tion in adult CF pa­tients. At the time, Ver­tex on­ly had 3 CF drugs, they on­ly cov­ered a por­tion of pa­tients, and they weren’t al­ways in­cred­i­bly ef­fec­tive. Then last year came Trikaf­ta, the ex­pen­sive triple-com­bi­na­tion that had sig­nif­i­cant ben­e­fits for around 90% of pa­tients.

So where did that leave Savara? Out of luck, it seems. On Fri­day, the com­pa­ny an­nounced that it was dis­con­tin­u­ing the Phase II tri­al, cit­ing both Ver­tex’s suc­cess and the toll Covid-19 took on pa­tient re­cruit­ment. The news spells the end of de­vel­op­ment for CF and shifts fur­ther fo­cus on the com­pa­ny’s trou­bled lead pro­gram for au­toim­mune pul­monary alve­o­lar pro­teinosis.

For Savara, Trikaf­ta and Covid-19 were a dou­ble-wham­my that hit on two dif­fer­ent fronts. First, the Ver­tex drug be­came the stan­dard of care for CF pa­tients. That meant 8 out of the 14 pa­tients they re­cruit­ed for the study start­ed tak­ing Trikaf­ta while they were tak­ing the Savara in­halant, con­found­ing any at­tempt to tease out whether im­prove­ments were a prod­uct of the Savara drug or the Ver­tex one. Then in March, the com­pa­ny stopped en­rolling pa­tients af­ter Covid-19 hit the US, over­whelmed hos­pi­tal sys­tems and dis­rupt­ed the en­tire clin­i­cal tri­al sys­tem.

Badrul Chowd­hury

CMO Badrul Chowd­hury not­ed 5 pa­tients who were al­so on Trikaf­ta saw three con­sec­u­tive tests with­out in­fec­tion. But it wasn’t clear enough to war­rant fur­ther in­vest­ment.

“While the re­sults of EN­CORE are in­ter­est­ing,” he said, “ad­di­tion­al con­trolled stud­ies would be re­quired to ac­cu­rate­ly un­der­stand the ther­a­peu­tic po­ten­tial of Mol­gradex in com­bi­na­tion with the triple-com­bi­na­tion mod­u­la­tor treat­ment.”

The end of the CF pro­gram con­sol­i­dates fo­cus on their lead in­di­ca­tion, where the ju­ry re­mains out. Last year, the com­pa­ny saw their shares crushed af­ter a Phase III study missed its pri­ma­ry end­point and mul­ti­ple sec­ondary end­points. Al­though the com­pa­ny talked about a high place­bo ef­fect, point­ing to pa­tient-re­port­ed out­comes and lat­er re­leas­ing slides show­ing end­points that “trend­ed” to­ward sig­nif­i­cance, few in­vestors seemed to be­lieve the sto­ry.

But then in De­cem­ber, the FDA hand­ed the com­pa­ny break­through sta­tus on the drug. They ev­i­dent­ly bought some por­tion of the ex­ec­u­tives’ sto­ry, though which part and what the path for­ward is for the com­pa­ny re­mains un­clear.

So­cial: Rob Neville, Savara CEO

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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Covid-19 roundup: WSJ of­fers in­side look at Ox­ford-As­traZeneca deal, in­clud­ing fi­nan­cial terms; Lil­ly plant un­der scruti­ny again

Oxford scientists developing a Covid-19 vaccine are working with British drugmaker AstraZeneca, and together the pair have become one of the frontrunners in the race to end the pandemic. But a new Wall Street Journal report out Wednesday offered a behind-the-scenes look at how that deal came together in the wake of a scholar-led revolt over a potential collaboration with Merck, and included previously unreported financial terms of the AstraZeneca deal.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.

CEO Marc Gleeson (Azura)

Azu­ra Oph­thalmics gets a $20M boost for its R&D work on eye dis­eases

Three years after closing a $16 million Series B, the same group of investors are back to give Azura Ophthalmics a $20 million boost.

That brings the Tel Aviv-Yafo, Israel-based biotech’s total fundraise to $38 million, and should pave the way for a registration study of its lead candidate in Meibomian gland dysfunction (MGD) and related eye diseases, CEO Marc Gleeson told Endpoints News.

The topical candidate, dubbed AZR-MD-001, is designed to address abnormal hyperkeratinization, or the build-up and shedding of proteins at the opening of or within the Meibomian gland. When Meibomian glands become dysfunctional, rapid evaporation of the tear film can occur, leading to dry eye disease.

Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Peter Kolchinsky and Raj Shah (file photo)

Pe­ter Kolchin­sky and Raj Shah's RA Cap­i­tal has $461M more to play with, af­ter 'rapid' in­vest­ment in the last 15 months

Just over 15 months after launching its first venture fund, RA Capital Management is ready for more. And this time the firm is bringing an even bigger load of cash to the table.

Announcing the close of its Nexus II fund on Wednesday, RA said it raised $461 million for investments in private companies across the biotech industry. The first venture fund, which raised $300 million, has churned through roughly 80% of its capital already, a pace that managing partner Raj Shah called unusually quick.