Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sud­den ex­plo­sion in treat­ments for spinal mus­cu­lar at­ro­phy, a neu­rode­gen­er­a­tive con­di­tion that once led pa­tients — of­ten young ones — with a grim prog­no­sis and no op­tions. The prog­no­sis still isn’t rosy, but now there are three FDA-ap­proved op­tions, enough to make the choice of one dif­fi­cult.

Now a fourth po­ten­tial op­tion has en­tered the mix. To­day, Schol­ar Rock an­nounced the re­sults from a proof-of-con­cept test­ing their SMA drug by it­self and in com­bi­na­tion with Io­n­is’ Spin­raza, show­ing that all pa­tient co­horts im­proved on stan­dard scales used for mea­sur­ing mo­tor func­tion in peo­ple with SMA.

An­a­lysts, who had been more fo­cused on the com­pa­ny’s can­cer pipeline, greet­ed the re­sults with cau­tious en­thu­si­asm. The com­pa­ny’s mar­ket val­ue dou­bled with­in hours. The Cam­bridge biotech $SRRK is now worth around $850 mil­lion.

“We view the da­ta as high­ly en­cour­ag­ing and pro­vid­ing par­tic­u­lar­ly ro­bust proof of con­cept with­in Type 2 ear­ly ther­a­py ini­tia­tors,” Cowen’s Marc Frahm said in a note to in­vestors. “As a re­sult, we be­lieve SRK-015 has a clear line of site to a $1B rev­enue op­por­tu­ni­ty.”

Jef­feries’s Michael Yee not­ed that it’s dif­fi­cult to in­ter­pret some of the open-la­bel da­ta, but said the re­sults were en­cour­ag­ing in a group of pa­tients who would nor­mal­ly ei­ther be sta­ble or de­clin­ing. SRK-015 al­so works dif­fer­ent­ly than ex­ist­ing SMA drugs, open­ing mul­ti­ple paths to help pa­tients and com­mer­cial­ize, Wed­bush’s David Nieren­garten added.

“Giv­en the unique MOA of SRK-015 in the SMA space, we an­tic­i­pate the com­mer­cial out­look of SRK-015 could be both as a com­bi­na­tion agent to sup­ple­ment ex­ist­ing ther­a­pies (e.g. nusin­ersen, Zol­gens­ma) and po­ten­tial­ly as a monother­a­py as well,” Nieren­garten said in a note to in­vestors.

Schol­ar Rock test­ed their treat­ment, an an­ti­body de­signed to in­hib­it the mus­cle growth re­cep­tor myo­statin, in 39 pa­tients with type 2 or type 3 SMA.

In the first group, 23 pa­tients be­tween the ages of 7 and 21 with type 3 SMA, a rel­a­tive­ly mild form of the dis­ease, were giv­en SRK-015 open-la­bel for 6 months. The 11 pa­tients who were not tak­ing Spin­raza saw a 0.7 point bump in their mo­tor score, while those who weren’t tak­ing it saw an 0.3 point bump. Over­all, 12 out of 23 pa­tients saw im­prove­ments of at least 1 point and 6 out of 23 saw im­prove­ments of at least 3 points. One pa­tient each on monother­a­py and com­bi­na­tion ther­a­py saw a 5-point in­crease.

In the sec­ond open-la­bel group, 15 pa­tients who had type 2 SMA or had type 3 and couldn’t walk re­ceived SRK-015 on top of Spin­raza. One missed the dos­es for Covid-19 re­lat­ed rea­sons. The rest, on av­er­age, im­proved by 1.4 points on a mo­tor scale, with 10 pa­tients im­prov­ing by at least 1 point, 1 by at least 3 points and 2 by at least 6 points.

In the third group, twen­ty pa­tients un­der the age of 5 with type 2 SMA were ran­dom­ized to re­ceive ei­ther high dose or low dose of SRK-015 along with Spin­raza. The dif­fer­ence be­tween the two groups was sub­stan­tial, with those who re­ceived the high dose im­prov­ing by more than twice as much as the low dose: 5.6 points com­pared with 2.4 points. All 9 evalu­able high dose pa­tients im­proved by at least 1 point, with 6 im­prov­ing by 3 and 5 im­prov­ing by 6.

Schol­ar Rock not­ed that the im­prove­ments had not yet plateaued when they reached the 6-month mark for ei­ther co­horts 2 and 3. So an­a­lysts are ea­ger to see the 12-month da­ta, both to track whether the im­prove­ments can con­tin­ue and whether any pos­si­ble place­bo ef­fect wanes.

It’ll now be one of two main points of fo­cus go­ing for­ward, with in­vestors al­so keep­ing a keen eye on an im­muno-on­col­o­gy pro­gram now in Phase I. Yung Chyung, Schol­ar Rock’s CMO, said they’ll soon talk to reg­u­la­tors about a piv­otal SMA tri­al.

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.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

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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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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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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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.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

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

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.