Jonathan Hirsch, Syapse founder and president

The re­al-world ev­i­dence project at the FDA adds Syapse to its list of part­ners in a push past tri­al bound­aries

The FDA is mak­ing good on its plans to ex­pand the amount and type of re­al-world ev­i­dence it has on hand to help guide its over­sight of mar­ket­ed drugs and push far past the bound­aries of clin­i­cal da­ta.

The On­col­o­gy Cen­ter of Ex­cel­lence is team­ing up with San Fran­cis­co-based Syapse to ex­pand be­yond elec­tron­ic health records — or EHRs — in build­ing a mul­ti-sourced pro­gram in­volv­ing da­ta drawn from EHRs and reg­istries, as well as mol­e­c­u­lar da­ta from test­ing labs. 

They al­so plan to eval­u­ate “re­al-world end­points for sol­id tu­mors and hema­to­log­i­cal ma­lig­nan­cies, char­ac­ter­iz­ing the us­age and clin­i­cal im­pact of mol­e­c­u­lar test­ing, un­der­stand­ing out­comes and ad­verse events in pa­tients re­ceiv­ing pre­ci­sion med­i­cines rel­a­tive to clin­i­cal tri­al pop­u­la­tions, and in­cor­po­rat­ing pa­tient-re­port­ed out­comes in­to RWE.” And they’ll be work­ing with on­col­o­gists on joint out­comes re­search. 

Ex-FDA com­mis­sion­er Scott Got­tlieb made the use of re­al-world ev­i­dence a key part of the agency’s man­date, out­lin­ing plans to gath­er da­ta on mar­ket­ed drugs to bet­ter eval­u­ate pro­posed la­bel changes as well as hunt for un­known safe­ty fac­tors that should guide pre­scrib­ing.

Sean Khozin FDA

“Re­al-world ev­i­dence from well-de­signed stud­ies meet­ing ap­pro­pri­ate da­ta qual­i­ty stan­dards can help to in­form de­ci­sion-mak­ing and pro­vide in­for­ma­tion re­gard­ing the im­pact of new ther­a­pies in re­al-world pa­tient pop­u­la­tions, par­tic­u­lar­ly those not rep­re­sent­ed in clin­i­cal tri­als,” said Sean Khozin, as­so­ciate di­rec­tor for on­col­o­gy reg­u­la­to­ry sci­ence and in­for­mat­ics in the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “This is es­pe­cial­ly crit­i­cal in pre­ci­sion med­i­cine, where un­der­stand­ing all of the fac­tors that may dri­ve safe­ty and re­sponse is both im­per­a­tive and dif­fi­cult to cap­ture at scale us­ing tra­di­tion­al clin­i­cal tri­als.”

Syapse joins CO­TA and Brigham and Women’s Hos­pi­tal in work­ing with the agency on re­al world da­ta. The FDA and Brigham and Women’s Hos­pi­tal are ex­pand­ing a pact reg­u­la­tors have with Ae­tion to see if they can use RWE — one of the buzzi­est of acronyms in bio­phar­ma — to pre­dict the re­sults of ran­dom­ized clin­i­cal tri­als that have yet to wrap up. And they’re us­ing a batch of 7 up­com­ing stud­ies to test its ac­cu­ra­cy.

“Ad­vanc­ing a deep­er un­der­stand­ing of re­al-world end­points and an­a­lyt­i­cal method­olo­gies is crit­i­cal to as­sur­ing that all stake­hold­ers can have con­fi­dence in the qual­i­ty of ev­i­dence pro­duced and ac­cel­er­at­ing the use of RWE in reg­u­la­to­ry de­ci­sion-mak­ing,” not­ed com­pa­ny founder and pres­i­dent Jonathan Hirsch in a pre­pared state­ment. “The net­work of health sys­tems that Syapse rep­re­sents of­fers a unique op­por­tu­ni­ty to learn from on­col­o­gy pa­tient jour­neys and pop­u­la­tions not well rep­re­sent­ed in tra­di­tion­al clin­i­cal tri­als.”

In a sec­ond big set­back for Covid-19 an­ti­body treat­ment hopes, Re­gen­eron halts en­roll­ment for more se­vere pa­tients

Regeneron has just delivered more bad news for the hope that neutralizing antibodies could be used to treat patients with more severe forms of Covid-19.

The New York biotech said today that an independent monitoring committee recommended halting enrollment of patients who need high-flow oxygen or mechanical ventilation in one of the trials on their antibody cocktail, after finding “a potential safety signal” and “an unfavorable risk/benefit profile.” The news comes a week after the NIH scrapped a trial of Eli Lilly’s Covid-19 antibody after finding it was having little effect on an initial cohort of hospitalized patients.

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Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

CEO Kenji Yasukawa (Astellas)

In ear­ly blow to Ken­ji Ya­sukawa's R&D re­vamp, Astel­las drops out of the TIG­IT race, cit­ing PhI fail­ure

Just after AstraZeneca jumped into the TIGIT race, Astellas quietly disclosed that it was leaving, dropping out of a hunt for an immunotherapy approach that has shown tantalizing promise but remains largely unproven.

Astellas revealed in their second quarter earnings today that they’ve ended development of the anti-TIGIT antibody they acquired in their up to $400 million buyout of Potenza in 2018. The Japanese pharma had been testing it in combination with Keytruda in a 300-person Phase I study on patients with advanced solid tumors. A smaller study testing the antibody alone was completed, 2 years ahead of schedule, in July.

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No­vo Nordisk qui­et­ly nabs a rare 'break­through' sta­tus in NASH for its cash cow

Earlier this month, the small French biotech Inventiva touted themselves as having won the first FDA breakthrough designation in NASH since Intercept grabbed the first one back in 2015, before the disease had become one of biotech’s hottest areas. Unbeknownst to them, though, a much larger European rival had already landed the status months prior.

Novo Nordisk, the Danish metabolic giant, disclosed in their Q3 report Friday that back in August, the FDA gave them breakthrough status for semaglutide, their blockbuster diabetes drug, in NASH, or non-alcoholic steatohepatitis. The designation sets Novo up as a leading contender in a new wave of companies developing treatments for a silent and widespread disease that, despite a series of setbacks, is still viewed by many as a huge potential market.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A p-val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a p-value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

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CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

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As­traZeneca sells off heart fail­ure and hy­per­ten­sion drugs to Chep­lapharm for $400M

Out with the old and in with the new: AstraZeneca is selling off two heart failure and hypertension drugs to Germany-based Cheplapharm, bagging $400 million and making way for development in other areas.

Cheplapharm paid $200 million for the European rights to Atacand (candesartan cilexetil) and Atacand Plus (candesartan cilexetil and hydrochlorothiazide) back in 2018. They’re now doubling that amount for commercial control in more than 70 countries.

News brief­ing: Ax­o­vant faces months of de­lay on lead Parkin­son's gene ther­a­py; Chi­nese CAR-T biotech nabs $100M

One of Axovant’s top gene therapy prospects for its second act is hitting a roadblock that could push its clinical timelines back by almost a year.

In an update, the biotech said it was informed about delays in CMC data and third-part fill-finish issues around mid-October by its manufacturing partner, Oxford Biomedica. Axovant has been developing a suspension-based process for the Parkinson’s drug; with that taking longer than expected, it now believes “it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021.”