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.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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