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.”

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

Brii Bio backs in­fec­tious dis­ease start­up while ink­ing deal for its lead TB drug, dou­bling down on an­tibi­otics

Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas. 

Ab­b­Vie scoops up op­tion to li­cense Har­poon's BC­MA drug for $50M up­front as biotech part­ner preps PhI/II tri­al

AbbVie is arming itself with another set of bow and arrows to shoot at BCMA in multiple myeloma — and this time it has turned to collaborators at Harpoon Therapeutics.

In paying $50 million for an option to license HPN217, AbbVie gains exclusive access to a Tri-specific T cell Activating Construct, or TriTAC, that is being steered to a Phase I/II trial. The dosing of the first patient will call for another $50 million in contingent milestone payment. Once Harpoon comes up with the data, its pharma giant partner will decide whether to officially add it to the arsenal.

Malte Peters, Endpoints UK Bio

Mor­phoSys’ chief sci­en­tist hits the ex­it, trig­ger­ing R&D re­or­ga­ni­za­tion as they shift fo­cus to US in an­tic­i­pa­tion of loom­ing FDA ap­proval

Just a few days after opening up its new US headquarters on Boston harbor in prep for what they believe is the imminent approval of a new CAR-T rival, MorphoSys says that its top scientist is stepping down and they’re reorganizing R&D under development chief Malte Peters.

We don’t know the details about what’s up, aside from the fact that they said in an announcement that 17-year company veteran Markus Enzelberger is leaving his job as chief scientific officer to explore new opportunities. And with his departure, they will be “integrating” research into the development operation.
This is the latest in a pair of high-profile changeups in the executive crew at MorphoSys this year. CEO Simon Moroney announced he would step aside near the beginning of the year. He was replaced by Jean-Paul Kress, the former CEO at Boston-based Syntimmune, which was bought out by Alexion.
The German biotech has been beefing up its US commercial team in preparation for the likely approval of their CD19-targeting antibody tafasitamab — or MOR208. Combined with lenalidomide they believe they can offer a more appealing alternative to Kymriah and Yescarta — the first 2 CAR-Ts, after a trial showed that patients suffering from treatment-resistant DLBCL, a common form of non-Hodgkin’s lymphoma, produced a median on progression-free survival rate 12.1 months with a median duration of response at 21.7 months. The overall response rate among 80 patients was 60% with a 43% complete response rate.
They’ve been discussing a mid-2020 market launch in the US.