Up­dat­ed In­cyte com­bo da­ta for epaca­do­stat/Keytru­da look promis­ing

In­cyte this morn­ing teed up a fresh look at da­ta from its ear­ly-stage study of its close­ly watched IDO1 drug epaca­do­stat com­bined with Mer­ck’s PD-1 check­point ther­a­py Keytru­da. And the up­date — a lit­tle more than week af­ter the first pre­lim­i­nary da­ta were re­port­ed — is en­cour­ag­ing some an­a­lysts to an­tic­i­pate a suc­cess for its piv­otal tri­al of the im­munother­a­py duo, which is now un­der­way.

Steven Stein, In­cyte CMO

In the lead up to the big ES­MO meet­ing this week­end, in­ves­ti­ga­tors for In­cyte $IN­CY say that they tracked pro­gres­sion-free sur­vival rates of 74 per­cent and 57 per­cent af­ter 6 months and 12 months. The study has not yet ma­tured enough for the re­searchers to pin down a me­di­an PFS rate. But the com­plete re­sponse rate has in­creased slight­ly to 26%.

In a state­ment, In­cyte not­ed:

The ob­jec­tive re­sponse rate (ORR) and dis­ease con­trol rate (DCR) re­mained con­sis­tent with the pre­vi­ous­ly pub­lished ab­stract da­ta, at 58 per­cent (12 months) and 74 per­cent (6 months), re­spec­tive­ly. All re­spons­es are con­firmed and on­go­ing (me­di­an fol­low-up among re­spon­ders 56 plus [range of 46 to 90 plus] weeks).

RBC’s Simos Sime­oni­dis of­fered a quick thumbs up on the re­sults this morn­ing, not­ing that the da­ta look good com­pared to ri­val com­bi­na­tions. He ticked off rea­sons for op­ti­mism:

1) af­ter three more months of fol­low-up, since the March 28th da­ta cut­off of the ab­stract, the PFS was not reached; this in­creas­es our con­fi­dence that the com­bo of epaca­do­stat+pem­bro will end up with mPFS that will be su­pe­ri­or of the ipi+ni­vo com­bo (mPFS 11.5 months); 2) the 12- and 6-month PFS dis­cussed in the poster of 57% and 74%, re­spec­tive­ly, com­pare fa­vor­ably with the re­spec­tive PFS of re­sults from tri­als of oth­er im­munother­a­py reg­i­mens; 3) the re­sponse rate re­mained ro­bust and steady, in­clud­ing an ad­di­tion­al pa­tient whose PR be­came a CR; all this while the re­spons­es re­mained durable; 4) 83% (5/6) of melanoma pa­tients treat­ed with 100mg or more of epaca­do­stat were re­spon­ders; 5) the safe­ty of the com­bi­na­tion re­mained be­nign, both in terms of num­bers (19% G3/4) and in terms of types of TEAEs. There are da­ta in the poster about re­spons­es in ad­di­tion­al tu­mors, in­clud­ing lung and kid­ney; how­ev­er, we have a hard­er time hav­ing as strong an opin­ion on them, giv­en the small num­ber of pa­tients test­ed in each tu­mor type. Our key take­away is that epaca­do­stat is a very ac­tive and safe agent in melanoma and this da­ta pre­sen­ta­tion ap­pears to con­firm/re­in­force the com­pa­ny’s de­ci­sion to move in­to the Phase III set­ting.

In­cyte has been run­ning Phase I and Phase II stud­ies of the com­bo in a range of tu­mor types. It launched the Phase III piv­otal tri­al in June, with plans to re­port da­ta in 2018.

These kinds of com­bi­na­tion stud­ies in­volv­ing check­point drugs have be­come all the rage in the on­col­o­gy field. In­cyte in par­tic­u­lar, though, has been busi­ly part­ner­ing with a va­ri­ety of drugs, spurring high hopes that it’s get­ting clos­er to the mar­ket with a lead­ing IDO1 drug. The ther­a­py tar­gets a mech­a­nism used by can­cer cells to avoid an im­mune sys­tem at­tack, and a com­bo with a check­point would seem to of­fer a one-two punch al­low­ing a full-on im­mune re­sponse to can­cer cells.

“We are ex­cit­ed to share fur­ther da­ta with ad­di­tion­al fol­low-up from the Phase 1 por­tion of the ECHO-202 study,” said Steven Stein, In­cyte’s chief med­ical of­fi­cer, in a state­ment. “The durable re­spons­es seen in pa­tients with treat­ment-naïve ad­vanced or metasta­t­ic melanoma reaf­firm the ac­tiv­i­ty of this im­munother­a­py com­bi­na­tion, and we look for­ward to the read-out of ECHO-301, the on­go­ing, piv­otal Phase III tri­al.”

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.

Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.

Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,500+ biopharma pros reading Endpoints daily — and it's free.