A snap­shot of up­beat PhII NASH da­ta — from a very small tri­al — trig­gers a fren­zy for Viking’s stock

Viking Ther­a­peu­tics $VK­TX has proven once again that noth­ing whips up biotech in­vestors like pos­i­tive Phase II num­bers for NASH.

The San Diego-based biotech’s shares rock­et­ed up more than 100% Tues­day morn­ing on Viking’s boast that VK2809 slashed liv­er fat con­tent — a bio­mark­er for the dis­ease — while de­liv­er­ing a drop of 20% or more in LDL in the mid-stage study. 

Jef­feries’ Michael Yee looked it over and boiled the da­ta down to this: “57-60% me­di­an rel­a­tive change in liv­er fat by MRI-PDFF in­clud­ing a 77-91% re­spon­der rate on >30% fat re­duc­tion hur­dle; and they looked at ‘su­per’ re­spon­ders of >50% fat re­duc­tion of which was 61-73% re­sponse rate….while cross-tri­al in non-iden­ti­cal pop­u­la­tions, MDGL was a bit low­er at 36-42% me­di­an rel­a­tive change and 60-75% achiev­ing the >30% re­duc­tion thresh­old. (2) LDL-cho­les­terol re­duc­tion was ‘greater than 20%’ and in the ball­park of MDGL al­so 19-21% on an op­ti­mal dose of MGL-3196.”

Ro­hit Loom­ba

That looks good rel­a­tive to the pack chas­ing a big ap­proval for NASH, a block­buster sized mar­ket, notes Yee. But he al­so ad­vis­es a lit­tle cau­tion be­fore the street los­es its head. The caveats: This is a 45-per­son Phase II, of­fer­ing lit­tle more than a snap­shot of the da­ta to come. Yee adds that he be­lieves that the biotech will need to run an­oth­er Phase II be­fore they move in­to a late-stage piv­otal, and that puts any po­ten­tial mar­ket­ing de­ci­sion years down the road.

He not­ed: “(D)ata is still ear­ly vs peers and has lim­it­ed safe­ty in­fo and will re­quire an­oth­er big­ger Phase II biop­sy study like­ly be­fore start­ing any Phase III.”

Viking, though, was stay­ing strict­ly op­ti­mistic about its chances.

“Pre­vi­ous stud­ies by our group have shown that a 30% or greater re­duc­tion in MRI-PDFF is as­so­ci­at­ed with high­er odds of his­to­log­ic re­sponse in NASH. The quan­tum of liv­er fat re­duc­tion along with LDL-low­er­ing prop­er­ties of VK2809 are po­ten­tial­ly like­ly to be ben­e­fi­cial in pa­tients with non-al­co­holic steato­hep­ati­tis (NASH) who have a sig­nif­i­cant risk of not on­ly liv­er fi­bro­sis pro­gres­sion but al­so car­dio­vas­cu­lar dis­ease,” stat­ed Ro­hit Loom­ba, a pro­fes­sor at Uni­ver­si­ty of Cal­i­for­nia at San Diego.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,200+ biopharma pros reading Endpoints daily — and it's free.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,200+ biopharma pros reading Endpoints daily — and it's free.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,200+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,200+ biopharma pros reading Endpoints daily — and it's free.

Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Roche. File photo

Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 67,200+ biopharma pros reading Endpoints daily — and it's free.

KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.