Ex-Mela chief Gul­fo joins the list of would-be FDA com­mish can­di­dates, col­or-cod­ed drug ap­proval plan in hand

Joseph Gul­fo

A few weeks ago, Joseph Gul­fo spurred a con­sid­er­able round of chat­ter around a pro­pos­al for a col­or-cod­ed ap­proach to ap­prov­ing new drugs. Es­sen­tial­ly, he wants drug de­vel­op­ers to be able to gain an OK based on a range of fac­tors, in­clud­ing bio­mark­er ev­i­dence of ef­fi­ca­cy.

Now, Gul­fo’s name has been raised — by STAT — as an­oth­er pos­si­ble can­di­date for the top job at the FDA, as the bio­phar­ma in­dus­try anx­ious­ly awaits word on the next com­mis­sion­er and how Pres­i­dent Trump plans to de­liv­er on a promise to speed up new ap­provals even as he bat­ters the in­dus­try with crit­i­cism of its pric­ing strate­gies.

In a re­cent op-ed for The Hill, Gul­fo wrote:

Clin­i­cal util­i­ty and clin­i­cal ben­e­fit are high­ly sub­jec­tive and per­son­al ex­pe­ri­ences, best judged by pa­tients and their doc­tors.

Whether to use a drug (based on the po­ten­tial ben­e­fit and risk tol­er­ance of the pa­tient), or to con­tin­ue us­ing a drug giv­en the ben­e­fit-risk pro­file in the in­di­vid­ual pa­tient, is a per­son­al health de­ci­sion.

That ap­proach is more along the lines of the Lib­er­tar­i­an po­si­tions tak­en by two oth­er ru­mored can­di­dates for top jobs at the FDA. Both Jim O’Neill and Bal­a­ji Srini­vasan want to drop stan­dards that they be­lieve have kept drugs out of the mar­ket­place, which can best de­cide on their prop­er use. O’Neill fa­mous­ly said that drugs should be ap­proved based on safe­ty alone, an ap­proach that Gul­fo de­scribes as sell­ing “safe snake oil,” STAT re­ports.

“The FDA has in­creas­ing­ly added new hur­dles for pre-ap­proval re­quire­ments by de­mand­ing long-term out­comes and sur­vival,” Gul­fo main­tains. “A great ex­am­ple of this is in the ap­proval of Pralu­ent and Repatha, med­i­cines that low­er LDL cho­les­terol.”

Gul­fo had his own con­tro­ver­sial ex­pe­ri­ence with the FDA, pur­su­ing a lengthy cam­paign to gain an ap­proval for Mela Sci­ence’s MelaFind sys­tem, even as ex­perts warned of its pos­si­ble mis­use. In the words of Mass­De­vice:

Mela Sci­ences, and Gul­fo, be­came some­thing of a poster child for charges of un­pre­dictabil­i­ty at the FDA dur­ing the pri­or decade.

It’s hard to say how far Gul­fo’s can­di­da­cy can get. STAT’s re­port in­di­cat­ed that he had met with a cou­ple of mem­bers of Trump’s tran­si­tion team, which is a far cry from an ac­tu­al face-to-face with the pres­i­dent.

Scott Got­tlieb re­mains a wide­ly ru­mored can­di­date for Com­mis­sion­er as well. But he’s been able to keep a low pro­file re­cent­ly, un­like the oth­er ri­vals for this po­si­tion.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,700+ biopharma pros reading Endpoints daily — and it's free.

Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.