FDA chief Got­tlieb is build­ing a reg­u­la­to­ry speed­way to ac­cel­er­ate gene ther­a­py de­vel­op­ment

In a ral­ly­ing cry for gene ther­a­py, FDA Com­mis­sion­er Scott Got­tlieb says he’s de­ter­mined to clear the path­way for drug de­vel­op­ers in a move to ac­cel­er­ate the first wave of gene ther­a­pies point­ed to the mar­ket.

The first ther­a­peu­tic area to ben­e­fit from new sur­ro­gate end­points will be he­mo­phil­ia, Got­tlieb said — im­me­di­ate­ly ring­ing a bell for com­pa­nies like Spark Ther­a­peu­tics $ONCE, Pfiz­er $PFE, Bio­Marin $BM­RN and uniQure $QURE, which are de­vel­op­ing cures for both ver­sions of the bleed­ing dis­or­der. Un­der the yet-to-be-an­nounced guide­lines, fac­tor pro­duc­tion may in some cas­es be suf­fi­cient as a mea­sure of ben­e­fit.

Got­tlieb dis­cussed the FDA’s pol­i­cy plans for gene ther­a­py Tues­day at the an­nu­al board meet­ing of the Al­liance for Re­gen­er­a­tive Med­i­cine. Quot­ing an MIT study that pre­dicts 40 FDA-ap­proved gene ther­a­py prod­ucts by the end of 2022, he ac­knowl­edged both the “breath­tak­ing” pace of progress and his agency’s role in fa­cil­i­tat­ing it all.

“FDA has more than 500 ac­tive in­ves­ti­ga­tion­al new drug ap­pli­ca­tions in­volv­ing gene ther­a­py prod­ucts,” Got­tlieb said. “We’ve re­ceived more than one hun­dred such ap­pli­ca­tions last year alone. This shows the in­ten­si­ty of sci­en­tif­ic work go­ing on in this field.”

To speed things along, Got­tlieb sug­gest­ed, cer­tain gene ther­a­pies may qual­i­fy for the re­gen­er­a­tive med­i­cine ad­vanced ther­a­py (RMAT) des­ig­na­tion — a sta­tus es­tab­lished by the 21st Cen­tu­ry Cures Act that con­fers all the ben­e­fits of fast track and break­through des­ig­na­tions. De­vel­op­ers may al­so even­tu­al­ly ap­ply for ac­cel­er­at­ed ap­proval, where the FDA would be will­ing to ac­cept more un­cer­tain­ty in ex­change for promis­ing ther­a­pies in “dev­as­tat­ing dis­eases.” Longterm ef­fec­tive­ness — or even tra­di­tion­al mea­sure­ments, such as the demon­stra­tion of a re­duc­tion in bleed­ing rates in he­mo­phil­ia — could come in post­mar­ket fol­low-ups.

“The use of reg­istries and re­al-world ev­i­dence are like­ly to play an in­creas­ing­ly im­por­tant role in this re­spect,” the com­mis­sion­er said. “Part of our goal is to move to­ward a sys­tem that al­lows more re­al-time sur­veil­lance of safe­ty ques­tions af­ter new prod­ucts are ap­proved.”

But that still leaves the in­her­ent prob­lems in de­vel­op­ing and com­mer­cial­iz­ing gene ther­a­pies to be solved.

When you com­pare re­views of cell and gene ther­a­pies from those of tra­di­tion­al drugs, Got­tlieb point­ed out, you see that the break­down of clin­i­cal ver­sus prod­uct is­sues is al­most com­plet­ed in­vert­ed. For these ther­a­pies, clin­i­cal ef­fi­ca­cy is of­ten es­tab­lished ear­ly, thus tak­ing up on­ly 20% of the re­view, while re­view­ers of­ten de­vote 80% of the process to work out man­u­fac­tur­ing and qual­i­ty con­cerns.

Got­tlieb spot­light­ed two man­u­fac­tur­ing-re­lat­ed is­sues hin­der­ing the de­vel­op­ment of gene ther­a­py. The in­ef­fi­cient process of pro­duc­ing gene ther­a­py vec­tors — the lentivirus­es and ade­no-as­so­ci­at­ed virus­es that de­liv­ers the “cor­rect” copies of genes to pa­tients — makes it pro­hib­i­tive­ly ex­pen­sive. Fur­ther­more, the con­ven­tion­al phar­ma par­a­digm, which sep­a­rates ear­ly-stage pi­lot man­u­fac­tur­ing from the com­mer­cial process, means some treat­ments would be caught up, or even aban­doned, in the tran­si­tion.

The FDA is try­ing to help on that front, through an ini­tia­tive to im­prove the yield of cell lines and by “ac­tive­ly pur­su­ing new in­vest­ments” in con­tin­u­ous man­u­fac­tur­ing (as op­posed to batch man­u­fac­tur­ing) plat­forms.

With a field that’s mov­ing ahead rapid­ly and a tech­nol­o­gy that’s go­ing to “trans­form med­i­cine and hu­man health,” the FDA is keen to ad­dress any chal­lenges in man­u­fac­tur­ing and clin­i­cal de­vel­op­ment, Got­tlieb said.


Im­age: Scott Got­tlieb.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Is there a recipe for M&A suc­cess? The best and worst buy­out deals in the past decade of­fer some keys to suc­cess — and fail­ure

It’s not easy achieving a solid win in M&A in this industry. But if you follow a few simple guidelines, you may be able to increase your odds of success.
Geoffrey Porges and the team at SVB Leerink went about the “notoriously difficult” task of scoring the biopharma buyout of 2009 to 2019. Sizing up current and expected revenue from the products that were gained, they came up with the 5 winners:
Merck/Schering Plough
Bristol/Medarex
Gilead/Pharmasset
Sanofi/Genzyme
AstraZeneca/Acerta
It says a lot about the field that it’s much easier sorting out the 5 worst deals, though there’s also a lot more competition for that title, notes Porges. As picked by the analysts:
J&J/Actelion
Merck/Cubist
Alexion/Synageva
AbbVie/Stemcentrx
Gilead/Kite

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