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.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: As­traZeneca signs 400M vac­cine sup­ply deal with EU; 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK

Amid talks with multiple players, the European Commission has reached its first vaccine supply deal with AstraZeneca, securing 400 million doses of the Oxford candidate for all of its member countries.

The pharma giant said in a press release that the deal builds on the existing agreement with Germany, France, Italy and the Netherlands, announced in June. It was unclear, however, whether that means simply extending the same 400 million doses to all EU countries or doubling the reservation to 800 million doses.

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