Scott Got­tlieb will push to pub­lish CRLs; Marathon's demise of­fers the in­dus­try a cau­tion­ary tale

End­points News as­sess­es the big bio­phar­ma sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Scott Got­tlieb did the right thing of­fer­ing to pub­lish those CRLs

Dur­ing Scott Got­tlieb’s last round of writ­ten an­swers to the Sen­ate com­mit­tee re­view­ing his nom­i­na­tion as FDA com­mis­sion­er, I was pleased to see him vol­un­tar­i­ly pro­pose that the agency start pub­lish­ing com­plete re­sponse let­ters. Got­tlieb has done that be­fore, but so far we haven’t seen a recom­mit af­ter his nom­i­na­tion.

Why is this so im­por­tant?

We know that there have been a num­ber of in­stances when drug com­pa­nies re­ly on the agency’s si­lence to put a PR spin on their CRLs. Or they just don’t say any­thing at all, which is just as com­mon.

Frankly, af­ter com­pa­nies have had a chance to talk up their prospects — of­ten for years — for drugs on­ly to see them turned back at the fin­ish line with­out any hon­est ac­count­ing for the re­jec­tion is un­ac­cept­able. Physi­cians, pa­tients, in­vestors, tri­al sub­jects (and, yes, jour­nal­ists) have a right to find out what trig­gers a reg­u­la­to­ry boo­by trap.

No one forced Got­tlieb’s arm on this. There was no spe­cif­ic query on CRLs. He pro­posed it, with a caveat on redact­ing pro­pri­etary in­fo, which is the right thing to do — one rea­son why we’ve been sup­port­ive of his nom­i­na­tion. It’s al­so a prac­ti­cal way to dis­tance him­self from ac­cu­sa­tions from some De­moc­rats his past busi­ness ties leave him hope­less­ly con­flict­ed.

The com­mit­tee vote send­ing the nom­i­na­tion to the Sen­ate floor will hope­ful­ly be act­ed on in the next cou­ple of weeks. The soon­er the bet­ter. It’s time to get start­ed on the next chap­ter on the FDA and bio­phar­ma.

As PTC preps a con­tro­ver­sial launch of de­flaza­cort, Marathon is wind­ing down

In a Sun­day news scoop, we re­vealed that Marathon Phar­ma­ceu­ti­cals is be­ing wound down now that PTC has ac­quired US rights to its steroid de­flaza­cort.

These days, get­ting held up as an ex­am­ple of ram­pant price goug­ing can cre­ate an en­vi­ron­ment that is too tox­ic to deal with.

The next step in this process will be to see how much of the tox­i­c­i­ty is ab­sorbed by PTC, which bought the drug and has the un­en­vi­able task now to try and come up with an ac­cept­able price af­ter Marathon failed spec­tac­u­lar­ly at $89,000 a year. For par­ents who had been buy­ing it from the UK’s Mas­ters at a lit­tle more than $1,000, that was a shock. How PTC hopes to make cash now is hard to un­der­stand.

You can be sure that PTC had a firm price in mind when they bought this drug. But it ap­pears we’ll have to wait in­to ear­ly May to get a look at the stick­er.  We’ll be wait­ing.

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.

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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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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FDA's ODAC shrugs off ob­jec­tions to Mesoblast's GVHD drug for chil­dren, vot­ing 8-2 in fa­vor and im­prov­ing the odds of an ap­proval

The FDA’s Oncologic Drugs Advisory Committee once again waved through an investigational drug, clearing the potential final hurdle before the agency’s decision.

Thursday’s winner was Mesoblast $MESO, an Australian stem-cell company that submitted its Ryoncil drug for the treatment of steroid refractory acute graft-versus-host disease in children younger than 12. ODAC gave Ryoncil the thumbs up by an 8-2 vote, shrugging off concerns about trial design and pushing aside the need for an additional study.

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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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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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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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