Equi­l­li­um's im­muno-in­flam­ma­to­ry drug wins FDA's or­phan drug sta­tus; Van­da sues FDA for man­dat­ing lengthy dog study

→ San Diego-based Equi­l­li­um has nabbed an or­phan drug des­ig­na­tion for EQ001 on the verge of be­gin­ning a Phase Ib/II for the im­muno-in­flam­ma­to­ry drug. Al­so known as itolizum­ab, the drug was in-li­censed from In­dia’s Bio­con, which sells it in the coun­try as a pso­ri­a­sis treat­ment. Equi­l­li­um $EQ, how­ev­er, is fo­cus­ing its US clin­i­cal work on the pre­ven­tion and treat­ment of acute graft-ver­sus-host dis­ease — a strat­e­gy that has al­lowed it to skip fi­nanc­ing rounds and jump straight in­to an IPO.

→ Ear­li­er this week, Pain Ther­a­peu­tics $PTIE di­rect­ed its ire against the “FDA’s pur­port­ed sham­bol­ic reg­u­la­tions.” On the same day Van­da Phar­ma­ceu­ti­cals $VN­DA al­so fumed at the agency, say­ing the US health reg­u­la­tor had im­posed a non-bind­ing re­quire­ment that a nine-month non-ro­dent tox­i­c­i­ty study be con­duct­ed be­fore its drug, tradip­i­tant, can be stud­ied in hu­mans. The com­pa­ny is test­ing the drug in a 12-week dog study, but the agency is in­sist­ing on a 9-month study. When Van­da re­fused to con­duct this study, which usu­al­ly in­volves young bea­gles as the test sub­jects that must be sac­ri­ficed to per­mit eval­u­a­tion of the an­i­mal’s tis­sues, the FDA slapped a par­tial clin­i­cal hold on Van­da tri­als test­ing tradip­i­tant for longer than 12 weeks and did not pro­vide a jus­ti­fi­ca­tion for its de­ci­sion, the com­pa­ny said. Con­se­quent­ly, Van­da has sued the agency.

“The FDA is ig­nor­ing a large body of pub­lished sci­en­tif­ic ev­i­dence which con­cludes that these chron­ic dog stud­ies do not of­fer any ad­di­tion­al use­ful in­for­ma­tion. That pol­i­cy is based on old, out­dat­ed sci­ence and re­quires the killing of too many dogs with­out any sci­en­tif­i­cal­ly jus­ti­fied pur­pose. Yet, com­pa­nies have been ret­i­cent to stand up to the FDA and de­mand that it change its pol­i­cy. Van­da is un­will­ing to ac­cept the sta­tus quo,” com­pa­ny chief Mi­hael Poly­meropou­los said in a state­ment. The al­le­ga­tions by Van­da — which al­so is­sued an open let­ter to the FDA — come months af­ter the agency said it was work­ing on new reg­u­la­tions to re­duce dog test­ing in drug de­vel­op­ment.

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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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.

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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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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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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