AveX­is wants $400M more for promis­ing SMA gene ther­a­py; Shang­Phar­ma part­ners with Ic­ahn School of Med­i­cine at Mount Sinai

AveX­is $AVXS is rais­ing more cash to fu­el its gene ther­a­py for spinal mus­cu­lar at­ro­phy with a pub­lic of­fer­ing of up to $400 mil­lion of shares at $117.5 per share. The plan, laid out in an SEC fil­ing, is to al­lo­cate $225 to $270 mil­lion to re­search, man­u­fac­tur­ing, clin­i­cal and reg­u­la­to­ry ac­tiv­i­ties — main­ly in SMA, but al­so for pro­grams in Rett syn­drome and ALS. Around $140 mil­lion to $175 mil­lion will go to pre-com­mer­cial ac­tiv­i­ties for the po­ten­tial launch of SMA block­buster con­tender AVXS-101, in­clud­ing med­ical af­fairs, com­mer­cial ini­tia­tives and li­cens­ing ac­tiv­i­ties. The Chica­go-based biotech is com­ing on strong in the SMA field, threat­en­ing to cut in­to Bio­gen’s Spin­raza fran­chise.

Shang­Phar­ma In­no­va­tion, a VC firm that fo­cus­es on seed in­vest­ments, has struck a deal with Ic­ahn School of Med­i­cine at Mount Sinai to fu­el re­search be­hind ear­ly-stage drugs. Un­der the mul­ti-year col­lab­o­ra­tion deal, Shang­Phar­ma will pro­vide R&D fund­ing at the school’s clin­i­cal in­sti­tutes in New York. Led by CEO Wal­ter Moos, Shang­Phar­ma will al­so pro­vide re­search ser­vices from CRO Shang­hai ChemPart­ner. “This part­ner­ship was fu­eled by the ground­break­ing tech­nolo­gies from in­no­va­tors here at Mount Sinai,” said Erik Li­um, se­nior VP of Mount Sinai In­no­va­tion Part­ners, in a state­ment. “Our ex­per­tise in com­mer­cial­ly-rel­e­vant trans­la­tion­al re­search, cou­pled with Shang­Phar­ma In­no­va­tion’s drug de­vel­op­ment ca­pa­bil­i­ties will ad­vance much need­ed med­i­cines to pa­tients world­wide.”

→ Lon­don’s Hik­ma Phar­ma­ceu­ti­cals has re­cruit­ed Suren­dera Tya­gi as its new CSO and glob­al head of R&D. Tya­gi comes from Ger­man heath­care gi­ant Fre­se­nius Kabi (ETR: FRE), where he led the com­pa­ny’s US In­no­va­tion & De­vel­op­ment Cen­ter and ex­pand­ed the Fre­se­nius’ on­col­o­gy busi­ness. At Hik­ma, Tya­gi will be first tasked with boost­ing non-in­jectable R&D to ex­pand the com­pa­ny’s pipeline. The hire comes at a time when Hik­ma’s gener­ics unit is tak­ing some hits, lead­ing to the com­pa­ny’s stock (LON:HIK) falling 47% over the past year. Tya­gi’s re­cruit­ment may be part of the com­pa­ny’s strat­e­gy to re­cov­er through di­ver­si­fi­ca­tion in the pipeline. Be­fore Fre­se­nius Kabi, Tya­gi worked at Dar­bur Phar­ma, Roche, IGEN, Ab­bott, and Schwarz Phar­ma.

Pfiz­er $PFE is team­ing up with Foun­da­tion Med­i­cine $FMI to de­vel­op, pro­vide reg­u­la­to­ry sup­port, and help com­mer­cial­ize com­pan­ion di­ag­nos­tics for Pfiz­er’s on­col­o­gy port­fo­lio. Foun­da­tion Med­i­cine makes an FDA-ap­proved ge­nom­ic pro­fil­ing test for sol­id tu­mors called Foun­da­tionOne CDx, which the col­lab­o­ra­tion hopes to im­prove on. The com­pa­ny al­so makes an­a­lyt­ics soft­ware called Foun­da­tion­In­sights, which Pfiz­er can lever­age to ac­cel­er­ate dis­cov­ery and de­vel­op­ment of can­cer drugs. Pfiz­er cur­rent­ly has 10 FDA-ap­proved can­cer med­i­cines that treat sol­id tu­mors and hema­to­log­ic ma­lig­nan­cies. Its on­col­o­gy pipeline al­so in­cludes 17 as­sets in clin­i­cal de­vel­op­ment and 19 Phase III stud­ies.

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.

Franz-Werner Haas, CureVac CEO

3 weeks, 3 big in­vest­ments capped by a $213M IPO bring Cure­Vac’s in­stant-cash raise to a cool bil­lion dol­lars

CureVac jumped onto Nasdaq Thursday night, landing with an extra $213 million after selling a batch of shares at $16 a pop. Add in an extra $118 million share purchase by founder Dietmar Hopp — who owns a controlling interest — and another $640 million from deals and the German biotech has raised a cool billion dollars in the space of just 3 weeks.

The company’s stock $CVAC will now start trading this morning, with analysts eager to find out whether the go-go atmosphere on Wall Street will swell the biotech’s share price.

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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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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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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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