Astel­las signs on pluripo­tent stem cell part­ners at Ex­CellThera; Lit­tle Uni­cy­cive brings home $25M af­ter float­ing a small IPO

Astel­las has signed up on rights for a batch of pluripo­tent stem cells from Mon­tre­al-based Ex­CellThera, cen­ter­ing on the in vit­ro use of their UM171 com­pound.

Astel­las is hand­ing over an un­spec­i­fied up­front pay­ment and promis­ing a slate of mile­stones to get the col­lab­o­ra­tion un­der­way.

Guy Sauvageau, CEO and founder of Ex­CellThera, not­ed:

While Ex­CellThera has fo­cused on de­vel­op­ing our core strength in oth­er ther­a­peu­tic ar­eas, Astel­las has in­vest­ed sig­nif­i­cant­ly in PSC-re­lat­ed pro­grams and is ab­solute­ly the right part­ner for us in that re­gard.

John Car­roll

Lit­tle Uni­cy­cive brings home $25M af­ter float­ing a small IPO

Not all biotech IPOs in­volve big, up­sized of­fer­ings and mega­cash win­nings. Take the case of Uni­cy­cive Ther­a­peu­tics.

The biotech has just raised $25 mil­lion gross by sell­ing 5 mil­lion shares at $5 a pop. That comes in at the low end of the range.

Uni­cy­cive is de­vel­op­ing drugs for kid­ney dis­ease, and gave “un­der­writ­ers a 45-day op­tion to pur­chase up to an ad­di­tion­al 750,000 shares of its com­mon stock and/or war­rants to pur­chase up to an ad­di­tion­al 600,000 shares of its com­mon stock, at the ini­tial pub­lic of­fer­ing price, less the un­der­writ­ing dis­counts and com­mis­sions.” — John Car­roll

Aslan gets a $45M loan to pay for a loom­ing PhII study

Af­ter ex­pe­ri­enc­ing a host of prob­lems with its ear­ly can­cer drugs, Sin­ga­pore-based Aslan Phar­ma­ceu­ti­cals is now tak­ing out a $45 mil­lion loan to fund mid-stage work on ASLAN003. They’ll start with a fo­cus on in­flam­ma­to­ry bow­el dis­ease with a Phase II planned for ear­ly 2022.

Aslan has been in plen­ty of hot wa­ter with in­vestors, es­pe­cial­ly af­ter a 2019 tri­al fail­ure. So they’ve turned to K2 HealthVen­tures for the first $20 mil­lion loan with the rest of the mon­ey sub­ject to “cer­tain terms and con­di­tions.”

“These re­sources pro­vide ad­di­tion­al work­ing cap­i­tal, strength­en our bal­ance sheet, and en­hance our fi­nan­cial flex­i­bil­i­ty by ex­tend­ing our ex­pect­ed cash run­way through late 2023, as we look for­ward to ex­pand­ing our clin­i­cal ac­tiv­i­ties for ASLAN003,” said Aslan CEO Carl Firth. — John Car­roll

Cure­Vac ex­ec­u­tives ditched shares be­fore vac­cine flop news broke: re­port

On Tues­day, Busi­ness In­sid­er re­port­ed that four mem­bers of Cure­Vac’s board ditched large blocks of shares worth more than $38 mil­lion as the com­pa­ny’s Covid-19 vac­cine can­di­date floppedWednes­day, the com­pa­ny clapped back, deem­ing the re­ports un­true.

The price of the Ger­man biotech dropped around 50% af­ter the dis­ap­point­ing ef­fi­ca­cy re­sults were an­nounced. But board mem­bers sold near­ly all of their re­main­ing shares, in­clud­ing com­pa­ny co-founder Flo­ri­an von der Mülbe, who ditched about half of his by June 21, when the four ex­ec­u­tives re­port­ed such to the SEC. Cur­rent chief tech­nol­o­gy of­fi­cer Mar­i­o­la Fotin-Mleczek sold 99% of shares, Busi­ness In­sid­er re­ports.

In a state­ment emailed to End­points News, a Cure­Vac spokesper­son said:

The re­ports that in­di­vid­ual mem­bers of the Board of Man­age­ment have sold al­most all of their blocks of shares is not true. We con­tra­dict this re­port­ing, the fol­low­ing is cor­rect: As stat­ed in the rel­e­vant SEC doc­u­ments, the Cure­Vac NV board mem­bers have blocks of shares in the form of vir­tu­al shares (Vir­tu­al Share Own­er­ship) as part of their com­pen­sa­tion. Parts of these vir­tu­al shares are con­vert­ed in­to re­al shares and made avail­able ac­cord­ing to de­fined de­vel­op­ment mile­stones for build­ing up the com­pa­ny – e.g. an IPO, start of a clin­i­cal ap­proval study for the first phar­ma­ceu­ti­cal prod­uct, etc. For the IPO on the New York tech­nol­o­gy com­pa­ny ex­change NAS­DAQ, this part was 10%. In our 20F doc­u­ment, you can see how that per­cent­age, af­ter tax, trans­lates in­to re­al­iz­able shares for the board mem­bers. Even if these shares were com­plete­ly sold, they would on­ly rep­re­sent a small pro­por­tion of the re­spec­tive block of shares held per board mem­ber. The rep­re­sen­ta­tion that an al­most com­plete sale of com­pa­ny shares by the board of di­rec­tors due to a lack of faith in Cure­Vac is not ap­plic­a­ble – we dis­agree.

Josh Sul­li­van

NASH liv­er dis­ease drug deemed safe in PhI­Ia

He­p­i­on Phar­ma­ceu­ti­cals an­nounced that its Phase IIa tri­al of a liv­er dis­ease drug for the treat­ment of non-al­co­holic steato­hep­ati­tis was safe and well-tol­er­at­ed, with few side ef­fects.

The drug — dubbed CRV431 — was pro­vid­ed in ei­ther a 75 mg or 225 mg oral dose once dai­ly, and reached max­i­mum con­cen­tra­tions with­in 30 hours.

“Sta­tis­ti­cal sig­nif­i­cance in a dose re­sponse on ALT is very en­cour­ag­ing, sug­gest­ing a rapid drug ef­fect. A thor­ough re­view of lit­er­a­ture by our group sug­gest­ed that a 10% to 15% de­cline in ALT in four weeks over place­bo would in­di­cate a ben­e­fi­cial drug ef­fect,” SVP of clin­i­cal phar­ma­col­o­gy Patrick Mayo said in a state­ment. “Our clin­i­cal phar­ma­col­o­gy group has al­ready de­vel­oped a pop­u­la­tion phar­ma­co­ki­net­ic-phar­ma­co­dy­nam­ic, or PK-PD, mod­el which pre­dicts CRV431 blood con­cen­tra­tion ef­fect on ALT re­duc­tions, which is not usu­al­ly pos­si­ble at this ear­ly stage in drug de­vel­op­ment.” — Josh Sul­li­van

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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Covid-19 roundup: J&J boost­er shot da­ta show promise; CD­C's ACIP meet­ing this week to dis­cuss Pfiz­er boost­ers

J&J revealed a summary of new Covid-19 vaccine data today, including new results showing booster shots may help with protection.

A Phase III study (ENSEMBLE 2) looked at booster shots at two different points in time: a second shot 56 days after the first shot, or a second shot six months after the first. The eight-week shot showed increased protection against symptomatic Covid-19, with the following levels of protection: