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

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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Chi­nese biotech teams with Lil­ly on car­diometa­bol­ic dis­eases; Sen­ate fi­nance chair goes af­ter BMS

Chinese biotech Abbisko Therapeutics is teaming up with Eli Lilly on a new collaboration.

The pair announced Monday they will partner on cardiometabolic diseases and go after an unnamed target, with Abbisko eligible for up to $258 million in milestones. Abbisko will handle primarily the discovery work with input from Lilly, after which Lilly will have the option to further develop and potentially bring the program to market.