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

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.