Third Rock-backed Al­lena looks to spring­board a $92M IPO off of a failed PhI­Ib

The IPO mar­ket for biotech has turned hot. But is it hot enough to get Al­lena Phar­ma­ceu­ti­cals in­to the pub­lic are­na?

Three years ago, Al­lena Phar­ma­ceu­ti­cals CEO Alex­ey Mar­golin was talk­ing about us­ing a $25 mil­lion B round to steer the New­ton, MA-based biotech up to the thresh­old of Phase III. A year lat­er, the $53 mil­lion C round was sup­posed to get it in­to the Phase III. And now, $96 mil­lion in, the team at Al­lena has craft­ed a $92 mil­lion IPO de­signed to fund Phase III — even though their drug just qui­et­ly failed a key and very small Phase IIb tri­al.

Alex­ey Mar­golin

Their lead drug is ALLN-177 for hy­per­ox­aluria, a con­di­tion in which an ex­cess lev­el of ox­alate is present in urine — a key build­ing block for dam­ag­ing kid­ney stones.

Ac­cord­ing to the S-1, their Study 713 failed to demon­strate a sta­tis­ti­cal­ly sig­nif­i­cant “re­duc­tion in uri­nary ox­alate ex­cre­tion from base­line to Week 4 of the tri­al” for sec­ondary hy­per­ox­aluria. But the biotech claimed a suc­cess for some post-hoc analy­sis and a sec­ondary end­point: time-weight­ed av­er­age 24-hour uri­nary ox­alate ex­cre­tion. And that’s what they want the FDA to sign off on now as the pri­ma­ry end­point for the piv­otal study, fo­cus­ing on more se­vere cas­es of en­teric hy­per­ox­aluria, which can be trig­gered by in­flam­ma­to­ry bow­el dis­ease or surgery.

The sec­ondary for en­teric hy­per­ox­u­luria — which ac­count­ed for 18 of the 71 pa­tients di­vid­ed be­tween the drug arms and the place­bo group — al­so failed.

From the S-1:

•  In the over­all pop­u­la­tion, re­duc­tion in 24 hour UOx ex­cre­tion from base­line to Week 4 (the pri­ma­ry end­point of the tri­al) was greater in sub­jects treat­ed with ALLN-177 (LS mean(1) = —8.75 mg/24 hour) com­pared to sub­jects who re­ceived place­bo (LS mean = —2.40 mg/24 hour); how­ev­er, the dif­fer­ence be­tween treat­ment groups (LS mean = —6.35 mg/24 hour) did not reach sta­tis­ti­cal sig­nif­i­cance (p(2) = 0.160).

•  In the sub­group with en­teric hy­per­ox­aluria, re­duc­tion in 24 hour UOx ex­cre­tion from base­line to Week 4 was sub­stan­tial­ly greater in sub­jects treat­ed with ALLN-177 (LS mean = —21.31 mg/24 hour) com­pared to sub­jects who re­ceived place­bo (LS mean = —4.86 mg/24 hour), and the treat­ment dif­fer­ence was LS mean = —16.45 mg/24 hour (p = 0.184). The mag­ni­tude of the treat­ment ef­fect was sub­stan­tial­ly greater than what was ob­served in the over­all pop­u­la­tion.

Mar­golin and Bob Gal­lot­to found­ed Al­lena in 2011 af­ter sell­ing Al­nara and its lead drug — an­oth­er oral en­zyme ther­a­py, for cys­tic fi­bro­sis, called lipro­ta­mase — to Eli Lil­ly. Lil­ly then hand­ed it off to An­thera $ANTH af­ter an FDA pan­el panned the drug, which was quick­ly for­mal­ly re­ject­ed. Then An­thera’s shares were crushed late last year af­ter the drug failed a new Phase III, though com­pa­ny ex­ecs blamed the tri­al de­sign on the set­back.

An­thera has a mar­ket cap of $18 mil­lion as it pur­sues an­oth­er piv­otal study of the drug.

Gal­lot­to left Al­lena two years ago.

While Al­lena tout­ed ear­ly-stage pos­i­tive re­sults for their lead ther­a­py, they nev­er put out a re­lease on the mid-stage fail­ure. Now in­vestors will get a chance to size it all up.

Ac­cord­ing to the SEC fil­ing, Mar­golin owns 6.6% of the stock, with most of the rest divvied up among its ven­ture back­ers: Pri­mar­i­ly Fra­zier Health­care, Third Rock, Besse­mer, Fi­deli­ty and HBM.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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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.

US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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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.