Oph­thal­mol­o­gy biotech up­start is ask­ing in­vestors to bet $85M-plus on their late-stage ri­val to Resta­sis and Xi­idra

Sev­en months af­ter Oys­ter Point Phar­ma bridged its way in­to a Phase III study with $93 mil­lion in ven­ture cash, the biotech is step­ping back up to see if pub­lic in­vestors are in a mood to back their play to jump in­to a big league mar­ket with a mi­nor league team.

The Prince­ton, NJ-based biotech teed up an IPO on Fri­day, out­lin­ing their case on go­ing af­ter dry-eye dis­ease — a block­buster mar­ket that ac­counts for tens of mil­lions of pa­tients. On the drug side, that’s a mar­ket that has been dom­i­nat­ed by Al­ler­gan’s cash cow Resta­sis — now be­ing carved up by gener­ics as Ab­b­Vie buys out the com­pa­ny — and Xi­idra, a ther­a­py which Take­da sold off to No­var­tis in a $5.3 bil­lion deal in the wake of the Shire ac­qui­si­tion. Most pa­tients get eye drops for the con­di­tion.

Tiny Oys­ter Point’s plan in­volves tak­ing a most­ly pos­i­tive Phase IIb study — where their drug, vareni­cline, a for­mu­la­tion of Chan­tix, man­aged to get the tear ducts to flow in a sta­tis­ti­cal­ly sig­nif­i­cant man­ner — and prov­ing it works in a sec­ond reg­is­tra­tional, con­fir­ma­to­ry sto­ry. The FDA, ac­cord­ing to the S-1, has signed off on us­ing the Phase IIb as their first or 2 re­quired piv­otals. And that study, they main­tain, is the first ever to show sta­tis­ti­cal sig­nif­i­cance against signs and symp­toms of dry eye dis­ease.

If they do get an ap­proval, they be­lieve that they can hire a sales force of 150 to 200 to get it out on the mar­ket. The biotech cur­rent­ly has a staff of 18, putting it in the lean­est and mean­est cat­e­go­ry of late-stage com­pa­nies.

Whether they mean that or plan to use it as a clas­sic bluff in prepa­ra­tion for a pos­si­ble buy­out deal is the big ques­tion. Star­tups tra­di­tion­al­ly have faced a moun­tain­ous chal­lenge sell­ing the drugs they man­age to get ap­proved, and that’s even true in spe­cial­ty can­cer mar­kets. For ma­jor mar­kets like dry eye dis­ease, the climb against Big Phar­ma and gener­ics is dis­tinct­ly more chal­leng­ing. 

The biotech has burned through $53 mil­lion and had $84 mil­lion banked at the end of H1.

New En­ter­prise As­so­ci­ates and Ver­sant are the 2 big in­vestors, with 32% of eq­ui­ty each. Jef­frey Nau, the CEO, has 2.1% of the shares go­ing in.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.

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).

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 87,700+ biopharma pros reading Endpoints daily — and it's free.