News brief­ing: Gy­ro­scope and Ta­laris set IPO terms; FDA grants ex­pand­ed nod to Chiesi's sick­le cell drug

In a week where two biotech IPOs were al­ready ex­pect­ed, an­oth­er two prospec­tive en­trants have set their de­but terms ahead of their launch lat­er this week.

Gy­ro­scope Ther­a­peu­tics and Ta­laris Ther­a­peu­tics each set IPO terms Mon­day morn­ing. Gy­ro­scope, based out of the UK, is plan­ning to of­fer 6.8 mil­lion ADSs at a price range of $20 to $22, while Ta­laris ex­pects to of­fer 8.8 mil­lion shares be­tween $16 and $18 each.

Should every­thing go ac­cord­ing to plan, Gy­ro­scope will pull in about $142 mil­lion while Ta­laris will raise rough­ly $150 mil­lion.

Both of those fig­ures are high­er than their ini­tial S-1 es­ti­mates of $100 mil­lion each, though that fig­ure has be­come a place­hold­er with the flood of biotechs en­ter­ing the mar­ket over the last 16 months or so.

Last year’s pan­dem­ic IPO boom hasn’t lost any steam in 2021, with 50 com­pa­nies hav­ing filed or priced their IPOs so far this year. Gy­ro­scope and Ta­laris are ex­pect­ed to join Val­ne­va and Aneb­u­lo in their de­buts lat­er this week, bring­ing the to­tal com­bined IPO raise close to $7 bil­lion, per the End­points News tal­ly. That fig­ure is well on pace to eclipse the amount raised in 2020 of $16 bil­lion-plus, which set a record for the biotech in­dus­try. — Max Gel­man

FDA grants sick­le cell ap­proval to Chiesi, ex­pand­ing use

Chiesi Glob­al Rare Dis­eases re­ceived FDA ap­proval for its sick­le cell dis­ease drug Fer­riprox, the com­pa­ny an­nounced Sat­ur­day.

The ap­proval ex­pands the use of the drug for pa­tients with sick­le cell or oth­er ane­mias, as well as pa­tients with tha­lassemia re­gard­less of pri­or iron chela­tion ex­po­sure. Fer­riprox is an oral­ly ac­tive agent that has proved ef­fec­tive in re­duc­ing iron con­cen­tra­tion by re­mov­ing tox­ic iron from or­gan tis­sues and ex­tra­cel­lu­lar flu­ids. The drug is tak­en as a 1,000 mg, twice-dai­ly tablet.

“We be­lieve that de­liv­er­ing an iron chela­tion ther­a­py that has no dosage ad­just­ment re­quired for pa­tients with mild to se­vere re­nal im­pair­ment may ad­dress a sig­nif­i­cant un­met need in SCD,” CEO Gi­a­co­mo Chiesi said in a state­ment.

The good news comes just days af­ter Chiesi and part­ner Pro­tali Bio­ther­a­peu­tics re­ceived a com­plete re­sponse let­ter Wednes­day for their Fab­ry dis­ease hope­ful. — Josh Sul­li­van

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).