Gray­bug seeks al­ter­na­tive op­tions for eye drugs; RA-backed epilep­sy out­fit se­cures $40M Se­ries C

Gray­bug Vi­sion is in search of a new home about 21 months af­ter land­ing on the Nas­daq. The oc­u­lar dis­ease-fo­cused biotech has seen its stock $GRAY plum­met amid the biotech bear mar­ket, nose­div­ing from a Sep­tem­ber 2020 list­ing at $16 to $1 ter­ri­to­ry the past few months.

The Bal­ti­more com­pa­ny’s stock is re­bound­ing Tues­day morn­ing, though, thanks to an an­nounce­ment be­fore the open­ing bell that the gene ther­a­py de­vel­op­er would seek a strate­gic al­ter­na­tive thanks to the help of fi­nan­cial ad­vi­sor Piper San­dler.

With $55.3 mil­lion in the bank as of March 31, Gray­bug is look­ing to emerge from the doom and gloom via an “ac­qui­si­tion, com­pa­ny sale, merg­er, di­vesti­ture of as­sets, pri­vate place­ment of eq­ui­ty se­cu­ri­ties, or oth­er strate­gic trans­ac­tions,” the com­pa­ny said in a state­ment. But, like all such state­ments, Gray­bug cau­tioned that such trans­ac­tions might nev­er hap­pen.

Gray­bug has on­ly made it in­to the clin­ic with one re­com­bi­nant AAV-based gene ther­a­py so far. That gene ther­a­py, GB-102, went through Phase II stud­ies in wet age-re­lat­ed mac­u­lar de­gen­er­a­tion. An­oth­er Phase II tri­al was slat­ed to be­gin in the fourth quar­ter of this year, Gray­bug said in a May up­date.

Gray­bug’s “vi­brant vi­sion” mis­sion is al­so an­gled to­ward re­tini­tis pig­men­tosa (GB-601), ge­o­graph­ic at­ro­phy (GB-701), glau­co­ma (GB-401) and corneal cloud­ing (GB-501). That last as­set was re­cent­ly picked up via an ac­qui­si­tion of Rain­Bio, a North Car­oli­na start­up, and was sched­uled for a sec­ond-quar­ter 2023 tri­al start, the com­pa­ny said in May. GB-401 was al­so an­tic­i­pat­ed to en­ter hu­man stud­ies ear­ly next year. — Kyle LaHu­cik

RA-backed Col­orado epilep­sy biotech scores Se­ries C

Cere­bral Ther­a­peu­tics walked away from its newest fi­nanc­ing with $40 mil­lion in cash, thanks to a round led by Lynx1 Cap­i­tal Man­age­ment. The com­pa­ny said in a state­ment that it plans to use the mon­ey to com­plete its Phase II tri­al of an im­plant­ed, re­fill­able catheter and pump sys­tem de­signed to de­liv­er a con­tin­u­ous dose of CT-010. The drug is a re­for­mu­lat­ed ver­sion of val­proic acid, first FDA-ap­proved in the 1970s to treat ab­sence seizures.

The sys­tem aims to de­liv­er the drug in­trac­ere­broven­tric­u­lar­ly, a more in­va­sive in­jec­tion tech­nique that gets sub­stances di­rect­ly in­to the cere­brospinal flu­id — by­pass­ing the blood-brain bar­ri­er in the process and lim­it­ing the body’s ex­po­sure to a drug. Be­yond that, the funds from the Se­ries C will “sup­port Phase 3 readi­ness,” ac­cord­ing to a re­lease.

The planned in­di­ca­tion is for un­con­trolled seizures in pa­tients with med­ical­ly re­frac­to­ry epilep­sy.

Oth­er in­vestors that tagged on­to the round in­clude ex­ist­ing back­ers RA Cap­i­tal Man­age­ment, Per­cep­tive Ad­vi­sors, Vi­vo Cap­i­tal LLC, and Gran­ite Point Cap­i­tal Man­age­ment LP. RA had al­so backed the com­pa­ny’s Se­ries B round in ear­ly 2020, where the biotech hauled $35 mil­lion to fund a Phase IIb tri­al for its drug/de­vice com­bo sys­tem. — Paul Schloess­er

Palleon Phar­ma­ceu­ti­cals and Hen­lius form a col­lab­o­ra­tion  

Mass­a­chu­setts-based biotech Palleon Phar­ma­ceu­ti­cals has an­nounced a strate­gic col­lab­o­ra­tion with Shang­hai’s Hen­lius Biotech.

The part­ners will look to de­vel­op and com­mer­cial­ize two bi­func­tion­al sial­i­dase pro­grams from Palleon’s im­muno-on­col­o­gy plat­form, called EA­GLE, or en­zyme-an­ti­body gly­co-lig­and edit­ing. This deal will see Hen­lius de­vel­op­ing Palleon’s bi­func­tion­al HER2-Sial­i­dase now in pre­clin­i­cal de­vel­op­ment and a sec­ond bi­func­tion­al sial­i­dase to be de­vel­oped with a pro­pri­etary tar­get pro­vid­ed by Hen­lius.

“We’re very pleased to part­ner with Hen­lius to de­vel­op and ex­pand Palleon’s pipeline to re­al­ize the po­ten­tial of bi­func­tion­al sial­i­das­es as a treat­ment op­tion for a wide range of can­cer types,” said Jim Brod­er­ick, CEO and founder of Palleon.

Un­der the terms of the agree­ment, Palleon will per­form the re­search with the par­ties and then share pre­clin­i­cal and glob­al clin­i­cal de­vel­op­ment re­spon­si­bil­i­ties and costs for the Bi­func­tion­al HER2-Sial­i­dase and a sec­ond bi­func­tion­al sial­i­dase.

Hen­lius has an ex­clu­sive li­cense to the two in­ves­ti­ga­tion­al ther­a­pies in Chi­na, Hong Kong, Macau and Tai­wan, while Palleon re­tains all oth­er glob­al rights. The sec­ond prod­uct will re­ceive a roy­al­ty-bear­ing ex­clu­sive li­cense to Hen­lius’ an­ti­body tech­nol­o­gy out­side Chi­na.

Palleon re­ceived an up­front pay­ment and is fur­ther el­i­gi­ble to re­ceive up to $196.5 mil­lion in cer­tain pre­de­ter­mined R&D and com­mer­cial mile­stones, in ad­di­tion to roy­al­ties up­on Hen­lius’ com­mer­cial­iza­tion in Chi­na.

Palleon, which is co-found­ed by the renowned chemist Car­olyn Bertozzi, re­ceived a $100 mil­lion Se­ries B in 2020 and has been build­ing out its C-Suite with in­dus­try ex­perts. Hen­lius has al­so been busy re­cent­ly as the com­pa­ny net­ted a deal with Organon, which will pay $73 mil­lion up­front, plus up to $30 mil­lion in 2022 mile­stone pay­ments, for two well-known brand copies that are in de­vel­op­ment. — Tyler Patchen

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

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Clo­vis says it needs mon­ey af­ter re­verse split vot­ed down; Cin­Cor touts PhII da­ta for hy­per­ten­sion drug

Clovis Oncology needs a serious leg up after posting a $71.3 million net loss in Q2.

According to the company’s Q2 report, based on its current cash, cash equivalents and liquidity the company will need to raise additional capital in the near term to continue going on beyond February of next year.

A proposed reverse stock split of Clovis’ common stock, which would have freed up more capital, was not approved at its annual meeting of stockholders. The company said in the report it is looking at other sources of funding such as strategic partnerships or licensing arrangements for one or more of its products or candidates.