Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Deci­bel Ther­a­peu­tics has large­ly piv­ot­ed to­ward gene ther­a­pies for the in­ner ear, its lead clin­i­cal can­di­date sim­ply aims to pro­tect can­cer pa­tients from chemother­a­py-in­duced hear­ing loss. On Tues­day, the biotech pre­sent­ed its first ef­fi­ca­cy da­ta for the pro­gram, and ex­ecs like what they see.

Deci­bel re­port­ed in­ter­im re­sults from a Phase Ib study show­ing the ex­per­i­men­tal drug, dubbed DB-020, large­ly pro­tect­ed a small group of pa­tients from los­ing their hear­ing. Re­searchers used a par­tic­u­lar­ly unique study de­sign, ad­min­is­ter­ing the com­pound in one of each pa­tients’ ears be­fore they re­ceived cis­platin chemother­a­py and place­bo in the oth­er.

The biotech had 17 evalu­able pa­tients as of the Feb. 4 cut­off date who had nev­er be­fore re­ceived cis­platin. Fol­low­ing the most re­cent chemo cy­cle, 15 of the 17 pa­tients ex­pe­ri­enced on av­er­age a 30 deci­bel-loss from base­line in the place­bo ear. Of those 15, eight pa­tients did not lose their hear­ing in the DB-020-treat­ed ear, and an­oth­er five were par­tial­ly pro­tect­ed from hear­ing loss.

All ears treat­ed with DB-020 lost ap­prox­i­mate­ly eight deci­bels on av­er­age from base­line, Deci­bel said.

DB-020 is a for­mu­la­tion of sodi­um thio­sul­fate in­ject­ed through the eardrum be­fore pa­tients re­ceive cis­platin-based chemother­a­py. The pro­ce­dure can be done in an ENT’s of­fice with­out the need for in­va­sive meth­ods, the com­pa­ny touts.

A Phase Ia study in healthy vol­un­teers had been com­plet­ed in 2019 and the com­pa­ny sub­se­quent­ly moved for­ward with the Phase Ib tri­al. The re­sults had been de­layed due to the Covid-19 pan­dem­ic, Deci­bel said in a press re­lease ear­ly last year. Af­ter kick­ing off the Phase Ib tri­al in Feb­ru­ary 2020, the com­pa­ny an­tic­i­pat­ed col­lect­ing fi­nal pri­ma­ry out­come mea­sures in April 2021 and com­plet­ing the study that Oc­to­ber. The biotech re­vised those time­lines dur­ing the pan­dem­ic, with the tar­get dates now De­cem­ber 2022 and De­cem­ber 2023, ac­cord­ing to changes to the tri­al’s reg­istry on clin­i­cal­tri­als.gov.

Deci­bel’s gene ther­a­py pro­grams still re­main in the pre­clin­i­cal phase, but the com­pa­ny is hop­ing to have an IND for its first such can­di­date sub­mit­ted by the end of the year. The fo­cus on gene ther­a­pies for re­gen­er­a­tion and bal­ance came af­ter a piv­ot away from ear-re­lat­ed con­di­tions such as tin­ni­tus and hear­ing loss a few years ago. Deci­bel is part­nered with Re­gen­eron on some of the pro­grams, and se­cured an ex­ten­sion to its re­search col­lab­o­ra­tion with the bio­phar­ma last No­vem­ber.

The shift was enough to gar­ner a Se­ries D in No­vem­ber 2020 and a quick jump to Nas­daq a few months lat­er. Like many com­pa­nies, Deci­bel has seen its stock price $DBTX sink in this year’s bear mar­ket, as it’s down 55% since the start of the year as of Mon­day’s close.

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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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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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.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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