Thomas Cannell, Sesen

FDA re­jects Sesen Bio's blad­der can­cer pitch, send­ing shares spi­ral­ing back to pen­ny stock ter­ri­to­ry

Sesen Bio re­ceived some bad news Fri­day af­ter­noon that it said was “un­ex­pect­ed,” briefly slash­ing shares by more than 80% and be­low $1 for the first time in near­ly a year.

The biotech re­port­ed it re­ceived a CRL from the FDA re­gard­ing its blad­der can­cer can­di­date Vicineum, an an­ti­body-drug con­ju­gate ac­quired in the com­pa­ny’s buy­out of Toron­to-based Viven­tia back in 2016. Trad­ing was halt­ed for about an hour on Fri­day, with Sesen en­ter­ing the halt at about $6 per share and emerg­ing bat­tered with a $0.86 stock price.

Ahead of the clos­ing bell, Sesen shares re­cov­ered mod­est gains to close at $2.11, down 57% from Fri­day’s open. Ex­pec­ta­tions had swirled among in­vestors lead­ing up to the PDU­FA date, sched­uled for next Wednes­day, as Sesen’s stock price rose about 43% from Thurs­day’s open to Fri­day’s halt.

“We are deeply dis­ap­point­ed by this un­ex­pect­ed re­sult, and it is an un­for­tu­nate day for pa­tients suf­fer­ing from BCG-un­re­spon­sive NMIBC,” CEO Thomas Can­nell said in a state­ment. “We re­main ded­i­cat­ed to our mis­sion to save and im­prove the lives of pa­tients by bring­ing new treat­ment op­tions to pa­tients, and we in­tend to work close­ly with the FDA to un­der­stand next steps.”

Sesen de­scribed the rea­son for the CRL as a com­bi­na­tion of man­u­fac­tur­ing is­sues and re­quests for more da­ta and sta­tis­ti­cal analy­ses. The Cam­bridge, MA-based com­pa­ny plans to meet with the FDA “as soon as pos­si­ble” and said reg­u­la­tors pro­vid­ed rec­om­men­da­tions for how to boost the da­ta pack­age.

Vicineum us­es a re­com­bi­nant fu­sion pro­tein at­tached to a ge­net­i­cal­ly en­gi­neered pep­tide, with re­searchers aim­ing to tar­get spe­cif­ic anti­gens on the sur­face of tu­mor cells to treat can­cers. The FDA had giv­en Sesen pri­or­i­ty re­view for the pro­gram back in Feb­ru­ary, with Can­nell say­ing at the time Sesen un­der­stood the agency’s guid­ance “very clear­ly” and “found the re­view process to be col­lab­o­ra­tive and en­gag­ing.”

Sesen had sub­mit­ted da­ta from an open-la­bel Phase III study that en­rolled 133 pa­tients with high-risk, BCG-un­re­spon­sive non-mus­cle in­va­sive blad­der can­cer.

The pa­tients had pre­vi­ous­ly been on BCG im­munother­a­py, and the most re­cent da­ta came from 93 in­di­vid­u­als whose can­cer had not spread from the blad­der in­to the mus­cle or oth­er tis­sue. Sesen re­port­ed that 39% of those pa­tients achieved a com­plete re­sponse af­ter three months, with fol­low-ups at six, nine and 12 months see­ing CRs of 28%, 21% and 15%, re­spec­tive­ly.

Vicineum had giv­en Sesen new life af­ter a for­mer lead pro­gram failed twice and was sub­se­quent­ly li­censed out to Roche. But Fri­day’s news sent Sesen back in­to deep pen­ny-stock ter­ri­to­ry, and it re­mains to be seen how or whether the biotech plans to re-work its datasets and CMC is­sues to the FDA’s lik­ing.

In an­tic­i­pa­tion of a po­ten­tial ap­proval, Sesen had been launch­ing sig­nif­i­cant com­mer­cial ef­forts over the last sev­er­al months, ef­forts that ramped up in re­cent weeks and now may need to be paused. The biotech re­port­ed it had com­plet­ed hir­ing about 60 new sales reps at the end of Ju­ly — rep­re­sent­ing 97% of its sales force — and hired a new as­sis­tant gen­er­al coun­sel and chief com­pli­ance of­fi­cer as the cal­en­dar turned to Au­gust. As re­cent­ly as Ju­ly 14, Sesen paint­ed a pos­i­tive pic­ture fol­low­ing a meet­ing with the FDA.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Flori­da man con­vict­ed of fal­si­fy­ing clin­i­cal tri­al re­sults sen­tenced to over 2 years in prison

A Florida man who falsified medical records in connection to clinical trials was sentenced to 30 months in prison in federal court Thursday.

Daniel Tejeda, 35, of Clewiston, was also ordered to pay $2.1 million in restitution. Tejeda was a project manager and study manager for the CRO Tellus Clinical Research, and made it appear that subjects were participating in trials when they weren’t. Two other research workers from Florida were sentenced in the same case in August for 46 and 30 months, respectively.

Troy Wilson, Kura Oncology CEO

FDA lifts par­tial hold on Ku­ra's Phase Ib AML pro­gram as biotech re­dou­bles mit­i­ga­tion ef­forts

Kura Oncology is clear to resume studies for its early-stage leukemia program after the FDA lifted a clinical hold Thursday afternoon.

Regulators had placed the hold on a Phase Ib study of KO-539, an experimental oral treatment for some genetic subsets of acute myeloid leukemia last November after a patient died while taking the drug. Kura expects to begin enrolling patients again imminently, CEO Troy Wilson told Endpoints News.