Bel­licum Phar­ma­ceu­ti­cals is halt­ing the Phase I/II tri­als for the on­ly two can­di­dates in its pipeline af­ter re­port­ing a se­ri­ous ad­verse event — and is now eval­u­at­ing “strate­gic al­ter­na­tives” for the com­pa­ny af­ter a rocky few years.

The com­pa­ny an­nounced Tues­day that the on­go­ing tri­als for its two Go­CAR-T cell can­di­dates in com­bi­na­tion with rim­iducid in pre-treat­ed can­cer pa­tients, BPX-601 and BPX-603, are com­ing to a halt. This comes af­ter a pa­tient on BPX-601 ex­pe­ri­enced se­ri­ous side ef­fects, in­clud­ing grade 4 cy­tokine re­lease syn­drome. This was the sec­ond dose-lim­it­ing tox­i­c­i­ty in the dose-es­ca­la­tion co­hort.

Bel­licum $BLCM shares dropped rough­ly 40% in pre-mar­ket trad­ing Wednes­day.

The BPX-601 tri­al was study­ing the safe­ty and pre­lim­i­nary ef­fi­ca­cy of the can­di­date in pa­tients with metasta­t­ic cas­tra­tion-re­sis­tant prostate can­cer. BPX-603 tar­get­ed HER2-pos­i­tive sol­id tu­mors.

“While clin­i­cal­ly mean­ing­ful ef­fi­ca­cy has been ob­served — in­clud­ing 5 of 9 mCR­PC pa­tients treat­ed achiev­ing PSA50 re­sponse, 4 of whom achieved PSA90 re­sponse — the Com­pa­ny be­lieves it does not have the nec­es­sary re­sources to op­ti­mize ei­ther the clin­i­cal dose and sched­ule of BPX-601 cells and the ac­ti­vat­ing agent rim­iducid, or the de­sign of the BPX-601 cell con­struct to achieve a fa­vor­able risk/ben­e­fit pro­file,” the press re­lease reads.

Rick Fair

There seemed to be hope in Feb­ru­ary when pres­i­dent and CEO Rick Fair said there were “en­cour­ag­ing” ear­ly da­ta from BPX-601. The com­pa­ny al­so re­port­ed oth­er ad­verse events at the time, how­ev­er, such as myelo­sup­pres­sion, grade 3 cy­tokine re­lease syn­drome and grade 4 im­mune ef­fec­tor cell neu­ro­tox­i­c­i­ty syn­drome.

It’s been a long road for Bel­licum, which laid off 79% of its staff in 2020 af­ter it re­port­ed dis­ap­point­ing re­sults for BPX-601 in pan­cre­at­ic can­cer. It al­so stopped dis­cov­ery re­search and new prod­uct de­vel­op­ment that year. At the time, the com­pa­ny said it would fo­cus on BPX-601, BPX-603 and a third can­di­date that’s no longer in de­vel­op­ment.

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.