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LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> BrainStorm puts together plan for new Phase 3b ALS trial after pulling previous filing: Having exhausted nearly all other avenues to get its ALS cell therapy approved, BrainStorm Cell Therapeutics is heaving a Hail Mary pass. The biotech announced Tuesday that it is moving forward with a Phase 3b study for the treatment, called NurOwn, and that it has agreed to a Special Protocol Assessment, or SPA, with the FDA. BrainStorm said the study is expected to launch sometime this year, but didn’t provide any more specifics in a press release or during an investor call Tuesday morning. SPAs are used infrequently by biopharma companies seeking FDA approval for their prescription drugs. Typically when a drugmaker develops a new therapy, they use either a new drug application (NDA) or biologics license application (BLA), depending on the composition of their treatment. But short of shutting down the company and halting NurOwn development, BrainStorm has no other options after withdrawing its BLA last year following a negative adcomm hearing. An SPA also does not guarantee smooth development process or future approval. According to FDA guidance, SPAs are to be used to support development “in the case where human efficacy studies are not ethical or feasible, of animal and any associated clinical trials which, in combination, are intended to form the primary basis of an effectiveness claim.” The guidance also indicates that the existence of an SPA does not guarantee the FDA will accept an NDA or BLA, or that trial results will support approval. The FDA may also not agree with every specific study protocol detail. Per BrainStorm, the new study will enroll ALS patients in the early stages of their disease and examine them over 24 weeks. The primary efficacy measurement will be the change in a functional rating scale specific to ALS (ALSFRS-R) compared to a placebo. After the initial 24-week period, all patients will be eligible to enroll in another 24-week open-label extension. BrainStorm shares $BCLI, which closed Monday at 73 cents per share, were down about 10% in early trading Tuesday morning. A rocky road NurOwn’s development has been anything but typical. BrainStorm withdrew its BLA last October after a long and often contentious process. NurOwn originally failed to achieve statistical significance in an earlier Phase 3 trial, reading out negatively in 2020. But after the FDA told BrainStorm its BLA didn’t meet the threshold for approval and the agency offered a rare public rebuke in 2021, the company filed the submission anyway after issuing a “correction” to its dataset. That resulted in the FDA declining to consider its application, as it issued a “refusal to file” letter in November 2022. BrainStorm continued to push ahead, taking… #lucidquest #genetherapy #celltherapy

I am participating LSN Webinar to share my two cents on growing interest of JP startups/biotechs on Go-to-market strategy abroad and what improvement is needed to achieve it.

Gene&Cell Therapy >> BrainStorm puts together plan for new Phase 3b ALS trial after pulling previous filing: Having exhausted nearly all other avenues to get its ALS cell therapy approved, BrainStorm Cell Therapeutics is heaving a Hail Mary pass. The biotech announced Tuesday that it is moving forward with a Phase 3b study for the treatment, called NurOwn, and that it has agreed to a Special Protocol Assessment, or SPA, with the FDA. BrainStorm said the study is expected to launch sometime this year, but didn’t provide any more specifics in a press release or during an investor call Tuesday morning. SPAs are used infrequently by biopharma companies seeking FDA approval for their prescription drugs. Typically when a drugmaker develops a new therapy, they use either a new drug application (NDA) or biologics license application (BLA), depending on the composition of their treatment. But short of shutting down the company and halting NurOwn development, BrainStorm has no other options after withdrawing its BLA last year following a negative adcomm hearing. An SPA also does not guarantee smooth development process or future approval. According to FDA guidance, SPAs are to be used to support development “in the case where human efficacy studies are not ethical or feasible, of animal and any associated clinical trials which, in combination, are intended to form the primary basis of an effectiveness claim.” The guidance also indicates that the existence of an SPA does not guarantee the FDA will accept an NDA or BLA, or that trial results will support approval. The FDA may also not agree with every specific study protocol detail. Per BrainStorm, the new study will enroll ALS patients in the early stages of their disease and examine them over 24 weeks. The primary efficacy measurement will be the change in a functional rating scale specific to ALS (ALSFRS-R) compared to a placebo. After the initial 24-week period, all patients will be eligible to enroll in another 24-week open-label extension. BrainStorm shares $BCLI, which closed Monday at 73 cents per share, were down about 10% in early trading Tuesday morning. A rocky road NurOwn’s development has been anything but typical. BrainStorm withdrew its BLA last October after a long and often contentious process. NurOwn originally failed to achieve statistical significance in an earlier Phase 3 trial, reading out negatively in 2020. But after the FDA told BrainStorm its BLA didn’t meet the threshold for approval and the agency offered a rare public rebuke in 2021, the company filed the submission anyway after issuing a “correction” to its dataset. That resulted in the FDA declining to consider its application, as it issued a “refusal to file” letter in November 2022. BrainStorm continued to push ahead, taking advantage of a little-known procedure called “File Over… #lucidquest #genetherapy #celltherapy

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LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Gamida to go private; Stoke aims to raise $75M: Plus, news about a TUDCA study, PDS Biotechnology and Allarity Therapeutics: Gamida Cell to go private: The commercial-stage cell therapy biotech will no longer trade on the Nasdaq after Highbridge Capital Management, its principal lender, restructures the company. The penny stock $GMDA was down 84% in Wednesday trading. Gamida secured FDA approval for its cell therapy Omisirge in April. Then, in January, it said it was “actively pursuing” alternative routes to keep going. — Kyle LaHucik Stoke eyes $75M offering: The Boston-area biotech plans to sell about $75 million worth of shares after its stock price popped in response to clinical data on a genetic epilepsy treatment candidate. — Kyle LaHucik TUDCA study reports Ph3 fail for experimental ALS supplement: The study, which was conducted by a consortium of European hospitals, did not show a difference between the supplement tauroursodeoxycholic acid, or TUDCA, and placebo on a primary endpoint that measures patient function (ALSFRS-R). It also did not meet any secondary endpoints like survival or changes in biomarkers. Investors were closely watching the study ahead of Amylyx’s Phase 3 readout, which came in negative earlier this month. Amylyx’s drug Relyvrio is made up of a higher concentration of taurursodiol, the active chemical in TUDCA. — Max Gelman PDS Biotechnology’s stock drops after it changed pivotal study plan: The biotech is no longer pursuing a Phase 3 study of its experimental drug PDS01ADC in combination with Merck’s Keytruda in HPV-positive head and neck cancer. It’s instead discussing a triple combination pivotal trial of PDS01ADC, Keytruda and a T cell activating immunotherapy that the company hopes to start later this year. PDS shares $PDSB were down 12% on Wednesday morning. — Lei Lei Wu Allarity Therapeutics pivots: The penny stock biotech is stopping work on its tyrosine kinase inhibitor dovitinib and the chemotherapy Ixempra, citing the need to cut costs. It is instead focusing on advancing stenoparib, a PARP and Tankyrase inhibitor currently in a Phase 2 monotherapy trial in advanced ovarian cancer. — Lei Lei Wu #lucidquest #genetherapy #celltherapy

Don’t miss the upcoming panel discussion on LBA and LC/MS/MS hybrid assays hosted by Bioanalysis Zone. Shashank Gorityala, Associate Director of LC/MS Operations, and a panel of experts will explore whether hybridization assays are the new norm and discuss the pros and cons, including equipment requirements, digestion considerations, multiplexing potential, and future trends. #LCMS #HybridAssays #biotherapeutics #InnovationInScience #bioanalysis #massspectrometry #drugdevelopment https://lnkd.in/gmhUzkzZ

A double-blind, placebo-controlled, proof-of-concept study with LD IL-2 in patients with AD is ongoing (and still blinded) at the Houston Methodist Hospital, with top-line results anticipated in mid-2024. While the primary objectives in this study are the customary safety endpoints, a secondary endpoint to be measured is the change in Treg percentage out of the total number of CD4 cells. This study is intended to serve as a proof-of-concept to support the development of COYA 302 as a treatment for AD, including a potential partner strategy. Read more in our latest letter to stockholders: https://bit.ly/4bIudwG #Science #NeurodegenerativeDiseases #Biotech

Gene&Cell Therapy >> Gamida to go private; Stoke aims to raise $75M: Plus, news about a TUDCA study, PDS Biotechnology and Allarity Therapeutics: Gamida Cell to go private: The commercial-stage cell therapy biotech will no longer trade on the Nasdaq after Highbridge Capital Management, its principal lender, restructures the company. The penny stock $GMDA was down 84% in Wednesday trading. Gamida secured FDA approval for its cell therapy Omisirge in April. Then, in January, it said it was “actively pursuing” alternative routes to keep going. — Kyle LaHucik Stoke eyes $75M offering: The Boston-area biotech plans to sell about $75 million worth of shares after its stock price popped in response to clinical data on a genetic epilepsy treatment candidate. — Kyle LaHucik TUDCA study reports Ph3 fail for experimental ALS supplement: The study, which was conducted by a consortium of European hospitals, did not show a difference between the supplement tauroursodeoxycholic acid, or TUDCA, and placebo on a primary endpoint that measures patient function (ALSFRS-R). It also did not meet any secondary endpoints like survival or changes in biomarkers. Investors were closely watching the study ahead of Amylyx’s Phase 3 readout, which came in negative earlier this month. Amylyx’s drug Relyvrio is made up of a higher concentration of taurursodiol, the active chemical in TUDCA. — Max Gelman PDS Biotechnology’s stock drops after it changed pivotal study plan: The biotech is no longer pursuing a Phase 3 study of its experimental drug PDS01ADC in combination with Merck’s Keytruda in HPV-positive head and neck cancer. It’s instead discussing a triple combination pivotal trial of PDS01ADC, Keytruda and a T cell activating immunotherapy that the company hopes to start later this year. PDS shares $PDSB were down 12% on Wednesday morning. — Lei Lei Wu Allarity Therapeutics pivots: The penny stock biotech is stopping work on its tyrosine kinase inhibitor dovitinib and the chemotherapy Ixempra, citing the need to cut costs. It is instead focusing on advancing stenoparib, a PARP and Tankyrase inhibitor currently in a Phase 2 monotherapy trial in advanced ovarian cancer. — Lei Lei Wu #lucidquest #genetherapy #celltherapy

Adnexi invites you to listen to this Global Genes RareCAST episode that hosts Sandra Shpilberg, our CEO and Co-founder to discuss the role of KOLs, DOLs and influence mapping in drug development and launch strategies. Listen here: https://buff.ly/3NTVJwN #biopharma #rarediseases #kol #dol #adnexi

Adnexi invites you to listen to this Global Genes RareCAST episode that hosts Sandra Shpilberg, our CEO and Co-founder to discuss the role of KOLs, DOLs and influence mapping in drug development and launch strategies. Listen here: https://buff.ly/3NTVJwN #biopharma #rarediseases #kol #dol #adnexi