Notizia - Recent Pharma, Healthcare and Biotech Happenings
Nov 04, 2025
Table of Contents
Intensity Therapeutics, Inc. (Nasdaq: INTS) announced the publication of its Phase 1/2 clinical results for INT230-6. The comprehensive study evaluated the intratumoral therapy’s safety, efficacy, and mechanism of action across patients with advanced, metastatic, or refractory solid tumors.
In heavily pretreated patients with over 20 different cancer types who had progressed after multiple prior therapy lines, INT230-6 demonstrated impressive outcomes. The therapy achieved a disease control rate of 75% and a median overall survival of 11.9 months, substantially outperforming the historical benchmark of 4 to 7 months typically reported in comparable Phase 1/2 studies. In a metastatic sarcoma subset, median overall survival extended to an exceptional 21.3 months.
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“The disease control rates and median survival observed in our study compare remarkably favorably to those historically seen for such a diverse patient population,” said Jacob S. Thomas, M.D., Assistant Professor of Clinical Medicine at the University of Southern California and lead author of the study. “The pharmacokinetic data revealed that greater than 95% of the active cytotoxic agents remained concentrated within the injected tumors, with minimal systemic leakage.”
A particularly notable finding emerged in an exploratory analysis comparing tumor burden coverage. Patients treated with INT230-6 covering greater than 40% of their total tumor burden achieved an 83.3% disease control rate and 18.7-month median overall survival, dramatically contrasting with the 50% disease control rate and 3.1-month survival observed in patients treated with less than 40% tumor coverage. Remarkably, approximately 20% of patients in the higher-coverage group demonstrated abscopal effects, with uninjected tumors shrinking following localized treatment.
“These abscopal effects and the substantial increase in activated T-cells infiltrating the tumor microenvironment highlight the potential of INT230-6 to drive both local and systemic anti-cancer activity,” said Anthony El-Khoueiry, M.D., senior author and Associate Director for Clinical Research at USC Norris Comprehensive Cancer Center. The therapy demonstrated an excellent safety profile with no dose-limiting toxicities reported among 64 monotherapy patients, with only seven patients experiencing Grade 3 adverse events and no Grade 4 or 5 treatment-related events.
Intensity has advanced INT230-6 into randomized controlled Phase 3 trials, including a dedicated soft tissue sarcoma study, informed by the robust data across multiple cancer types.
UCB announced that the U.S. Food and Drug Administration has approved KYGEVVI (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency (TK2d). This landmark approval addresses a devastating, ultra-rare, life-threatening genetic mitochondrial disease with no previously available therapeutic options beyond supportive care.
TK2d is characterized by progressive and severe muscle weakness (myopathy) and carries an extraordinarily high disease burden. Patients experiencing initial symptoms on or before the age of 12 years face a critical prognosis, with many progressing to premature death, often occurring within three years of symptom onset. The prevalence is estimated at 1.64 cases per 1,000,000 people.
The FDA approval is supported by robust safety and efficacy data from one Phase 2 clinical study, two retrospective chart review studies, and an expanded access program, collectively analyzing 82 unique patients. In the efficacy analysis using a matched control group of untreated patients, treatment with KYGEVVI reduced the overall risk of death by approximately 86% (95% confidence interval: 61%, 96%) from treatment initiation. The median treatment duration was 4 years, with patients receiving a median dose of 762 mg/kg/day.
“This approval represents a pivotal moment for the TK2d community who previously faced no FDA-approved therapeutic alternatives,” said Donatello Crocetta, Chief Medical Officer at UCB. “We extend heartfelt gratitude to the patients, families, advocates, and clinical trial teams who have partnered with us on this crucial journey.”
The most common adverse reactions observed during treatment included diarrhea, abdominal pain, vomiting, and elevated liver transaminases. KYGEVVI received multiple FDA designations, including Orphan Drug, Breakthrough Therapy, Priority Review, and Rare Pediatric Disease designations. With this approval, UCB received a Rare Pediatric Disease Priority Review Voucher redeemable for future regulatory submissions.
“I’ve studied mitochondrial diseases for over three decades and witnessed firsthand the profound impact TK2d has on patients and families. This approval marks a significant milestone in how we can support and manage this debilitating condition,” noted Dr. Michio Hirano, Professor of Neurology at Columbia University Irving Medical Center. UCB expects KYGEVVI to be commercially available in the United States in Q1 2026.
BrainXell Therapeutics, a San Diego-based biotechnology company, announced disclosure of significant preclinical scientific data for BXT-110, its lead investigational program in induced pluripotent stem cell (iPSC)-derived therapies for Parkinson’s disease.
BXT-110 represents an innovative regenerative approach for Parkinson’s disease, the second most common neurodegenerative disorder characterized by progressive loss of midbrain dopaminergic neurons. These essential neurons regulate dopamine production and control motor function, mood regulation, and cognitive processes. Restoring dopaminergic neurons has emerged as a significant therapeutic goal in Parkinson’s disease management.
The data presented highlight BrainXell’s progress in developing a robust, reproducible, and scalable approach for BXT-110, an autologous source of midbrain dopaminergic progenitor cells. Using both patient- and donor-derived iPSC lines, the team generated highly enriched cell populations with strong consistency across target markers. Notably, in vivo studies demonstrated significant graft survival and substantial functional recovery in a 6-OHDA rodent Parkinson’s disease model, with efficacy observed as early as 12 weeks and sustained for up to six months post-transplantation.
“These results underscore our capability to advance iPSC-derived therapies that can be transformative for patients living with neurodegenerative diseases,” said Katherine Vega Stultz, Chief Executive Officer of BrainXell Therapeutics. “Our data presentations reflect our commitment to rigorous science and deep expertise in iPSC biology and neuronal differentiation.”
The preclinical results establish BXT-110 as an efficacious, highly reproducible personalized cell-therapy candidate supporting its advancement as a potential disease-modifying treatment for Parkinson’s disease. BrainXell is advancing the program toward IND-enabling studies, building upon the foundation of research pioneered by Dr. Su-Chun Zhang, the company’s Advising Chief Scientific Officer.
CARsgen Therapeutics Holdings Limited, a company focused on developing innovative CAR T-cell therapies, announced clinical data for two allogeneic CAR-T product candidates. CT0596, a BCMA-targeting therapy for relapsed/refractory multiple myeloma, and CT1190B, a CD19/CD20-targeting therapy for relapsed/refractory non-Hodgkin’s lymphoma, both demonstrated initially favorable safety profiles and encouraging efficacy signals.
CT0596 in Relapsed/Refractory Multiple Myeloma
In an investigator-initiated trial evaluating CT0596, eight relapsed/refractory multiple myeloma patients received the BCMA-targeted CAR-T therapy developed on CARsgen’s proprietary THANK-u Plus™ platform. These patients represented a heavily pretreated population with a median of 4.5 prior therapy lines (range: 3-9), with five having received triple-class drugs and five with prior stem cell transplantation history. CAR-T cell doses escalated from 1.5×10⁸ to 4.5×10⁸ cells.
All eight patients achieved evaluable efficacy outcomes at a median follow-up of 2.56 months. Five patients achieved partial response or above, including three achieving complete or stringent complete response, one achieving partial response, and one achieving very good partial response. Notably, six of eight patients achieved minimal residual disease negativity at week four. One patient has maintained ongoing stringent complete response and minimal residual disease negativity for nearly six months. All eight patients showed CAR-T cell expansion, with no dose-limiting toxicities, treatment discontinuations, or deaths observed. Four patients experienced Grade 1 cytokine release syndrome, all resolving within 2-10 days.
CT1190B in Relapsed/Refractory Non-Hodgkin’s Lymphoma
Concurrent investigator-initiated trials evaluated CT1190B in 14 patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma, including follicular lymphoma, mantle cell lymphoma, and diffuse large B-cell lymphoma. At the recommended lymphodepletion dose, six evaluable patients achieved an overall response rate of 83.3%, comprising four complete responses and one partial response. Notably, all three follicular lymphoma patients achieved complete responses despite receiving multiple prior therapies, including immunochemotherapy, autologous stem cell transplantation, and advanced targeted therapies.
Safety findings revealed cytokine release syndrome, cytopenia, and infections as primary signals, with no immune effector cell-associated neurologic syndrome or graft-versus-host disease observed. Peak CAR-T cell expansion reached levels on the order of 10⁵ copies/µg genomic DNA in optimally dosed patients.
CARsgen anticipates submitting an Investigational New Drug application for CT0596 in the second half of 2025, advancing toward potential therapeutic availability for patients with urgent unmet needs in plasma cell malignancies and autoimmune diseases.
In an unprecedented industry move, Danish pharmaceutical giant Novo Nordisk launched an unsolicited ~$9 billion counteroffer to acquire obesity-focused clinical-stage biotech Metsera, directly challenging Pfizer’s previously announced $7.3 billion deal. The aggressive bid marks a dramatic escalation in competition for next-generation obesity treatments and signals strategic repositioning under Novo’s new leadership.
Pfizer’s initial agreement valued Metsera at $4.9 billion plus up to $2.4 billion in potential milestones. However, Novo Nordisk’s substantially higher valuation has triggered a landmark legal battle in the Delaware Court of Chancery, with Pfizer accusing the Copenhagen-based competitor of breach of contract and tortious interference while challenging the regulatory viability of Novo’s proposal based on antitrust and manufacturing structure concerns.
At the center of this high-stakes corporate confrontation lies Metsera’s innovative lead candidate, MET-097i, a first-in-class ultra-long-acting GLP-1 receptor agonist designed for potentially monthly dosing, a significant advancement over weekly injection regimens required by market leaders Novo’s WEGOVY and Eli Lilly’s ZEPBOUND. Recent Phase IIb data demonstrated remarkable efficacy in the VESPER-1 study, achieving a mean placebo-subtracted weight loss of 14.1% at 28 weeks at the highest dose (1.2 mg), with some patients losing as much as 26.5% of body weight.
“These results demonstrate MET-097i’s remarkable potency, requiring 5-10 times lower doses than current GLP-1 options while achieving comparable efficacy to tirzepatide,” explained the clinical findings. The drug’s tolerability profile proved equally compelling in the VESPER-3 trial, with only 13% increased nausea and 11% increased vomiting compared to placebo at the highest monthly dose, substantially lower than competing agents. Discontinuation rates due to adverse events reached only 2.9%, compared to up to 10% for other obesity medications.
Based on these positive results, Metsera is advancing toward Phase III trials in late 2025, with potential FDA approval projected for 2027 or 2028. Beyond MET-097i monotherapy, Metsera’s pipeline includes MET-233, an ultra-long-acting amylin analog potentially combinable as the industry’s first monthly multi-hormone therapy, plus MET-815, an oral prodrug formulation.
The competitive intensity of Novo vs Pfizer reflects extraordinary market growth dynamics. The anti-obesity medication market, valued at approximately $12 billion in 2024, is projected to reach $88 billion by 2034, representing a compound annual growth rate of 20.6%. Novo Nordisk’s OZEMPIC and WEGOVY, combined with Eli Lilly’s MOUNJARO and ZEPBOUND, have collectively generated $71 billion in US revenue since 2018.
For Pfizer, the potential loss of Metsera represents a significant setback following repeated clinical disappointments. The company terminated three separate in-house GLP-1 programs: Lotiglipron (discontinued late 2023 for elevated liver enzymes), Danuglipron (abandoned April 2025 for suspected drug-induced liver injury), and PF-06954522 (discontinued August 2025). Acquiring Metsera represented Pfizer’s critical opportunity to re-establish competitive positioning in this rapidly expanding therapeutic category.
Pfizer has secured early FDA antitrust clearance and requested a temporary restraining order to block Metsera from terminating their agreement pending the November 13, 2025, shareholder vote. The outcome will have far-reaching implications for M&A precedent in pharmaceutical consolidation among dominant market players.
Article in PDF
Nov 04, 2025
Table of Contents
Intensity Therapeutics, Inc. (Nasdaq: INTS) announced the publication of its Phase 1/2 clinical results for INT230-6. The comprehensive study evaluated the intratumoral therapy’s safety, efficacy, and mechanism of action across patients with advanced, metastatic, or refractory solid tumors.
In heavily pretreated patients with over 20 different cancer types who had progressed after multiple prior therapy lines, INT230-6 demonstrated impressive outcomes. The therapy achieved a disease control rate of 75% and a median overall survival of 11.9 months, substantially outperforming the historical benchmark of 4 to 7 months typically reported in comparable Phase 1/2 studies. In a metastatic sarcoma subset, median overall survival extended to an exceptional 21.3 months.
“The disease control rates and median survival observed in our study compare remarkably favorably to those historically seen for such a diverse patient population,” said Jacob S. Thomas, M.D., Assistant Professor of Clinical Medicine at the University of Southern California and lead author of the study. “The pharmacokinetic data revealed that greater than 95% of the active cytotoxic agents remained concentrated within the injected tumors, with minimal systemic leakage.”
A particularly notable finding emerged in an exploratory analysis comparing tumor burden coverage. Patients treated with INT230-6 covering greater than 40% of their total tumor burden achieved an 83.3% disease control rate and 18.7-month median overall survival, dramatically contrasting with the 50% disease control rate and 3.1-month survival observed in patients treated with less than 40% tumor coverage. Remarkably, approximately 20% of patients in the higher-coverage group demonstrated abscopal effects, with uninjected tumors shrinking following localized treatment.
“These abscopal effects and the substantial increase in activated T-cells infiltrating the tumor microenvironment highlight the potential of INT230-6 to drive both local and systemic anti-cancer activity,” said Anthony El-Khoueiry, M.D., senior author and Associate Director for Clinical Research at USC Norris Comprehensive Cancer Center. The therapy demonstrated an excellent safety profile with no dose-limiting toxicities reported among 64 monotherapy patients, with only seven patients experiencing Grade 3 adverse events and no Grade 4 or 5 treatment-related events.
Intensity has advanced INT230-6 into randomized controlled Phase 3 trials, including a dedicated soft tissue sarcoma study, informed by the robust data across multiple cancer types.
UCB announced that the U.S. Food and Drug Administration has approved KYGEVVI (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency (TK2d). This landmark approval addresses a devastating, ultra-rare, life-threatening genetic mitochondrial disease with no previously available therapeutic options beyond supportive care.
TK2d is characterized by progressive and severe muscle weakness (myopathy) and carries an extraordinarily high disease burden. Patients experiencing initial symptoms on or before the age of 12 years face a critical prognosis, with many progressing to premature death, often occurring within three years of symptom onset. The prevalence is estimated at 1.64 cases per 1,000,000 people.
The FDA approval is supported by robust safety and efficacy data from one Phase 2 clinical study, two retrospective chart review studies, and an expanded access program, collectively analyzing 82 unique patients. In the efficacy analysis using a matched control group of untreated patients, treatment with KYGEVVI reduced the overall risk of death by approximately 86% (95% confidence interval: 61%, 96%) from treatment initiation. The median treatment duration was 4 years, with patients receiving a median dose of 762 mg/kg/day.
“This approval represents a pivotal moment for the TK2d community who previously faced no FDA-approved therapeutic alternatives,” said Donatello Crocetta, Chief Medical Officer at UCB. “We extend heartfelt gratitude to the patients, families, advocates, and clinical trial teams who have partnered with us on this crucial journey.”
The most common adverse reactions observed during treatment included diarrhea, abdominal pain, vomiting, and elevated liver transaminases. KYGEVVI received multiple FDA designations, including Orphan Drug, Breakthrough Therapy, Priority Review, and Rare Pediatric Disease designations. With this approval, UCB received a Rare Pediatric Disease Priority Review Voucher redeemable for future regulatory submissions.
“I’ve studied mitochondrial diseases for over three decades and witnessed firsthand the profound impact TK2d has on patients and families. This approval marks a significant milestone in how we can support and manage this debilitating condition,” noted Dr. Michio Hirano, Professor of Neurology at Columbia University Irving Medical Center. UCB expects KYGEVVI to be commercially available in the United States in Q1 2026.
BrainXell Therapeutics, a San Diego-based biotechnology company, announced disclosure of significant preclinical scientific data for BXT-110, its lead investigational program in induced pluripotent stem cell (iPSC)-derived therapies for Parkinson’s disease.
BXT-110 represents an innovative regenerative approach for Parkinson’s disease, the second most common neurodegenerative disorder characterized by progressive loss of midbrain dopaminergic neurons. These essential neurons regulate dopamine production and control motor function, mood regulation, and cognitive processes. Restoring dopaminergic neurons has emerged as a significant therapeutic goal in Parkinson’s disease management.
The data presented highlight BrainXell’s progress in developing a robust, reproducible, and scalable approach for BXT-110, an autologous source of midbrain dopaminergic progenitor cells. Using both patient- and donor-derived iPSC lines, the team generated highly enriched cell populations with strong consistency across target markers. Notably, in vivo studies demonstrated significant graft survival and substantial functional recovery in a 6-OHDA rodent Parkinson’s disease model, with efficacy observed as early as 12 weeks and sustained for up to six months post-transplantation.
“These results underscore our capability to advance iPSC-derived therapies that can be transformative for patients living with neurodegenerative diseases,” said Katherine Vega Stultz, Chief Executive Officer of BrainXell Therapeutics. “Our data presentations reflect our commitment to rigorous science and deep expertise in iPSC biology and neuronal differentiation.”
The preclinical results establish BXT-110 as an efficacious, highly reproducible personalized cell-therapy candidate supporting its advancement as a potential disease-modifying treatment for Parkinson’s disease. BrainXell is advancing the program toward IND-enabling studies, building upon the foundation of research pioneered by Dr. Su-Chun Zhang, the company’s Advising Chief Scientific Officer.
CARsgen Therapeutics Holdings Limited, a company focused on developing innovative CAR T-cell therapies, announced clinical data for two allogeneic CAR-T product candidates. CT0596, a BCMA-targeting therapy for relapsed/refractory multiple myeloma, and CT1190B, a CD19/CD20-targeting therapy for relapsed/refractory non-Hodgkin’s lymphoma, both demonstrated initially favorable safety profiles and encouraging efficacy signals.
CT0596 in Relapsed/Refractory Multiple Myeloma
In an investigator-initiated trial evaluating CT0596, eight relapsed/refractory multiple myeloma patients received the BCMA-targeted CAR-T therapy developed on CARsgen’s proprietary THANK-u Plus™ platform. These patients represented a heavily pretreated population with a median of 4.5 prior therapy lines (range: 3-9), with five having received triple-class drugs and five with prior stem cell transplantation history. CAR-T cell doses escalated from 1.5×10⁸ to 4.5×10⁸ cells.
All eight patients achieved evaluable efficacy outcomes at a median follow-up of 2.56 months. Five patients achieved partial response or above, including three achieving complete or stringent complete response, one achieving partial response, and one achieving very good partial response. Notably, six of eight patients achieved minimal residual disease negativity at week four. One patient has maintained ongoing stringent complete response and minimal residual disease negativity for nearly six months. All eight patients showed CAR-T cell expansion, with no dose-limiting toxicities, treatment discontinuations, or deaths observed. Four patients experienced Grade 1 cytokine release syndrome, all resolving within 2-10 days.
CT1190B in Relapsed/Refractory Non-Hodgkin’s Lymphoma
Concurrent investigator-initiated trials evaluated CT1190B in 14 patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma, including follicular lymphoma, mantle cell lymphoma, and diffuse large B-cell lymphoma. At the recommended lymphodepletion dose, six evaluable patients achieved an overall response rate of 83.3%, comprising four complete responses and one partial response. Notably, all three follicular lymphoma patients achieved complete responses despite receiving multiple prior therapies, including immunochemotherapy, autologous stem cell transplantation, and advanced targeted therapies.
Safety findings revealed cytokine release syndrome, cytopenia, and infections as primary signals, with no immune effector cell-associated neurologic syndrome or graft-versus-host disease observed. Peak CAR-T cell expansion reached levels on the order of 10⁵ copies/µg genomic DNA in optimally dosed patients.
CARsgen anticipates submitting an Investigational New Drug application for CT0596 in the second half of 2025, advancing toward potential therapeutic availability for patients with urgent unmet needs in plasma cell malignancies and autoimmune diseases.
In an unprecedented industry move, Danish pharmaceutical giant Novo Nordisk launched an unsolicited ~$9 billion counteroffer to acquire obesity-focused clinical-stage biotech Metsera, directly challenging Pfizer’s previously announced $7.3 billion deal. The aggressive bid marks a dramatic escalation in competition for next-generation obesity treatments and signals strategic repositioning under Novo’s new leadership.
Pfizer’s initial agreement valued Metsera at $4.9 billion plus up to $2.4 billion in potential milestones. However, Novo Nordisk’s substantially higher valuation has triggered a landmark legal battle in the Delaware Court of Chancery, with Pfizer accusing the Copenhagen-based competitor of breach of contract and tortious interference while challenging the regulatory viability of Novo’s proposal based on antitrust and manufacturing structure concerns.
At the center of this high-stakes corporate confrontation lies Metsera’s innovative lead candidate, MET-097i, a first-in-class ultra-long-acting GLP-1 receptor agonist designed for potentially monthly dosing, a significant advancement over weekly injection regimens required by market leaders Novo’s WEGOVY and Eli Lilly’s ZEPBOUND. Recent Phase IIb data demonstrated remarkable efficacy in the VESPER-1 study, achieving a mean placebo-subtracted weight loss of 14.1% at 28 weeks at the highest dose (1.2 mg), with some patients losing as much as 26.5% of body weight.
“These results demonstrate MET-097i’s remarkable potency, requiring 5-10 times lower doses than current GLP-1 options while achieving comparable efficacy to tirzepatide,” explained the clinical findings. The drug’s tolerability profile proved equally compelling in the VESPER-3 trial, with only 13% increased nausea and 11% increased vomiting compared to placebo at the highest monthly dose, substantially lower than competing agents. Discontinuation rates due to adverse events reached only 2.9%, compared to up to 10% for other obesity medications.
Based on these positive results, Metsera is advancing toward Phase III trials in late 2025, with potential FDA approval projected for 2027 or 2028. Beyond MET-097i monotherapy, Metsera’s pipeline includes MET-233, an ultra-long-acting amylin analog potentially combinable as the industry’s first monthly multi-hormone therapy, plus MET-815, an oral prodrug formulation.
The competitive intensity of Novo vs Pfizer reflects extraordinary market growth dynamics. The anti-obesity medication market, valued at approximately $12 billion in 2024, is projected to reach $88 billion by 2034, representing a compound annual growth rate of 20.6%. Novo Nordisk’s OZEMPIC and WEGOVY, combined with Eli Lilly’s MOUNJARO and ZEPBOUND, have collectively generated $71 billion in US revenue since 2018.
For Pfizer, the potential loss of Metsera represents a significant setback following repeated clinical disappointments. The company terminated three separate in-house GLP-1 programs: Lotiglipron (discontinued late 2023 for elevated liver enzymes), Danuglipron (abandoned April 2025 for suspected drug-induced liver injury), and PF-06954522 (discontinued August 2025). Acquiring Metsera represented Pfizer’s critical opportunity to re-establish competitive positioning in this rapidly expanding therapeutic category.
Pfizer has secured early FDA antitrust clearance and requested a temporary restraining order to block Metsera from terminating their agreement pending the November 13, 2025, shareholder vote. The outcome will have far-reaching implications for M&A precedent in pharmaceutical consolidation among dominant market players.
