This week’s Neuroscience update highlights key developments, including regulatory actions, clinical trial progress, data releases, and strategic partnerships. The focus is on advancements that may have a significant impact on treatment options and patient outcomes.
In Today’s Newsletter
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In Today’s Newsletter
🧬 Apertura Gene Therapy & Finding Hope for FRRS1L Collaboration [1] [US • 18 Feb 2026]
Context: Apertura and Finding Hope for FRRS1L team up to develop gene therapy using the TfR1 CapX capsid for treating FRRS1L disease, a rare neurological condition.
Key Point: TfR1 CapX shows strong preclinical potential for delivering genetic medicine to the CNS.
Implication: This collaboration could lead to groundbreaking treatments for rare neurological diseases.
💊 FDA Accepts NDA for Tirabrutinib [2] [US • 20 Feb 2026]
https://www.targetedonc.com/view/fda-accepts-new-drug-application-for-btk-inhibitor-tirabrutinib
Context: Tirabrutinib, a selective BTK inhibitor, is accepted for review under the FDA’s accelerated approval pathway for treating relapsed/refractory CNS lymphoma.
Key Point: Phase 2 results show a promising 67% overall response rate.
Implication: Tirabrutinib could provide a new treatment option for a rare and aggressive lymphoma.
💡 Lario Therapeutics Awarded $2.4M for CNS Expansion [3] [UK • 19 Feb 2026]
https://finance.yahoo.com/news/lario-therapeutics-awarded-2-4m-083000415.html
Context: Lario receives funding to expand its research into neuronal calcium channels for treating Parkinson’s disease and PTSD.
Key Point: The funding is a significant boost for Lario’s platform focused on selective small-molecule inhibitors.
Implication: This support could advance treatment options for neurological and psychiatric conditions with high unmet needs.
💉 FDA Accepts NDA for Teva’s Olanzapine Injectable [4] [US • 20 Feb 2026]
Context: Teva’s NDA for TEV-749, an extended-release injectable formulation of olanzapine, is accepted by the FDA for treating schizophrenia.
Key Point: TEV-749 aims to provide a convenient once-monthly treatment for patients with schizophrenia.
Implication: If approved, TEV-749 could improve medication adherence and patient outcomes for schizophrenia.
💥 Roche Halts Enspryng Development in Duchenne Muscular Dystrophy [5] [Switzerland • 23 Feb 2026]
https://www.fiercepharma.com/pharma/roche-halts-development-enspryng-duchenne-muscular-dystrophy
Context: Roche discontinues Enspryng development in Duchenne muscular dystrophy after reassessing the trial’s regulatory viability.
Key Point: Development of Enspryng in DMD failed to meet the required criteria for regulatory approval.
Implication: Roche focuses efforts on more promising indications, such as neuromyelitis optica spectrum disorder.
🧠 Merck & Mayo Clinic Partner for AI Drug Development [6] [US • 23 Feb 2026]
Context: Merck collaborates with Mayo Clinic to apply AI and clinical data in developing treatments for neurological disorders, including multiple sclerosis.
Key Point: The partnership will use AI to analyze large-scale clinical data for better-targeted drug discovery.
Implication: This collaboration could accelerate breakthrough treatments for MS and other challenging CNS conditions.
🚫 Ionis Scraps Alzheimer’s Treatment for Down Syndrome [7] [US • 20 Feb 2026]
Context: Ionis halts its early-stage Alzheimer’s treatment trial (ION269) for people with Down syndrome due to slow enrollment.
Key Point: The trial saw minimal patient participation, leading to the decision to drop the candidate.
Implication: This setback highlights the challenge of developing Alzheimer’s therapies for high-risk populations such as those with Down syndrome.
⚖️ J&J Pauses Enrollment in Alzheimer’s Trial [8] [US • 19 Feb 2026]
Context: Johnson & Johnson temporarily halts enrollment in a Phase 2 trial of its anti-tau Alzheimer’s therapy, partnered with AC Immune.
Key Point: Enrollment was paused due to challenges with recruitment.
Implication: The trial continues, but recruitment issues raise concerns about the potential for accelerated progress in Alzheimer’s treatments.
🧬 SylamoreBio & Daiichi Sankyo Collaborate on SyLEC™ Technology [9] [US • 23 Feb 2026]
Context: SylamoreBio and Daiichi Sankyo collaborate to enhance SyLEC™ delivery technology, aiming for better therapeutic delivery across the blood-brain barrier.
Key Point: The collaboration will focus on CNS diseases and efficient delivery of therapeutic cargo.
Implication: This partnership could improve the delivery of treatments for neurological disorders, providing better efficacy and accessibility.
💡 Lario Therapeutics Awarded Additional Funding [10] [UK • 19 Feb 2026]
https://finance.yahoo.com/news/lario-therapeutics-awarded-2-4m-083000415.html
Context: Lario receives additional grant funding to expand its research on calcium channels for neurological and psychiatric diseases, including Parkinson’s and PTSD.
Key Point: The funding will support Lario’s work on selective small-molecule inhibitors targeting neuronal calcium channels.
Implication: Lario’s platform could lead to new treatment options for CNS disorders with high unmet medical needs.
Why it Matters
- Apertura Gene Therapy’s collaboration could result in a pioneering gene therapy approach for rare neurological diseases.
- FDA’s acceptance of tirabrutinib’s NDA provides hope for patients with limited treatment options for CNS lymphoma.
- Lario’s expansion into Parkinson’s and PTSD could lead to more effective treatments for these complex diseases.
- Teva’s olanzapine injectable could provide a more accessible treatment option for schizophrenia, improving patient adherence.
- Roche’s halting of Enspryng’s DMD development redirects focus on diseases with better treatment potential.
- Merck & Mayo Clinic’s AI collaboration might accelerate the development of new treatments for MS and other neurological conditions.
- Ionis’s setback in Alzheimer’s treatment underscores the challenges in developing therapies for Down syndrome patients.
- J&J’s pause in Alzheimer’s trial enrollment reflects the ongoing difficulties in recruiting participants for Alzheimer’s studies.
- SylamoreBio & Daiichi Sankyo’s partnership may revolutionize CNS drug delivery technology, addressing key therapeutic challenges.
- Lario’s grant funding strengthens its position in developing precision treatments for neurological disorders like Parkinson’s.
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FAQ
What is the significance of Apertura’s collaboration with Finding Hope for FRRS1L?
The partnership aims to advance gene therapy for FRRS1L, a rare and debilitating neurological disease [1].
What will tirabrutinib be used for if approved?
Tirabrutinib targets relapsed/refractory primary central nervous system lymphoma, offering a potential new treatment option [2].
What does Lario’s funding mean for Parkinson’s and PTSD?
Lario’s funding boosts its research on calcium channels as therapeutic targets for these disorders [3].
How could Teva’s olanzapine injectable impact schizophrenia treatment?
TEV-749 offers a once-monthly, subcutaneous treatment for schizophrenia, which could improve patient adherence and outcomes [4].
Why did Roche halt Enspryng’s development in DMD?
Roche’s decision was based on unmet regulatory requirements, leading to the discontinuation of the trial in Duchenne muscular dystrophy [5].
What is Merck & Mayo Clinic’s AI collaboration aiming to achieve?
The partnership aims to accelerate drug discovery for neurological disorders by leveraging AI and clinical data [6].
Why did Ionis stop its Alzheimer’s trial for Down syndrome?
The trial experienced slow patient enrollment, leading Ionis to discontinue the program [7].
What does J&J’s pause in enrollment mean for its Alzheimer’s trial?
The pause in enrollment reflects challenges in recruitment but does not affect the study’s progress [8].
What is SylamoreBio’s collaboration with Daiichi Sankyo aiming to achieve?
The collaboration seeks to advance drug delivery technologies for CNS diseases, overcoming biological barriers [9].
What will Lario’s additional funding support?
The funding will support further research into calcium channels as targets for Parkinson’s and PTSD treatment [10].
Entities / Keywords
Apertura Gene Therapy; TfR1 CapX; FRRS1L; gene therapy
Deciphera Pharmaceuticals; tirabrutinib; BTK inhibitors; CNS lymphoma
Lario Therapeutics; neuronal calcium channels; Parkinson’s disease; PTSD
Teva Pharmaceuticals; olanzapine; schizophrenia; injectable
Roche; Enspryng; Duchenne muscular dystrophy
Merck; Mayo Clinic; AI drug discovery; multiple sclerosis
Ionis Pharmaceuticals; Alzheimer’s disease; Down syndrome
Johnson & Johnson; AC Immune; Alzheimer’s; tau
SylamoreBio; Daiichi Sankyo; SyLEC; CNS drug delivery
Lario Therapeutics; Parkinson’s; PTSD; calcium channels
References
[2] https://www.targetedonc.com/view/fda-accepts-new-drug-application-for-btk-inhibitor-tirabrutinib
[3] https://finance.yahoo.com/news/lario-therapeutics-awarded-2-4m-083000415.html
[5] https://www.fiercepharma.com/pharma/roche-halts-development-enspryng-duchenne-muscular-dystrophy
[10] https://finance.yahoo.com/news/lario-therapeutics-awarded-2-4m-083000415.html