SJLife Sciences

???? Neuroscience News – 7 day recap: 22/11 ? ?? Clinical News uniQure dosed their first patient in a Phase I/IIa clinical trial dubbed GenTLE evaluating AMT-260 targeting the treatment of refractory mesial temporal lobe epilepsy Cerevance dosed their first patient in a Phase 3 clinical trial dubbed ARISE evaluating Solengepras targeting the treatment of Parkinson’s disease Ractigen Therapeutics announced they’ve been granted FDA Orphan Drug Designation for RAG-21 targeting the FUS gene for the treatment of ALS Sangamo Therapeutics, Inc. announced FDA clearance of their IND application for ST-503, a treatment targeting chronic neuropathic pain Voyager Therapeutics, Inc. selected a lead development candidate, VY1706, for their tau silencing gene therapy program targeting Alzheimer’s. Voyager plan to file an IND and CTA TrueBinding received FDA IND approval for TB006 – a potential advancement for the treatment of Parkinson’s disease QurAlis successfully completed dose-escalation phase in their Phase 1 clinical trial ANQUR evaluating QRL-201 for the treatment of ALS Sage Therapeutics made the decision to stop?the development of dalzanemdor after it missed the primary endpoint of improving cognitive function in a Phase II targeting Huntingdon’s. They will also discontinue the ongoing open-label PURVIEW study which is primarily assessing the safety of dalzanemdor ?? Financial News Synapticure Inc. raised $25M in a series A allowing them to expand their virtual care for patients and caregivers who live with neurodegenerative diseases. This will be achieved by expanding partnerships with healthcare providers and payers, further investment in technology, fast-tracking clinical research and scaling their national medical group CND Life Sciences received a grant worth $4.2M from The Michael J. Fox Foundation for Parkinson's Research. This will support CND in an 18 month clinical initiative dubbed the Syn-Q study for those suffering with Parkinson's Vincere Biosciences have been awarded a $500,000 grant from The MJ Fox Foundation allowing them to develop their treatments targeting Parkinson’s Tiziana Life Sciences announced a grant application has been approved from The ALS Association. This will fund a 20-patient clinical trial aimed at evaluating the safety and early-stage parameters of disease improvement in ALS ?? Collaborations Alamar Biosciences, Inc. entered a collaboration with The MJ Fox Foundation to accelerate biomarker discovery for Parkinson’s disease and other neurodegenerative diseases Neuron23 entered a collaboration with Roche to incorporate a digital biomarker developed by RIS themselves. This will be used as the primary endpoint in a Phase 2 trial dubbed NEULARK testing NEU-411 for early-stage Parkinsons Verge Genomics announced Eli Lilly and Company have opted to pursue the development of two validated drug targets for ALS due to their on-going collaboration which was established in July 2021

Fierce Pharma’s Fiercest Women in Life Sciences 2024. Time to cast your vote! It should go without saying why we're highlighting this - equality and diversity is still in a terrible position. Fierce Pharma's Fiercest Women in Life Sciences 2023 article highlights this with some really key stats. Please read last years article - even if you remember it - read it again and remind yourself why this is such an important topic. https://lnkd.in/eYT5F_HK Below is a link to the article & voting page for 2024 but firstly let’s stop and take note of last years top 10: Amy Emerson Ana (Arroyo) Hooker, MBA Bari Kowal Dena Marrinucci Hanadie Yousef Lynelle Hoch Maha Katabi, PhD, CFA Marlena Fejzo Priya Abani Reshema Kemps-Polanco https://lnkd.in/g7Sb6AH8 https://lnkd.in/gcPmbuWE Vote now!

We are thrilled to announce that AC Immune and Takeda have won the #Deal of the Year (>€500M) at the European Lifestars Awards - Celebrating Life Science Leaders. This recognition highlights our transformative license and option agreement centered on ACI-24.060, our active immunotherapy candidate targeting toxic forms of amyloid beta in Alzheimer’s disease. Currently under evaluation in the ABATE Phase 1b/2 trial, ACI-24.060 has also received Fast Track designation from the U.S. FDA, emphasizing its potential to address this critical unmet medical need. This #partnership with Takeda embodies our commitment to pioneering precision prevention for neurodegenerative diseases. Thank you to our dedicated team at AC Immune and our partners at Takeda for making this possible. Together, we will reshape the future of #Alzheimer’s #treatment and #prevention. #Neurodegeneration #PrecisionMedicine #Award #Biotech #Biopharma

???? Neuroscience News – 7 day recap: 01/11 ? ?? Clinical News Capsida Biotherapeutics have been granted ODD by the FDA for CAP-002, a preclinical gene therapy targeting developmental and epileptic encephalopathy (DEE) Anavex Life Sciences achieved pre-specified efficacy in a Phase IIb/III trial with blarcamesine in an Alzheimer’s trial. The trial showed blarcamesine, an oral, once daily treatment, meaningfully slowly clinical decline in early Alzheimer’s patients Sinaptica Therapeutics have met primary and key secondary endpoints in a 12 month Phase 2 study to slow Alzheimer’s progression. They’re achieving this through non-invasive personalised neuromodulation Cognito Therapeutics, Inc. announced new data for Spectris? in a Phase 2 study dubbed OVERTURE aiming to reduce Alzheimer’s Dependence Score in patients with mild to moderate AD. Spectris has reduced the dependence score by 56.4% and also indicating preserved function Supernus Pharmaceuticals, Inc. presented data of their Phase 2a study of SPN-820 in adults suffering from major depressive disorder (MDD). Data demonstrated rapid and meaningful decreases in depressive symptoms including an 80% decrease in suicidal ideation Noema Pharma announced first patients dosed in a Phase 2b study of Gemlapodect, a first-in-class investigational therapy for Tourette Syndrome NKGen Biotech, Inc. presented new data of troculeucel in a Phase 1/2a trial targeting Alzheimer’s. Data has shown positive preliminary cognitive results after newly analysed biomarker data from initial dose-escalation Phase 1 trial showed a reduction in key dementia-related biomarkers ?? Financial News AbbVie have purchased Aliada Therapeutics for $1.4B in cash. AbbVie will take ownership of the entire pipeline including ALIA-1758 currently in a Phase 1 clinical trial for Alzheimer’s. Also taking ownership of Aliada’s novel BBB platform to enhance discovery efforts Axonis Therapeutics raised $115M in Series A financing. Axonis are targeting neurological disorders with the aim being to restore functional inhibitions in the CNS. Funds will be used to advance their pipeline and lead development candidate AXN-027 - aimed to treat epilepsy and pain - will enter human testing Leal Therapeutics announced $45M in financing. Leal are developing novel therapeutics for those suffering with CNS disorders. Lead programs are LTX-002 for those suffering with genetic or sporadic ALS and LTX-001 for those with schizophrenia which will be now be pushed into the clinic NRG Therapeutics have nominated their first development candidate, NRG5051, and in doing so have achieved a key milestone leading to a $5M grant from The Michael J. Fox Foundation. The candidate is aimed to treat Parkinsons and potentially other diseases such as ALS ?? Collaborations Biogen and Neomorph, Inc. have entered into a research collaboration to both discover and develop molecular glue degraders for Alzheimer’s, rare neurological and immunological diseases.

Today we announced the start of a first-in-human Phase 1 clinical trial for our antisense oligonucleotide AIC468. With this candidate, we aim to treat BK virus infections in kidney transplant patients. With no approved treatments for BK virus, this trial represents a significant step forward in addressing an urgent unmet medical need. The trial will assess the safety, tolerability, and pharmacokinetics in healthy volunteers.? ?? With multiple programs now in the clinic, AiCuris remains committed to developing therapies that can make a meaningful impact on the lives of immunocompromised patients.? ?? To read the full announcement, go to our website: https://lnkd.in/edJKPVGA

Axonis is very excited to share the news that we have raised a $115 million Series A financing to support the development of novel neuromedicines targeting KCC2. The financing was co-led by Cormorant Asset Management, LP and venBio, with significant investments from Sofinnova Investments , MRL Ventures Fund LLC, Perceptive Advisors, Solasta Ventures and Lumira Ventures. Our lead program, AXN-027, is being advanced for the treatment of epilepsy and pain. https://lnkd.in/eXWMmc7v

???? Neuroscience News – 7 day recap: 25/10 ? ?? Clinical News Cerevance published full results from their Phase 2 clinical trial of solengepras which is designed to treat Parkinson’s disease through a once daily oral use. The trial demonstrated a significantly reduced OFF time in those trialled also finding it to be well tolerated. Nido Biosciences have been granted EMA Orphan Drug Designation for their lead candidate NIDO-361 which is aimed at treating Kennedy’s disease. This comes off the back of completion of enrolment for their Phase 2 study. OKYO Pharma have announced a first patient has been dosed with their asset OK-101 which is the first IND granted by the FDA specifically for patients with Neuropathic Corneal Pain (NCP). Muscular Dystrophy Association have announced their groundbreaking MDA Kickstart Program which is aimed at affecting over 300 neuromuscular conditions (95% of which affect less than 1,000 individuals in the US) has been granted an FDA Rare Pediatric Disease Designation and Orphan Drug Designation for congenital myasthenic syndrome which is caused by a CHAT gene deficiency. Alto Neuroscience announced their lead asset ALTO-100, a treatment for major depressive disorder (MDD) failed to meet all endpoints in a Phase 2b trial. The NHS have decided not to proceed with Donanemab deeming it too expensive compared to the benefits, stating other treatments are being developed in the space. Orion Corporation have terminated ODM-111 a treatment for acute and chronic pain due to the therapeutic window of the molecule being too narrow. Important note: the non-clinical and Phase 1 trial, with shorter durations of exposure to ODM-111, didn’t reveal any significant safety concerns. ?? Financial News Seaport Therapeutics have raised $225M in an oversubscribed Series B round of funding, a mere 6 months after launching with $100M. This will support key clinical milestones for their neuropsychiatric medicines. Coya Therapeutics, Inc. have announced a private placement of approx. $10M gross funds due to the purchase and sale of shares. EydisBio, Inc. have been awarded a $0.5M grant from the The National Institutes of Health allowing them to investigate innovative approached in Alzheimer’s disease. ?? Collaborations Roche have entered a second research collaboration with Dyno Therapeutics in order to advance next-gen gene therapies for neurological diseases. Roche will make an upfront payment of $50M with the pledge of more than $1B in pre-clinical, clinical and commercial milestones plus royalties on potential net sales. AbbVie and Gedeon Richter have announced a new collaboration after working together for nearly two decades. The new collab will see the co-development and license agreements of potential treatments for neuropsychiatric conditions. Roche have dropped another Alzheimer’s candidate, UCB0107, the third this year and this time with UCB. The termination of agreement will see all rights handed back to UCB.