Machado-Joseph Disease

Funding supports ongoing Phase 1/2a clinical trial evaluating VO659 in patients with spinocerebellar ataxia type 3 and type 1 and Huntington's disease Financing led by Ackermans & van Haaren along with Droia Ventures, EQT Life Sciences and Kurma Partners with participation from Polaris Partners, Pureos Bioventures and Eurazeo LEIDEN, Netherlands, Jan. 5, 2024 /PRNewswire/ -- Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced a $60 million (€54 million) Series B financing. The financing was led by new investor Ackermans & van Haaren (AvH) with existing co-leads Droia Ventures, EQT Life Sciences and Kurma Partners along with prior investors Polaris Partners, Pureos Bioventures and Eurazeo. Continue Reading Micah Mackison, CEO of Vico Therapeutics "This financing positions us to accelerate our efforts to advance our lead program VO659, currently in a Phase 1/2a clinical study of patients with spinocerebellar ataxia types 3 and 1 and Huntington's disease," said Micah Mackison, chief executive officer at Vico. "We are pleased that a team of highly sophisticated investors, led by new investor AvH, have joined in this financing round, further validating our progress and the significant potential of VO659 to transform the lives of patients living with these devastating neurodegenerative diseases for which there are currently no disease-modifying treatments available." The financing will support overall company operations including advancement of the ongoing Phase 1/2a multi-center, open-label basket trial of VO659 in patients living with spinocerebellar ataxia type 3 (SCA3) and type 1 (SCA1) and Huntington's disease (HD). The trial is designed to assess the safety and tolerability of multiple ascending doses of VO659 administered intrathecally in participants with mild to moderate SCA3, SCA1 and early manifest HD, with the first patient dosed in April 2023. The financing will also help support discovery and research efforts to further expand the company's pipeline in other genetic neurological diseases. The company also announced that Jeroen Vangindertael, Ph.D., investment manager at AvH, will join Vico's board of directors. Prior to joining AvH in September 2020, Dr. Vangindertael was at BNP Paribas Fortis where he supported biotechnology companies through private equity investments. "We are proud to support Vico in this financing round as they continue to advance their lead clinical program and further develop a portfolio of novel antisense oligonucleotides," said Dr. Vangindertael. "We are impressed by Vico's progress in the clinic and believe their strong management team is well positioned to execute on their mission to bring their innovative therapies to patients in need." "The investment in Vico fits perfectly in AvH's Life Sciences investment strategy where we aim to offer growth capital to ambitious biotech companies building their clinical pipeline," added Piet Dejonghe, co-CEO of AvH. About Vico Therapeutics B.V. Vico Therapeutics is a clinical-stage genetic medicines company developing antisense oligonucleotide (ASO) RNA modulating therapies for patients with severe neurological diseases. Our lead product candidate, VO659, is currently in Phase 1/2a clinical development for the treatment of spinocerebellar ataxia types 3 and 1 and Huntington's disease and is the only ASO in the clinic that targets the underlying CAG repeat expansions that cause all nine known polyglutamine diseases. Vico's research platform designs fit-for-purpose ASOs by applying precision chemistry with mechanisms of action ideally suited to target genetic neurological diseases. For more information, visit . Media Contact Jenna Urban Berry & Company Public Relations [email protected] 212-253-8881 SOURCE Vico Therapeutics B.V.

Pictured: A doctor holds an MRI of the brain/iStock, Chinnapong Several late-stage clinical trials are expected to produce data in the final months of 2023 that could spell new approaches to treating Alzheimer’s disease, amyotrophic lateral sclerosis, Huntington’s disease and multiple sclerosis. Mizuho Securities recently published a report highlighting some of the trials to watch as the year comes to a close, and BioSpace spoke with Graig Suvannavejh, managing director of equity research in biopharmaceuticals and biotechnology at the firm, to find out more. Alzheimer’s Disease Eisai and Biogen’s subcutaneous Leqembi In July, the FDA fully approved Leqembi (Lecanemab), a monoclonal antibody developed by Eisai and Biogen that binds to soluble and insoluble amyloid-beta peptides to treat Alzheimer’s. Leqembi is currently administered only through intravenous injection, which limits the locations where patients can obtain treatment. To enhance its accessibility, the partners launched an open-label, randomized Phase I clinical trial in September 2022 to evaluate the bioequivalence of a single subcutaneous dose (720 milligrams) of Leqembi compared to an equivalent dose delivered through intravenous injection. Data from the subcutaneous study are anticipated in October 2023, and regulatory submissions for the maintenance dosing regimen and subcutaneous formulation are expected in the first quarter of 2024. Currently, to receive treatment, “You have to drive to an infusion center and sit in a chair for a few hours,” Suvannavejh told BioSpace. He emphasized the strategic value of a treatment that could be administered under the skin at home by a caregiver and said this readout is important for Leqembi’s commercial success. Huntington’s Disease Wave Life Sciences’ WVE-003 WVE-003, developed by Wave Life Sciences, is an allele-selective oligonucleotide that attacks mutant proteins that produce the symptoms of Huntington’s disease (HD). It is designed to target the huntingtin (HTT) mRNA that carries a single nucleotide polymorphism that appears on the mutant HTT (mHTT) allele. By lowering the quantity of the mutant form of this protein, WVE-003 allows the wildtype (wtHTT) allele to function, preserving its beneficial effects in the central nervous system. “HD is a tragic and unrelenting disease that passes through generations of families and has been compared to having ALS, Parkinson’s and Alzheimer’s all at once,” Paul Bolno, Wave’s president and CEO, told BioSpace by email. “Currently, there are no disease-modifying treatments for HD, so our primary goal is to develop a treatment that slows or stops progression of the disease.” The Phase Ib/IIa SELECT-HD trial is evaluating WVE-003’s ability to reduce mutant HTT in the cerebrospinal fluid compared to a placebo. The study aims to optimize the dose level—30 mg, 60 mg or 90 mg—and frequency of WVE-003 administration to reach the desired early indicators. "If WVE-003 is successful in the clinic, it has potential to be truly transformational,” Bolno said. Beyond treating symptomatic disease, he said Wave’s mHTT-lowering approach, which spares wtHTT, may also be beneficial in treating patients earlier, perhaps before symptoms of the disease present. “We are resolutely focused on what clinical success would mean to these families—and to future generations.” Wave expects to deliver additional single-dose and available multi-dose biomarker and safety clinical data in the second half of 2023. Amyotrophic Lateral Sclerosis Seelos Therapeutics Inc.’s SLS-005 Developed by Seelos Therapeutics, SLS-005 (Trehalose) is being evaluated in the HEALEY ALS Platform Trial, a novel approach by which companies can conduct ALS clinical trials more efficiently. “Currently, there are no treatments available that address the entire broad spectrum [of] patients with ALS,” Seelos Founder and CEO Raj Mehra told BioSpace in an email. “SLS-005, if successful in the HEALEY trial, would become the first drug to treat the broad ALS population based on a well-established and specific mechanism of action," he said. The registrational Phase II/III trial is examining the effectiveness of SLS-005 at stabilizing proteins and promoting autophagy in the brain to clear toxic protein aggregations characteristic of ALS. The results compare disease severity following a 90.5 mg/mL infusion of SLS-005 or a placebo for 24 weeks. Seelos plans to release top-line data from the trial before the end of 2023. The company initiated an Expanded Access Program in April 2023 for ALS patients who are ineligible for the clinical trial. “SLS-005 is not just one drug for one disease but one drug for multiple diseases,” Mehra said. The company is also evaluating SLS-005 in a late-phase registration-directed trial for SCA3 (or Machado-Joseph disease), an autosomal dominant cerebellar ataxia that occurs due to CAG repeat expansions in the ataxin protein. SLS-005 received the FDA’s Orphan Drug designation in 2020. Multiple Sclerosis Atara Biotherapeutics’ ATA188 Developed by Atara Biotherapeutics, ATA188 is a T-cell immunotherapy that targets Epstein-Barr virus (EBV)-infected B cells to treat nonactive secondary progressive multiple sclerosis (MS) and nonactive primary progressive MS. Most treatment options for relapsing-remitting MS reduce flares but do not inhibit the worsening of disability in progressive forms of the disease. Current research supports the role that EBV plays in the development of MS. ATA188 is composed of T-cells obtained from healthy donors previously infected with EBV. The cells are primed to recognize and attack EBV proteins to potentially slow or halt disease progression. Atara initiated the Phase I/II EMBOLD clinical trial in 2017 to determine the safety, tolerability and optimal dose of ATA188 and to evaluate its effect on clinical disability using the Expanded Disability Status Scale. The second half of the study evaluated the drug in participants with progressive forms of MS. Data from this phase, which will detail the drug’s ability to ease disease-related disability, is anticipated in November 2023. Stacy Kish is a freelance science writer based in Pittsburgh, PA. Reach her at earthspin.blog.

Seelos Therapeutics’ intranasal racemic ketamine candidate has missed the primary endpoint in a Phase II trial of major depressive disorder patients with acute suicidal ideation, spooking investors as the company deals with the aftermath of recent financial strain. The company’s stock $SEEL tanked by more than 75% on the Nasdaq to $0.24 in response to Wednesday’s news. Seelos’ Phase II trial was set to enroll 220 MDD patients requiring hospitalization due to suicide risk, but financial constraints meant only 147 took part in the trial. These constraints also forced Seelos to temporarily halt enrollment in its Machado-Joseph disease study in March.