Phase 1 study in humans
Drum candidate AKV9 (formerly NU-9) has got the green light from the FDA for Phase 1 first-in-human study to evaluate safety and tolerability. Key member of the research team that developed the compound, Prof Hande Ozdinler, has foreshadowed potential applicability to the treatment of the HSPs, separate from its primary purpose at this stage, which is the treatment of motor neurone disease (ALS).
WINNETKA, Ill.–(BUSINESS WIRE)–Akava Therapeutics, Inc., a pioneering biopharmaceutical company focused on developing first-in-class small molecule therapeutics that act through inhibition of key pathways in neural crest-derived targets for a variety of neurodegenerative diseases and cancers, celebrates a significant achievement.
On July 3rd, the U.S. Food and Drug Administration cleared Akava’s Investigational New Drug (IND) application for the treatment of amyotrophic lateral sclerosis (ALS) to proceed with a Phase I first-in-human study in healthy subjects using Akava’s investigational new drug, AKV9 (formerly NU-9). The study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of AKV9.
SOURCE: Business Wire, August 22, 2023
Akava Therapeutics, Inc. Announces FDA Clearance of Investigational New Drug Application for the Treatment of Amyotrophic Lateral Sclerosis
Akava Therapeutics, Inc. press release
Can we participate in this human study?
Editor’s note: No, for 2 reasons – firstly it is a Phase 1 clinical trial. These are done with healthy people, with the aim of testing safety and tolerability of the drug candidate. If the trial progresses to Phase 2, participants with motor neuron disease (ALS) will receive the drug to determine its effect on the disease.
Why this clinical trial is being closely watched by people with HSP is that this drug may work on a disease mechanism that some of the HSPs share in common with ALS and other neurodegenerative conditions as well. Success in this clinical trial for ALS would lead on to clinical trials for AKV9 to treat HSPs.
Thank you for your answer. How long will the Phase 1 clinical trial take?
Editor’s note: The best guess without knowing any of the design details is that the trial itself may take around 6 months, depending on a number of variables, any and all of which could alter this timeframe quite a bit. It is assumed that the trial will start with a group taking the lowest dose, with the results assessed before starting the next group on a higher dose, and so on. If they are well organised and have all the requirements in place to begin the trial, the results may be known by the end of March next year.
If this ALS phase 1 clinical trial is successful and AKV9 drug is also suitable for treating HSPs, can ALS and HSPs be conducted in phase 2 clinical trials simultaneously?
Editor’s note: A successful Phase 1 trial will most likely be applicable for the HSPs if the intended dosing range is similar. However separate Phase 2 trials would be necessary for ALS and the HSPs as these must be specific to a particular disease. An initial Phase 2 trial for the HSPs may focus on just one HSP type or possibly a group of HSP types with the same disease-causing mechanism. It is possible that several Phase 2 trials would need to be conducted to test treatment efficacy/effectiveness across a range of HSP types if there are initial signs of success to warrant this. These would be called Phase 2a trials for testing preliminary effectiveness. If successful, a number of multisite/multinational Phase 2b trials including larger numbers of participants would need to be conducted successfully as a minimum before gaining regulatory approval to market the drug. So it is a lengthy road – and a costly one.
Please let me know if they have AKV9 clinical trials in San Diego or LA. I live in San Diego but will drive to LA.
That’s very good news! I have HSP SPG7!
Approximately how long could it take until there could be an effective medication! Thank you in advance for your response!
Editor’s note: There is no way of estimating this. Until a therapeutic treatment is proven effective in a clinical trial, any time estimate is just a guess. Once effectiveness is established for a treatment, it could be available in a further 2-3 years once additional clinical trials have confirmed the results, regulatory approvals applied for and obtained, and the pharmaceutical industry produces and markets the treatment.
Will their be trials for those with HSP in the Houston area and when and where if you know? Thank you and thank you for all you have done for so many of us!!!
Editor’s note: Way too early to know if there might be HSP trials in the future. They are starting with Phase 1 trials with the focus on ALS initially.
Editor’s note: Potentially yes. Variants in the ALS2 gene are associated with three overlapping conditions – see here for more: https://www.omim.org/entry/205100?search=als2%20gene&highlight=als2%20gene. Your neurologist and/or geneticist may be able to provide a more specific guide to potential applicability, but early days yet for this drug candidate, with a phase I trial planned.
My daughter has a mutation in the ALS2 gene. Would this be something that could potentially benefit her?
Thank you for the update on the stages of research on AKV9 approval process. I am a Maryland resident with HSP Spg4 and will follow the results.