AKV9 from Silverman Lab/Northwestern offers hope.

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Megatron

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akavatx.com

Pipeline - AKAVA THERAPEUTICS

AKAVA Therapeutics’ programs are all based on some type of inhibition: inhibition of protein aggregation, inhibition of enzymes, and inhibition of tumor growth. We, therefore, refer to our therapeutics as inhibition therapeutics.
akavatx.com akavatx.com
The Silverman Lab at Northwestern University, and affiliated Akava Therapeutics, have been granted Orphan Drug status by the FDA for their compound, AKV9. Dr. Silverman essentially invented Lyrica. The team working on this is incredibly impressive. Based on their literature, they are essentially treating diseased motor neurons and it appears restoring their functionality, increasing axon length, etc. I've been following this since my diagnosis nearly a year ago. I have asked them if they would allow one of my ALS clinics to administer this compound to me and await their answer. I hope I have followed all the rules; this is my first post since discovering this resource.
 
I read about NU-9 a few years ago and thought it sounded promising. I'm happy to hear things are still moving forward with this possible drug. The data looks compelling in the mouse models. My PALS was UMN-dominant and it was a glimmer of hope for us. While it didn't arrive in time for him, it would be wonderful if it would be a successful treatment for others with ALS. I hope they will let you give it a shot!
 
Nice! I didn't see that AKV9 was granted orphan drug status. I've been following this one since it was first announced as well! Their team is incredible, as you said.
 
While the Silverman Lab didn't have a current clinical trial for me to join for AKV9, they gave me a link to the Healey trial at Mass General. This will be a massive, multi-treatment platform trial with hundreds of trial participants. It's an entirely new approach to the global ALS problem. Everyone should go to their website and attempt to participate!
 
The Healey platform trial has been going on for several years. They are currently enrolling regimen F and we have had results from several regimens already. If you are interested in reading you will find a number of posts on it here.

There are weekly update webinars at 5 pm ET Thursdays with an opportunity for questions. Old webinars including ones on the science of each regimen are archived on the Healey platform site. To be clear there is no need to travel to MGH there are sites across the US - over 40

IIRC the main criteria are to be within 3 years of symptom onset with svc greater than 50. Some of the regimens have additional criteria based on safety issues and like virtually all trials you can’t take any experimental meds and need to be on stable doses of ALS approved meds for at least 30 days

CTG Labs - NCBI

www.clinicaltrials.gov www.clinicaltrials.gov
 
Attached please find the response this date from MassGeneral/Healey ALS Platform Trial. It's frustrating to be excluded due to one or two factors.

I am sorry to learn of your ALS diagnosis, and appreciate your interest in the HEALEY ALS Platform Trial. If you would like to learn more about what it means to use this platform trial approach in research, the following short video provides a good overview: Something New Is Here.



We are partnering with more than 70 research centers across the USA and each center is responsible for enrolling its own participants. Please reach out to any of the centers that are within your geographical area to explore enrollment opportunities The Platform Trial involves regular in-person study visits over the course of 6 months, so we encourage patients to pursue enrollment at a conveniently located center. You are welcome to contact multiple sites if there are a few feasible options.

As with every clinical trial, there are necessary inclusion and exclusion criteria. If yo A few key criteria to note are that participants need to be within 36 months of ALS weakness symptom onset, have a Vital Capacity greater than or equal to 50%, and have the ability to swallow pills and liquids at the time of the Master Protocol Screening visit. Please let me know if you have any questions after reviewing the criteria.

If you would like to email a Platform Trial site/(s) to express interest in participating, it is helpful to provide the research team with the following information: where you live, where you currently receive clinical care for ALS, the month/year of weakness onset due to ALS, most recent Vital Capacity measurement and when that was obtained, and whether you are able to swallow pills and liquids. Given the amount of interest we have received for this trial, it can sometimes take sites up to 3-5 business days to personally respond via email. If you would like assistance communicating with one of our research sites, please let me know and I will follow up on your behalf.
Please note that we have completed the enrollment goal of 160 people for the first five regimens in the Platform Trial (Regimens A, B, C, D, and E). The sixth regimen, Regimen F, is currently enrolling and we plan to get many Platform Trial sites up and running for Regimen F over the course of the next two months. Sites will be activated on a rolling basis. Once a site is ready to begin enrolling participants, it will be listed as "recruiting"You are welcome to contact sites prior to activation if you would like to be added to their list for Regimen F.

Regimen F is evaluating the safety and efficacy of an investigational product called ABBV-CLS-7262, which is manufactured by the company Calico Life Sciences in collaboration with AbbVie. To read the press release about Regimen F, please click here. To watch Calico’s short video about the science behind ABBV-CLS-7262, click here. Please see the attached PDF brochures to learn more. We also hosted a Drug Science Q&A Webinar about Regimen F on March 27th, and the webinar recording is a great resource to learn about the science that led to ABBV-CLS-7262 being included in the Platform Trial.

Regimen F Drug Science Webinar Recording:
View: https://www.youtube.com/watch?v=bw3HI59Yj-k

Short Video- Lumbar Puncture Step by Step:
View: https://www.youtube.com/watch?v=3omIOEnll8o

We are also in the process of getting our Platform Trial sites up and running for Regimen G, which is evaluating Denali Therapeutic’s investigational product DNL343. To read the press release about Regimen G, click here. Some sites may already be open for both Regimen F and Regimen G, so it is best to discuss enrollment opportunities with your local study team. I’ve attached the brochure for Regimen G if you’d like to learn more. We will be hosting a public Regimen G Drug Science Q&A Webinar on Thursday, July 20th (5:00-6:00pm Eastern), click here to register in advance.

Our weekly webinars are a great way to stay updated on trial progress, so I encourage you to attend. To view information regarding our weekly Platform Trial Q&A webinars (Thursdays, 5:00-5:30pm Eastern time), . Registration is required and can be done on our website by checking the box next to the webinar you are interested in attending. There is no limit to the number of webinars you can attend, so please register in advance for as many as you wish. If you are unable to attend, recordings of these webinars will be made available i


Please don’t hesitate to reach out with any follow up questions. I would be happy to schedule a phone call to discuss the trial with you if that would be helpful.

Warmly,

Catherine

________________

Catherine Small, BS

Pronouns: she, her, hers

Patient Navigator, HEALEY ALS Platform Trial

T 833-425-8257
 
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My PALS started the regimen F trial yesterday. He has a 75% chance of getting an active drug. Keeping our fingers crossed. If he’s on the placebo arm, he can switch to the active drug in 6 months. Dr Bedlack is very hopeful about this medication.
Lee
 
How does this MassGeneral/Healey ALS Platform Trial work for a person that lives in Wisonsin ? Are there Doctors around here or a a few states away that are in this ? Sounds intresting and some kind of hope Thanks for any help on this
 
There are 60 plus sites in the US for Healey. See here CTG Labs - NCBI

It looks like Milwaukee is not recruiting just now but 1 that may change soon or 2 perhaps there is another site geographically accessible.

Every Thursday ( except holidays) at 5 pm Eastern there is a webinar by mgh. Also there are trial navigators you can contact with questions. I can not link due to forum rules. Just google mgh healey platform and you should get the section of the clinic site devoted to this. In it you will find links to sign up for the webinar plus old webinars plus information and links to the navigators
 
I was reading through some press releases from this team and came across one from May 2022 that said they were hoping to start phase 1 trials early this year, and phase 2 in early 2024.

As far as I can tell there hasn't been any movement, and I haven't been able to find any updates on their timeline. Anyone heard anything? I reached out via email but assume they won't get back to me. Though I'll update if they do.
 
Looks like they are still writing the IND (Investigational New Drug application) and conducting the studies to support the application. They can't dose any humans until there is an approved IND.

It is very common to miss the initial notion of the development timeline.
 
Other than their timeline being pushed back, their original hope was phase 2 starting 1 year after phase 1. Is that a normal timeline? In the past I haven't followed current early-stage trials too closely, but I was surprised to see how quickly they planned to get to phase 2.
 
I think there is a recent video with Dr Silverman re NU 9 on everything als that should have some updates on trial status.

The FDA has new leadership -- would be good to know what the process is for quick approvals following successful phase 2s. Seems like there may still be a major problem with the FDA regarding their lack of urgency for our particular disease ---- no one really knows apparently.
 
Yes, it would be aggressive to expect dosing in Phase 2 to take place a year after data lock in Phase 1. Past data analysis and reporting, Manufacturing has to ramp up, a protocol has to receive all the necessary IRB approvals, centers have to be recruited/operationalized, then they have to recruit patients in turn, etc. You can work ahead on Phase 2 planning in Phase 1, but there's only so much you can do in advance.

Orphan Drug designation doesn't speed up the timeline, though financial incentives are provided. The mfr will likely file for Fast Track and Priority Review designations once there is some data to support. Receiving those would help speed things up. Also, a small spinout like this will usually look to partner with a bigger biopharma at some point, if all goes well.

There is always an end of Phase 2 meeting w/ the FDA and they are aware of the urgency of bringing new ALS treatments to market. The better the early efficacy signal vs. any toxicity, the more behind-the-scenes but active support they are likely to provide, within existing regulatory frameworks.

What some manufacturers and patient groups (not referring to Akava) fail to understand is they would be better served to negotiate with the FDA off camera, rather than try to publicly shame them into action, which seldom works.
 
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