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After two meetings with the U.S. Food and Drug Administration (FDA), <a href="https://coyatherapeutics.com/">Coya Therapeutics</a> says it&#8217;s received constructive feedback — and has reached an alignment with the agency regarding development plans for COYA 302, its therapy candidate for people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The company held the two meetings — a pre-investigational new drug (IND) meeting and a type C meeting — to seek advice from the FDA. Now, according to COYA, the two parties are in agreement regarding key areas of COYA 302&#8217;s development, to include its manufacturing, preclinical research, and clinical studies, which should support the filing of an IND application seeking clearance to start clinical trials.
That application is expected to be submitted in the first half of 2024, the company said in a <a href="https://www.businesswire.com/news/home/20240105424614/en/Coya-Therapeutics-Announces-Successful-Pre-IND-and-Type-C-Meetings-with-the-FDA-to-Advance-the-Development-of-COYA-302-for-Treatment-of-Amyotrophic-Lateral-Sclerosis-ALS">press release</a>.
If it&#8217;s accepted, Coya will soon thereafter launch a double-blind clinical trial to test COYA 302 in people with ALS. In a double-blind study, neither researchers nor participants know what treatment, if any, patients are receiving.
“This important feedback allows us to advance our development program in ALS with a planned double-blind controlled study, with the potential for bringing forward a much-needed therapy for ALS patients,” said Fred Grossman, Coya’s president and chief medical officer.
[recommended-reading id=82985]
<h3>In proof-of-concept study, COYA 302 showed marked benefits</h3>
ALS is characterized by progressive damage to motor neurons, the nerve cells that control voluntary movements. It renders the brain less able to control muscle movements.
<a href="https://coyatherapeutics.com/science-and-technology/">COYA 302</a> is an experimental approach that combines a low dose of the immune signaling molecule interleukin-2 (IL-2) and a fusion protein named CTLA-4 Ig (abatacept).
IL-2 is intended to reduce inflammation by increasing the levels and activity of anti-inflammatory immune cells called regulatory T-cells, or Tregs. CTLA-4 Ig, meanwhile, aims to decrease the activity of immune cells that promote inflammation and damage in ALS.
The therapy, administered via a subcutaneous or under-the-skin injection, has already been tested in four ALS patients in a proof-of-concept clinical trial. The participants received treatment for 48 weeks, or nearly one year, and were followed for eight more weeks after stopping COYA 302.
Results of the small study showed that ALS progression was markedly slowed during the treatment period, with patients experiencing similar levels of disability after 48 weeks compared with before treatment.
Prior to treatment, participants were losing a mean of 1.1 points per month in their ALS Functional Rating Scale (ALSFRS-R) scores, indicating a lower ability to perform daily tasks each month. During the entire study period, scores dropped a total of 1.5 points, on average.
However, once patients stopped COYA 302, progression rates returned to those observed before the trial started, indicating a need for continuous treatment.
Similarly, there was a statistically significant increase in Treg anti-inflammatory activity during the treatment, which also decreased to starting, or baseline, levels eight weeks after treatment discontinuation.
The study also evaluated biomarkers of inflammation and oxidative stress — a type of cell damage associated with inflammation. Available data up to 16 weeks, or about four months, suggested a decrease in these biomarkers, consistent with the observed increase in Treg function.
[quote=&#8221;center&#8221;]We believe that gaining alignment with FDA through multiple regulatory meetings on the path to filing an IND in [the second quarter of] 2024 is an important next step in advancing the program in patients with ALS.[/quote]
The therapy was generally well tolerated, with no serious side effects reported and no treatment discontinuations. The most common side effects were mild reactions at the injection site.
Coya plans to continue working closely with the FDA throughout COYA 302&#8217;s development program, the company said.
“We believe that gaining alignment with FDA through multiple regulatory meetings on the path to filing an IND in [the second quarter of] 2024 is an important next step in advancing the program in patients with ALS,” said Howard H. Berman, PhD, Coya’s CEO.

Coya aligns with FDA on plans for developing COYA 302 for ALS

The biotechnology company <a href="https://projenx.com/">ProJenX</a> recently received $15 million in funding to advance its experimental oral treatment <a href="https://projectals.org/project-als-introduces-prosetin/">prosetin</a> for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
This Series A financing round is essentially the next round of funding after seed money was used to rapidly develop the investigational treatment.
Both rounds were led by Medical Excellence Capital, which <a href="https://alsnewstoday.com/news/projenx-develop-als-therapy-prosetin-brain-disease/">created ProJenX</a> last year in collaboration with Columbia University researchers and <a href="https://projectals.org/">Project ALS</a>, an organization that identifies and funds research for potential <a href="https://alsnewstoday.com/als-treatment/">ALS therapies</a>.
&#8220;We are focused on accelerating the development of prosetin to meet the critical need for new treatments for people <a href="https://alsnewstoday.com/living-with-als-2/">living with ALS</a>,&#8221; Stan Abel, ProJenX president and CEO, said in a <a href="https://www.prnewswire.com/news-releases/projenx-announces-initial-closing-of-15m-series-a-financing-and-appoints-rick-hartz-to-board-of-directors-301975260.html">company press release</a>.
[recommended-reading id=82931]
<h3>Oral prosetin designed to reach brain and reverse stress in nerve cells</h3>
In ALS, certain proteins abnormally fold and form toxic clumps in nerve cells. This causes stress in certain cellular compartments, including in the endoplasmic reticulum (ER), a cell organelle involved in protein production, modification, and transport, and ultimately results in nerve cell damage and death.
ProJenX&#8217;s lead compound, prosetin, is a first-in-class oral therapy designed to reach the brain and reverse ER stress in nerve cells. It works by inhibiting the function of a protein called mitogen-activated protein kinase kinase kinase kinase (MAP4K).
Project ALS and Columbia University researchers developed the therapy candidate, which was found to <a href="https://www.projectals.org/wp-content/uploads/2019/10/Development-of-MAP4-Kinase-Inhibitors-as-Motor-Neuron-Protecting-Agents.pdf">potently block MAP4K</a> in an ALS animal model, lower inflammation, and improve nerve cell survival.
Soon after ProJenX&#8217;s launch, a first-in-human <a href="https://clinicaltrials.gov/study/NCT05279755">Phase 1 clinical trial (NCT05279755)</a> began <a href="https://alsnewstoday.com/news/dosing-begins-phase-1-clinical-trial-prosetin-als/">testing prosetin</a> in people with ALS and in healthy volunteers. In the trial&#8217;s initial two parts, researchers are assessing the safety, tolerability, and pharmacokinetics of single and ascending doses of the compound versus a placebo in healthy participants. Pharmacokinetics refers to the movement of drug into, through, and out of the body.
After those portions are completed, the same measures will be evaluated in ALS patients. The company is planning to launch this third portion in people with ALS early next year.
&#8220;We are encouraged by the therapeutic window emerging from our phase 1 trial and ongoing nonclinical studies and are looking forward to advancing prosetin to people living with ALS in early 2024,&#8221; Abel said.
The overall aim of the study is to establish the optimal prosetin dose and gather biomarker data that help inform the design of upcoming trials.
[recommended-reading id=83944]
<h3>Prosetin named orphan drug by FDA in 2020</h3>
Prosetin was <a href="https://alsnewstoday.com/news/prosetin-granted-orphan-drug-designation-for-als/">granted orphan drug designation</a> by the U.S. Food and Drug Administration for ALS in 2020. Such a designation offers a range of incentives to developers of medications for rare disorders, including seven years of market exclusivity if an approval is ultimately granted.
ProJenX also announced the board appointment of Rick Hartz, senior vice president, global pharma and human health business development at Merck (known as MSD outside North America).
&#8220;I am excited to join the ProJenX Board and contribute to its mission to advance promising therapies for ALS and other neurodegenerative diseases,&#8221; Hartz said. &#8220;With an orphan drug designation, prosetin is poised to advance toward a global pivotal trial next year and I look forward to working with my fellow board members and the ProJenX team to help guide the growth of the company.&#8221;

Tag: drug development

Coya aligns with FDA on plans for developing COYA 302 for ALS

ProJenX receives $15M in funding to further develop prosetin for ALS