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<a href="https://myrotx.com/">Myrobalan Therapeutics</a> will use a $400,000 grant from the <a href="https://www.als.org/">ALS Association</a> — along with $24 million in new financing — to help advance the development of its oral CSF1 receptor (CSF-1R) inhibitor program, intended to reduce neuroinflammation and promote nerve repair in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The ALS Association grant was awarded via the nonprofit&#8217;s Lawrence and Isabel Barnett Drug Development Program, which supports the preclinical evaluation of new and repurposed therapies for the neurodegenerative disorder. It follows on the heels of a <a href="https://myrotx.com/news-events/myrobalan-therapeutics-completes-series-a-financing-of-24-million-to-develop-potentially-first-in-class-cns-therapeutics-with-restorative-potential/">Series A funding by Myrobalan</a> last month that raised nearly a quarter of a billion dollars to further the development of the company&#8217;s treatment program for people with ALS.
Myrobalan&#8217;s CSF-1R inhibitor program was specifically designed for use in diseases of the central nervous system, or CNS, which comprises the brain and spinal cord. The company is advancing the clinical biomarker strategy toward the first clinical trial of its CSF-1R inhibitor, which is expected to start next year, according to a <a href="https://www.businesswire.com/news/home/20240206866405/en/Myrobalan-Therapeutics-Receives-a-Grant-from-the-ALS-Association-to-Advance-its-CSF1R-Inhibitor-Program">Myrobalan press release</a>. The new monies will keep the program on track, according to the release, which said Myrobalan was &#8220;very pleased to receive this grant from the ALS Association.&#8221;
“This recognition affirms Myrobalan’s commitment to CNS drug development and empowers us to enhance our development efforts with respect to CSF-1R and ALS,” said Jing Wang, PhD, the company&#8217;s CEO and co-founder.
&#8220;Ultimately, our goal is to make a CSF-1R inhibitor therapy accessible to all patients grappling with ALS,&#8221; Wang said.
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<h3>Myrobalan aiming for 1st clinical trial of CSF-1R inhibitor next year</h3>
ALS is characterized by the progressive death of motor neurons, the nerve cells that control muscle movement. Loss of these neurons leads to <a href="https://alsnewstoday.com/symptoms-of-als/">symptoms</a> like muscle weakness and wasting, with a progressively worsening impact on a patient&#8217;s ability to perform tasks of daily living.
Although <a href="https://alsnewstoday.com/causes-of-als/">its causes</a> are largely unknown, several bodily systems are dysregulated in ALS, contributing to the disease&#8217;s onset and progression. Known ALS-related processes include inflammation of the brain — neuroinflammation — and demyelination, or the loss of the myelin sheath that protects nerve fibers and helps them send signals efficiently.
Causing these processes are alterations in microglia, the CNS’ resident immune cells, and myelin-producing cells called oligodendrocytes.
The company is working on oral, small molecule CSF-1R inhibitors. Myrobalan&#8217;s hypothesis is that intermittent treatment with CSF-1R inhibitors depletes microglia — whose ability to adapt is then impaired, thereby reducing inflammation and allowing remyelination, or the formation of new myelin.
[quote=&#8221;center&#8221;]We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies.[/quote]
According to Myrobalan, its CSF-1R inhibitor program was specifically designed for central nervous system diseases, and has shown potency, selectivity, and favorable CNS distribution in preclinical studies.
The ALS Association award, granted to scientists worldwide, is part of the nonprofit&#8217;s philanthropic funding.
“We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies,” said Kuldip Dave, PhD, ALS Association’s senior vice president of research.
“Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it,” Dave said.
In addition to ALS, Myrobalan plans to develop its CSF-1R inhibitors for other neurodegenerative indications, such as <a href="https://multiplesclerosisnewstoday.com/multiple-sclerosis-overview/">multiple sclerosis</a> and <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer’s disease</a>. The company also is developing other compounds to reduce microglia-driven neuroinflammation and improve remyelination.

$400K ALS Association grant will advance CSF-1R inhibitor program

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>.
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<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.
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<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: financing

$400K ALS Association grant will advance CSF-1R inhibitor program

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