A biopharmaceutical company studying ALS revealed a recent positive study that is giving hope to well-known Orange County executive Augie Nieto.
“As someone who has lived with ALS for nearly two decades, it is gratifying to see the positive results of this research,” Nieto said.
“We will not stop until a cure for ALS is a reality.”
Irvine-based Eledon Pharmaceuticals Inc. (Nasdaq: ELDN) on May 31 revealed “positive topline results” from a Phase 2a trial that demonstrated safety, target engagement and biomarker responses in patients living with ALS, which stands for Amyotrophic Lateral Sclerosis.
The study enrolled 54 adults with ALS at 13 treatment sites in the United States and Canada. Nieto wasn’t part of the study.
“The data was some of the best data in companies that I’ve been with,” Eledon Chief Executive David-Alexandre Gros told the Business Journal.
“The data itself is new and exciting. The ALS community was excited to see it as well.”
Eledon is currently working to determine what its next trial will look like.
Lou Gehrig
ALS is sometimes referred to as Lou Gehrig’s disease, named after the famed New York Yankee slugger who died of it at the age of 37.
It’s a progressive, neurodegenerative disease that affects nerve cells in the brain and spinal cord, causing muscle weakness that leads to paralysis. Symptoms include loss of ability to speak, swallow, walk, grasp objects or breath independently. There is no cure.
Some 30,000 people in the United States suffer from ALS, including 5,000 new cases diagnosed annually. About 600,000 worldwide have the disease. The average age at the time of the diagnosis is 55.
About 90% of ALS cases are sporadic, developing for unknown reasons while 10% of cases are inherited from a parent.
Most ALS patients typically live only three to five years after being diagnosed.
“The unmet need is incredibly significant,” said Steve Perrin, Eledon’s president and chief medical officer.
“A lot of people lose their ability to walk and speak. It’s a big burden on the person and their families.”
Medical issues now confine Nieto to a motorized wheelchair that allows him to communicate via computer.
Augie’s Quest
In 2005, Nieto was diagnosed with the illness. Previously, he was best known for developing Life Fitness, a Rosemont, Ill.-based maker of products for gyms and health clubs; he also invented the first workout machine to incorporate a computer. Life Fitness is reported to do over $1 billion in sales annually.
He founded the firm in the 1970s, and ran the company for decades. He stepped down from the firm in 2000, a few years after it was sold to Brunswick Corp. in 1997. In 2019, Nieto returned to Life Fitness, now privately held, as an investor and board member.
Shortly after his ALS diagnosis, Nieto and his wife, Lynne, started Augie’s Quest to Cure ALS, which has raised more than $160 million to fund research to find a cure for the disease.
“Augie decided he wasn’t just going to walk away,” Perrin recalled. “He wanted to try to help the community.”
Nieto has been able to survive because he falls into “a small group of people” who has paid a significant amount of detail to his healthcare, Perrin said.
Limited Research
Since ALS affects relatively few people in the U.S., research has been small.
Nieto decided to fight the dreaded disease, reaching out in 2006 to Perrin, who at that time was associate director of molecular profiling for Biogen Inc.
“I told him it would cost $100 million to identify one potential treatment and it’s a risky endeavor,” Perrin recalled. “Augie grabbed me by the shoulder and said ‘I’ll help you.’”
Perrin has worked since 2007 at the ALS Therapy Development Institute, which was founded by James Heywood after his younger brother Stephen was diagnosed with ALS.
The family’s battle was described in Pulitzer Prize winner Jonathan Weiner’s “His Brother’s Keeper,” as well as the movie “So Much So Fast.”
Nieto is currently chairman of the institute, which is the world’s largest research organization dedicated to ALS. Along with Life Fitness, Nieto is also currently active in business circles as an adviser to private equity firm North Castle Partners and is on the boards of Curves, Jenny Craig and Hyrdomassage.
Corporate Changes
Eledon has undergone many changes in the past two years.
In 2020, it hired as CEO Gros, who has a long history in healthcare investments, including Sanofi S.A., Merrill Lynch and McKinsey. Eledon raised $108 million in a private placement and had a 1-for-18 reverse stock split.
Gros in early 2021 changed the name of the company from Novus Therapeutics Inc., which had failed to make effective drugs for ear infections, to Eledon.
Eledon in 2020 acquired Anelixis Therapeutics, which was developing a promising drug to treat ALS. Nieto was prominent in the creation in 2013 of Anelixis, whose CEO was Perrin.
1 of 300
Eledon is targeting the CD40 Ligand pathway to develop potential treatments for persons requiring an organ or cell-based transplant, living with autoimmune disease, or living with ALS.
Out of 300 drugs researched for ALS, only one “worked quite profoundly,” Perrin said.
Originally discovered by the ALS Therapy Development Institute and now called tegoprubart, that one standout is an anti-CD40L antibody believed to work by decreasing inflammation in skeletal muscle as well as in the brain and spinal cord that may drive ALS progression.
After the Phase 1 study validated the drug’s safety, the Phase 2a trial showed 23 of 32 bio-inflammatory bio markers were “knocked down,” Gros said.
“We’re validating our biology,” Gros said. “By doing so, we hit targets. We moved bio markers. We decreased inflammation and we showed signs of clinical benefits as a result.”
Tegoprubart, which could become the most effective treatment for ALS, has received orphan drug designation from the U.S. Food and Drug Administration.
The FDA grants orphan designation status to drugs and biologics that are intended to treat, diagnose or prevent rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.
Last week, the Canadian government granted approval for an ALS treatment to Amylyx Pharmaceuticals Inc. (Nasdaq: AMLX), a biopharmaceutical research company based in Cambridge, Mass. Amylyx said the approval for its treatment, called Albrioza, was the first marketing approval worldwide.
“Clinical data demonstrated a statistically significant and clinically meaningful benefit in functional outcomes for people with ALS taking Albrioza compared to people taking placebo, either as a stand-alone therapy or when added to existing treatments for ALS,” Amylyx said in a statement.
In the trading session after the announcement, shares climbed 25%; the company had a $935 million market cap at press time.
Stock Decline
Wall Street hasn’t been as impressed with Eledon. Its stock price has steadily fallen from about $26 in October 2020. Even in the trading session after the latest news about its clinical trial results, the stock declined 10% to around $3.19.
“It’s hard to know why” the shares fell after the announcement, Gros said. “It’s been a tough year for the market, companies in biotech.”
The company’s market cap is close to $50 million, which Gros pointed out is less than the amount of cash it has on hand, $76.7 million as of March 31. Eledon currently says it has enough cash to survive until 2024.
“We’re not focused on a single day” in the stock market, Gros said.
“If we focus on what’s doing right for patients, then over time everything else will fall into place.”
