Novus Acquires Anelixis, Plans to Move AT-1501 Into Phase 2 Trial for ALS

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by Forest Ray PhD |

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CNM-Au8 trial update

Novus Therapeutics has acquired Anelixis Therapeutics and its portfolio, including the antibody AT-1501, a potential treatment for amyotrophic lateral sclerosis (ALS) that the company is planning to move into a Phase 2 clinical trial for ALS and other diseases.

AT-1501 is an antibody against a protein called CD40 ligand (CD40L), found on the surface of some immune cells and involved in regulating the immune response, which can trigger inflammation in the spinal cord. The CD40L pathway is reported to be overactive in ALS patients.

“We are excited about AT-1501 and the potential to develop and commercialize the next generation anti-CD40L antibody, a well-validated target with broad therapeutic possibilities,” Keith A. Katkin, chairman of Novus’ board of directors, said in a press release.

The potential treatment is a humanized antibody targeting CD40L, meaning that the antibody comes from a non-human source but has been modified for use in patients. In addition to ALS, it is being developed to possibly treat people undergoing organ and cell transplant, and those with autoimmune diseases, and other neurodegenerative diseases.

AT-1501 aims to slow the onset of ALS or symptom progression by blocking or delaying the activation of CD40L’s inflammatory response. In animal models of the disease, it was seen to significantly delay disease onset, increase survival, and improve body weight (meaning it improved muscle health), leading the U.S. Food and Drug Administration (FDA) to name it an orphan drug for ALS.

The compound completed Phase 1 testing in healthy volunteers and in eight ALS patients last year. Results showed AT-1501 to be well-tolerated at all doses tested, and to behave as expected inside the body.

The upcoming Phase 2 trial (NCT04322149) in 54 adults with ALS is set for 12 sites within the United States. Participants will all receive ascending doses of AT-1501 intravenously (directly into the vein) every two weeks for a total of 11 weeks.

It has not yet begun recruiting, but is expected to open shortly. Contact information can be found here.

Novus also entered an agreement  to sell certain stock — called non-voting convertible preferred stock — to a group of investors led by BVF Partners.

This sale is expected to raise about $108 million, which the company will use to support the upcoming ALS clinical trial, and Phase 2 studies in three other indications: people undergoing kidney (renal) and islet cell transplants and those with nephritis, an autoimmune disease linked to lupus.

“The activation of CD40/CD40L signaling is critical to mediating antibody and cellular inflammatory response,” said Steven Perrin, PhD, founder and CEO of Anelixis and now Novus’ president and chief scientific officer.

“We are developing antibodies to inhibit the activation of this pathway with the hope of offering new treatment modalities for people living with conditions such as autoimmune nephritis and ALS, or those requiring a potentially life-saving transplant,” he added.

Novus also appointed several new people to leadership roles within the company, including former Anelixis personnel. In addition to Perrin, Walter Ogier, former chairman of the board for Anelixis, will join Novus’ board of directors.