People with Duchenne muscular dystrophy (DMD) have gained a powerful new ally in CureDuchenne Ventures, a nonprofit organization dedicated to funding drug discovery and development for the rare disease.

CureDuchenne’s first beneficiary will be Lexicon Pharmaceuticals Inc., of The Woodlands, Texas, for its drug candidate, LX2931. CureDuchenne has committed to raise $5 million for clinical development of the drug in DMD. The disease affects 1 out of 3,500 boys, and there is currently no treatment.

CureDuchenne already has raised more than $11 million, funding seven research projects.

According to Debra Miller, CEO of CureDuchenne, the nonprofit organization has had a successful venture philanthropy model for the past 10 years in which it has partnered with the biotech industry to bring early stage research to clinical stage.

“Now the picture has gotten much bigger. As we’re moving research projects into clinical trials, it has become much more expensive,” Miller told BioWorld Today.

CureDuchenne Ventures will open the funding opportunities to other philanthropists and investors. “This is our coming-out party,” Miller said.

According to Lexicon, LX2931 is an inhibitor of sphingosine-1-phosphate lyase (S1P lyase), which is part of the inflammatory pathways of the body. The company has been developing the compound as a potential treatment for an array of autoimmune diseases such as rheumatoid arthritis.

In a 12-week, randomized, double-blind, placebo-controlled Phase IIa study, patients treated with 150 mg once daily of LX2031 showed an improvement in the percentage of patients with rheumatoid arthritis achieving a response at week 12, 60 percent vs. 49 percent for placebo. But the data also showed that patients treated with 70-mg or 110-mg once-daily dosages did not improve at week 12, 44 percent and 41 percent, respectively, vs. placebo.

S1P lyase is thought to have significance in muscular dystrophy as well.

“There’s a compelling body of evidence in a number of species that indicates this pathway the drug works on may be involved in muscle regeneration,” said Michael Kelly, chief scientific officer of CureDuchenne. He added that Lexicon is the only company with a drug in clinical development targeting that pathway. “We think it’s a great opportunity both for Lexicon and Duchenne.”

Kelly said the $5 million that CureDuchenne Ventures intends to raise will take Lexicon through “the next few years” of research, including proof-of-concept and Phase II studies in DMD.

CureDuchenne’s research portfolio includes Prosensa Holding NV’s exon skipping drugs, PTC Therapeutics Inc.’s ataluren, AVI-Biopharma’s AVI-4658 and some studies involving FDA-approved drugs and stem cells.

Glaxosmithkline plc is developing a drug that skips exon 51 that CureDuchenne said would be applicable to approximately 15 percent of Duchenne patients, and Prosensa is testing another compound for skipping exon 44 that, if successful, will restore dystrophin to muscle cells.

Now CureDuchenne would like to do similar work on “grander scales,” according to Kelly. “We realize there’s going to be different approaches. . . . That’s why we’ve got a broader platform to attract investors,” he said.

In addition to LX2931 , Lexicon’s research portfolio includes LX4211 , in Type I and Type II diabetes; telotristat etiprate (LX1032) in carcinoid syndrome and ulcerative colitis; LX1033 in irritable bowel syndrome; and LX7101 in glaucoma.

LX4211 is designed to inhibit sodium-dependent glucose transporter 1 (SGLT1) and sodium-dependent glucose transporter 2 (SGLT1). SGLT1 is associated with glucose and galactose absorption in the gastrointestinal tract, and SGLT2 allows glucose reabsorption by the kidney.

Lexicon found that the compound produced a statistically significant in fasting plasma glucose in a Phase IIa study in patients with Type II diabetes, as well as decreases in postprandial glucose and hemoglobin A1c (HbA1c).

Patients had improvements in metabolic and cardiovascular benchmarks such as weight, blood pressure and triglycerides, and had a favorable safety profile.

A separate study showed that a single dose of LX4211 increased circulating levels of GLP-1 and PYY, which are viewed as important regulators of glycemic and appetite control.

Lexicon received fast-track status from the FDA for LX1033 for diarrhea-predominant irritable bowel syndrome. That compound is in Phase II testing.

In October 2012, Lexicon reported positive results for telotristat etiprate in a Phase II trial in metastatic carcinoid syndrome. The primary efficacy endpoint of that trial was reduction of bowel movements from baseline. Patients had a 46.4 percent median reduction from baseline at week 12, with the number of daily bowel movements steadily decreasing over time. (See BioWorld Today, Oct. 15, 2012.)

Lexicon’s last financing event was a public offering of 17.5 million shares of common stock at $2.25 per share, for net proceeds of about $37.2 million.