Samumed and United Therapeutics Partner to Advance Potential IPF Inhalation Therapy, SM04646

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by Diogo Pinto |

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Samumed and United Therapeutics announced an exclusive license agreement for North American rights to Samumed’s investigational therapy SM04646 to treat idiopathic pulmonary fibrosis (IPF).

Under the agreement, United Therapeutics will pay Samumed $10 million up-front, plus up to $340 million in developmental milestones in addition to other royalties.

“We are excited to partner with United Therapeutics because of its culture of integrity, level of commitment, and expertise in pulmonology and drug-device combinations,” Osman Kibar, PhD, chief executive officer of Samumed, said in a press release. “We look forward to delivering what could be a first-in-class, disease-modifying treatment option for IPF patients.”

SM04646 is a small molecule inhibitor of the Wnt signaling pathway, which was previously found to be involved in IPF development. The molecule is believed to reduce the activity of genes associated with fibrosis. It is administered as an inhaled aerosol.

Preclinical studies on SM04646 confirmed its anti-fibrotic properties, including in primary human lung fibroblasts, the key cells promoting fibrosis, and a mouse model of human IPF, the bleomycin-induced PF model.

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Samumed opened a Phase 1 clinical trial assessing the safety and tolerability of SM04646 in healthy individuals (ACTRN12615001349538) in 2015, and is now conducting another in people with mild to moderate IPF (ACTRN12617000854336). Both studies take place in Australia.

In healthy individuals, SM04646 was tested to date in 17 people, ages 18 to 50, at four increasing doses  (0.7, 2, 7, and 20 mg).

Results showed the nebulized inhalation solution was well-tolerated, with no serious adverse events at any of the doses. These results will be compared to those obtained in the IPF patients.

In this second trial, patients will be treated with SM04646 at escalating doses (2, 7, and 20 mg) for 28 days, then followed for safety for about another 30 days. Other primary trial goals include changes in vital signs (blood pressure, respiratory rate) and lung function (assessed by spirometry).

The U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to SM04646 as a potential IPF treatment, a status that can speed its development.

Under the recent agreement, Lung Biotechnology — a subsidiary of United Therapeutics — will  conduct and fund all further development, regulatory, and commercialization activities within the U.S. and Canada.

“I’ve been impressed with Samumed’s exhaustive work on the pleiotropic Wnt pathway,” said Martine Rothblatt, PhD, chairman and chief executive officer of Lung Biotechnology. “Our months of due diligence have energized our belief on SM04646’s fibrosis-modulating properties, and hence unique potential for addressing IPF.”

Samumed will continue to advance SM04646 for all markets outside of North America.