RARE Daily

Amolyt Aspires to Be a Leading Rare Disease Company

August 27, 2020

Thierry Abribat, the founder and CEO of Amolyt Pharma, had already founded and sold two rare disease drug companies—Alize Pharma and Alize Pharma 2—when he started his latest company.

The first two companies were formed to develop single assets to proof-of-concept and then sold to larger biotech/pharmaceutical companies. Millendo Therapeutics acquired Alize Pharma and Jazz Pharmaceuticals acquired Alize Pharma 2. The third time around, Abribat wanted to do something different.

“I wanted to create my dream biotech by going deeper and having the opportunity to create a portfolio in rare endocrine diseases and move them forward to approval,” Abribat said.

To do that, he decided to rebrand what began as Alize Pharma 3 to reflect his ambitious growth plan.

The new name, Amolyt Pharma, reflects his desire to build a leading company in rare diseases with operations in both Europe and the United States and a portfolio of products targeting significant unmet medical needs.

The name Amolyt, Abribat says, is derived from ammolite, a rare gemstone originating from living organisms.

“Its spiral structure reflects the journey that we envision a patient, who is using our therapeutic peptides, will take from disease to recovery and to the return of a fully functioning life,” says Abribat. “Ultimately, the rebrand highlights our commitment to provide innovative treatments that safely and effectively restore balance.”

The rebranding comes less than one year after raising $75 million (€67 million) in series A financing from a strong international syndicate, establishing a presence in Boston with a management team experienced in bringing endocrine and metabolic drugs to approval, and an initial portfolio of two promising preclinical therapeutic peptides to treat rare endocrine diseases.

Abribat says the way his team works is to look at a disease and try to find the best solution. “We don’t have a particular technology we are pushing,” he said. “We are just trying to find the best solution for each disease we are treating.”

The first disease Amolyt is focusing on is hypoparathyroidism, a rare endocrine disorder characterized by a deficiency in parathyroid hormone (PTH) that results in dysregulation of calcium and phosphorus, two essential minerals in the body. It affects about 80,000 people in the United States and about 110,000 people in Europe. Only 25 percent of hypoparathyroidism cases are due to genetic conditions or autoimmune disorders, with the majority of cases resulting from thyroid surgery, especially due to cancer.

These patients have a hard time regulating their blood calcium. Calcium is very important for health and if not treated, patients can die. Most of these patients are treated with calcium and vitamin D supplementation. But hypoparathyroidism is more than just calcium, says Abribat, it’s also associated with cognitive and neurological symptoms and affects quality of life. And over the long term, it can be toxic to the kidneys.

Abribat believes the best approach is to restore the PTH that is missing in these patients, and to do that, it is developing a parathyroid hormone analog, AZP-3601, specifically designed to restore PTH in patients suffering from hypoparathyroidism.

Amolyt is collaborating with Massachusetts General Hospital, which is where AZP-3601 was discovered. MGH scientists have found that the PTH receptor has several conformations and they have engineered a PTH analog to have high affinity to the conformation of a receptor that could potentially regulate serum calcium. Amolyt believes this approach will help normalize calcium levels, alleviate symptoms and prevent kidney disease while preserving bone integrity.

The company plans to begin its first clinical study in healthy volunteers in the fall of 2020 to see what effect AZP-3601 will have on calcium levels. Abribat says they should be able to have some early signals by the end of the year.

Amolyt is also working on other programs that are earlier in development, including AZP-3404, a peptide derived from insulin-like growth factor binding protein 2 (IGFBP2) that regulates fat and glucose metabolism.

“We are working to find the best target indication for this peptide,” says Abribat. The company is also looking for an additional program to build out its portfolio in rare endocrine diseases.

“I have been working in rare disease for a long time. To me it’s important to continue working in the space,” Abribat says. “These patients are really in need of new treatments so its important that we stay focused in the rare disease space. As a small company we can be agile in helping these patients.”

Patient advocacy and the patient voice are very important to Amolyt, and the company is working to establish a long-term relationship with the patient organization for hypoparathyroidism in the United States.

“It’s a fantastic ecosystem to work in rare disease where you have the patient focused group, you have the investigators, and the biotech industry,” said Aribat. “It’s a good partnership and I hope we will be able to bring something useful for those patients.”

Photo: Thierry Abribat, founder and CEO of Amolyt Pharma

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