Alexion Pharmaceuticals Inc. is looking to substantially expand its product portfolio by potentially launching at least 10 drugs by 2023, according to CEO Ludwig Hantson.
The expansion of the company's clinical-stage pipeline is a mix of business development and internal research, Hantson told investors on the company's Oct. 23 third-quarter 2019 earnings call.
Business development efforts
He said the company expects to add four new assets to its pipeline, including two drugs that are in late-stage development. "We continue to look to add additional rare diseases to our portfolio through further disciplined business development and internal research efforts," Hantson said in the earnings call.
Alexion's $930 million acquisition of Achillion Pharmaceuticals Inc. will add two clinical-stage oral Factor D inhibitors, ACH-4471 and ACH-5228, to the company's pipeline that can be advanced to treat multiple rare diseases. The deal, seen as "high risk, high reward" by analysts, is expected to close by the first half of 2020, subject to closing conditions and necessary regulatory approvals.
Currently, ACH-4471 is being studied in two mid-stage trials as a treatment for a rare blood disorder, paroxysmal nocturnal hemoglobinuria, and a rare kidney condition called C3 glomerulopathy.
On the conference call, Executive Vice President and Head of Research and Development John Orloff said the Achillion deal will provide Alexion the opportunity to expand into new treatments areas, including diseases of the eyes and visual system.
Alexion also agreed to co-develop and commercialize Stealth BioTherapeutics Corp.'s genetic disorder drug elamipretide for an upfront payment of $30 million in October. The company expects results from the phase 3 trial of the drug in patients with primary mitochondrial myopathy, or PMM, during the first quarter of 2020. Currently, there are no approved treatments for the disease, which is characterized by debilitating skeletal muscle weakness, chronic fatigue and exercise intolerance.
Other potential indications for elamipretide include Barth syndrome, and eye diseases such as Leber's hereditary optic neuropathy and geographic atrophy associated with dry age-related macular degeneration.
Additionally, Alexion acquired an exclusive license for Eidos Therapeutics Inc.'s rare disease medicine AG10 in Japan for $25 million. Alexion will develop AG10 in Japan, while San Francisco-based Eidos is evaluating the drug in a phase 3 study in the U.S. and Europe to treat ATTR cardiomyopathy, a condition where the protein accumulates in the heart.
Hantson said the company's ongoing business development efforts, which comprise 11 deals over a two-year period, have added 7 clinical-stage products to its pipeline.
Alexion expects to complete enrollment of patients with Wilson disease in the phase 3 trial of ALXN1840 in early 2020. Wilson disease is a rare genetic disorder, in which the body stores excess copper particularly in the liver, brain and corneas of the eyes.
Additionally, the biotechnology company plans to conduct a phase 2/3 trial for CAEL-101 as a treatment for a rare disorder called light chain amyloidosis. Orloff said the trial will start after a review with regulators.
Alexion plans to initiate a phase 2 study of ALXN1830 to treat warm autoimmune hemolytic anemia, an autoimmune disorder, early next year.
Meanwhile, Ultomiris, which is touted by Alexion as an improved version of its older drug Soliris, continued to boost revenue for the company and brought in $90 million in the third quarter, up from $54 million in the second quarter of the year.
The rare disease therapy, which is approved to treat paroxysmal nocturnal hemoglobinuria, is administered every eight weeks compared to one injection of Soliris every two weeks.