Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) and AnaptysBio, Inc. (NASDAQ: ANAB) have announced an exclusive global license agreement for the development and commercialization of imsidolimab, an IL-36R antagonist MAb, following the successful completion of two registration-enabling global phase 3 trials, Gemini-1 and Gemini-2. These trials evaluated the safety and efficacy of imsidolimab in patients with generalized pustular psoriasis (GPP), a rare and potentially life-threatening skin disorder caused by mutations in the IL36RN gene.
The agreement entails an upfront payment of $10 million and a $5 million payment for existing drug supply from Vanda to Anaptys, with additional potential payments of up to $35 million for future regulatory approval and sales milestones. Anaptys is also set to receive a 10% royalty on net sales.
Following the agreement, Vanda is preparing BLA and MAA applications for the US and EU, with a focus on commercialization. Imsidolimab, a fully humanized IgG4 antibody, is expected to provide regulatory and patent exclusivity into the late 2030s.
The positive phase 3 data from the Gemini-1 and Gemini-2 trials showed that patients achieved rapid disease clearance through week 4 after a single dose of infused imsidolimab, maintaining clear to almost clear skin for at least 24 weeks, with no clinically meaningful safety signals. This represents a promising new treatment option for patients with GPP, a severely debilitating and sometimes life-threatening skin disease.
The Gemini-1 trial, conducted with 45 patients, demonstrated that 53% of patients who received a single dose of 750mg IV imsidolimab achieved a GPP physician global assessment (GPPPGA) score of 0/1 (clear or almost clear skin) at week 4, compared to 13% of patients on placebo. In the subsequent Gemini-2 trial, patients who were re-randomized to monthly maintenance dosing of 200mg SC imsidolimab maintained a GPPPGA score of 0/1, demonstrating the sustained efficacy of the treatment.
The safety and tolerability profile of imsidolimab was favorable, with no treatment-related serious adverse events (SAEs) or SAEs leading to discontinuation reported in imsidolimab-treated patients.
GPP prevalence estimates in the general population vary considerably, between 1.76 and 124 patients per million persons worldwide, with the majority of GPP cases caused by genetic variants in IL36RN. Loss-of-function mutations in IL36RN are mostly missense mutations in a recessive pattern, resulting in unrestricted IL-36 activity.
Vanda Pharmaceuticals Inc. is looking to add imsidolimab to its product portfolio for rare orphan disorders and explore its potential in the treatment of additional inflammatory conditions where IL-36 homeostatic balance is dysregulated. This aligns with Vanda's rare disease expertise in the US and EU, as well as its anti-inflammatory portfolio that includes Ponvory® for multiple sclerosis, psoriasis, and ulcerative colitis.
AnaptysBio, Inc., on the other hand, expressed confidence in Vanda as an ideal partner for imsidolimab, citing Vanda's strong regulatory and commercial capabilities in the US and Europe, evidenced by successful recent launches in specialty and rare diseases, and their commitment to invest in label expansion across their therapeutic portfolio, including their growing presence in inflammatory disease.
This exclusive global license agreement marks a significant step forward in addressing the unmet medical needs of patients with GPP, offering hope for a differentiated therapeutic option in the near future. Following these announcements, the company's shares moved -5.9%, and are now trading at a price of $16.88. Check out the company's full 8-K submission here.
