Pipeline & science

CX11 is an investigational oral small molecule GLP-1 RA designed to address cardiometabolic conditions. GLP-1 RAs have been shown to lower bodyweight, improve insulin sensitivity, and reduce glucose and overall appetite. CX11 aims to overcome limitations of current injectable GLP-1 RAs by offering convenient, once-daily oral administration, and weight reduction comparable to injectable GLP1 RAs. CX11 presents favorable tolerability results, scalability and accessibility as a small molecule product candidate.

In December 2024, CORXEL acquired the worldwide (excluding Greater China) rights to CX11 from Vincentage.

Clinical data from Vincentage’s Phase 2 trial in China demonstrated competitive weight loss with a favorable safety and tolerability profile. CX11 is currently being advanced in a U.S. Phase 2 trial in Obesity, a Global Phase 2 trial in T2DM conducted by CORXEL and being evaluated in a Phase 3 registrational trial in China by Vincentage, positioning it as a potential best-in-class oral therapy for management of obesity and overweight conditions, and T2DM.

JX10 (formerly BIIB131) is an investigational drug for acute ischemic stroke (AIS). Its proposed mechanism of action includes both thrombolytic and anti-inflammatory activities. By restoring critical blood flow following acute stroke, it may benefit more patients over current standard of care by extending the otherwise short treatment window.

JX10 is one of the SMTP compounds with (1) pro-thrombolytic, (2) anti-inflammatory, and (3) antioxidative activities.

A Phase 2a study including 90 participants in Japan established preliminary safety and efficacy in AIS patients with late presentations up to 12 hours from symptom onset. This study achieved the primary endpoints for both safety and efficacy as summarized below.

This was a multicenter, single-dose, double-blind, randomized, placebo-controlled, dose-escalation study conducted in Japan under the sponsorship of TMS Co. The study enrolled patients with acute ischemic stroke ineligible for the therapies with tissue-type plasminogen activator (t-PA) or thrombectomy. JX10 (at a dose of 1, 3, or 6 mg/kg) and placebo were administered within 12 hours of the symptom onset to 52 and 38 patients, respectively.

In January 2024, CORXEL acquired the global rights for JX10, excluding Japan, from Biogen. TMS regains Japan rights of JX10 and will join forces with CORXEL to expeditiously develop and launch JX10, facilitated by the formation of a Joint Development Committee. CORXEL is now conducting a global registrational trial to further evaluate the efficacy and safety of intravenous ("IV") JX10 in AIS participants between 4.5 and 24 hours LKW. 

CX12 (amylin), an oral small molecule amylin RA drug candidate, is designed to act through amylin receptors to regulate satiety, slow gastric emptying, and suppress post-prandial glucagon. Injectable amylin receptor agonists have demonstrated significant body weight reduction results. Moreover, combining amylin agonism with GLP-1 receptor activation has shown to produce synergistic effects on body weight loss and metabolic improvement.

Oral small-molecule amylin RAs offer several key advantages. They enable convenient dosing, improving patient adherence and accessibility for long-term obesity management. Unlike peptides, small molecules have better stability, lower manufacturing costs, and do not require cold-chain storage. Additionally, their pharmacokinetic properties can be optimized for precise receptor selectivity and duration of action, with the goal of yielding clinically meaningful weight loss while minimizing gastrointestinal side effects.

CX12 is currently in preclinical stage, with ongoing chemical optimization aimed at enhancing its potency, selectivity and drug-like properties. 

LNZ100 (aceclidine) eye drops are being developed by LENZ Therapeutics. Aceclidine is a small molecule acetylcholine receptor agonist that causes pupil contraction, or miosis, creating a pinhole effect that improves near vision. Studies have shown that aceclidine’s mechanism of action (MOA) is well positioned to create a pinhole pupil effect while avoiding the impairment of distance vision called myopic shift. Aceclidine’s unique pupil selective MOA, in which miosis is decoupled from myopic shift, is expected to allow it to target a broad patient population.

CORXEL acquired the Greater China rights for the development and commercialization of the products in April 2022. In August 2024, CORXEL announced that the investigational ophthalmic products, LNZ100 (aceclidine) eye drops, have completed patient enrollment in a Phase 3 clinical study in China for the treatment of presbyopia.

For more information, visit: https://lenz-tx.com/