Pipeline & science

Pipeline of nine novel assets in late-stage clinical development and one in pre-clinical stage

Cardiovascular

Asset
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approval
  • China studies conducted by JIXING
  • Global studies conducted by partner

Aficamten

(formerly CK274, cardiac myosin inhibitor)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
China cohort of Ph3 SEQUOIA-HCM got positive results
Ph3 SEQUOIA-HCM got positive results,Ph3 MAPLE-HCM started
Preclinical
Phase 1
Phase 2
Phase 3
Approval
Ph3 CTA Approved
Ph3 is open to enrollment
Preclinical
Phase 1
Phase 2
Phase 3
Approval
To be initiated
To be initiated

Aficamten

Aficamten is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties and as may translate into next-in-class potential in clinical development. Aficamten was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with hypertrophic cardiomyopathy (HCM). In preclinical models, aficamten reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state.

 The development program for aficamten is assessing its potential as a treatment that improves exercise capacity and relieves symptoms in patients with HCM as well as its potential long-term effects on cardiac structure and function. Aficamten was evaluated in SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), a pivotal Phase 3 clinical trial in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Aficamten is currently the subject of two ongoing Phase 3 clinical trials: MAPLE-HCM (Metoprolol vs Aficamten in Patients with LVOT Obstruction on Exercise Capacity in HCM), evaluating aficamten as monotherapy compared to metoprolol as monotherapy in patients with obstructive HCM, and ACACIA-HCM (Assessment Comparing Aficamten to Placebo on Cardiac Endpoints In Adults with Non-Obstructive HCM), evaluating aficamten in patients with symptomatic non-obstructive HCM. Aficamten received Breakthrough Therapy Designation for the treatment of symptomatic obstructive HCM from the U.S. Food & Drug Administration (FDA) as well as the National Medical Products Administration (NMPA) in China. 

For further information about our partner, Cytokinetics, please visit cytokinetics.com

Etripamil Nasal Spray

(short-acting calcium channel blocker)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
Ph3 in progress
Ph3 completed, US NDA submitted
Preclinical
Phase 1
Phase 2
Phase 3
Approval
To be initiated
Ph2 in progress

Etripamil Nasal Spray

Etripamil is Milestone's lead investigational product. It is a novel calcium channel blocker nasal spray being developed for elevated and often highly symptomatic heart-rate attacks associated with PSVT and atrial fibrillation with a rapid ventricular rate (AFib-RVR). It is designed to be a rapid-response therapy that is self-administered by the patient, without the need for direct medical oversight. If approved, etripamil is intended to provide health care providers with a new treatment option to enable virtual care and patient self-management. If approved, the portable treatment, studied as self-administered, may provide patients with active management and a greater sense of control over their condition.

Etripamil, proposed brand name CARDAMYST™, is well studied with a robust clinical trial program that includes a completed Phase 3 clinical-stage program for the treatment of PSVT and soon-to-be-reported Phase 2 proof-of-concept trial for the treatment of patients with AFib-RVR.

For further information about our partner, Milestone Pharmaceuticals, please visit milestonepharma.com

Omecamtiv Mecarbil

(cardiac myosin activator)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
China NDA under review
EMA NDA under review

Omecamtiv Mecarbil

There is no cure for heart failure but there are various options to actively manage the disease and slow down progression. Unfortunately, current therapies all have limitations so that heart failure remains a leading cause of hospitalization and readmission in elderly people. Omecamtiv Mecarbil is a novel, selective cardiac myosin activator for the potential treatment for heart failure with reduced ejection fraction (HFrEF) by increasing cardiac contractility.

For further information about our partner, Cytokinetics, please visit cytokinetics.com

JX09

(formerly PB6440, aldosterone synthase inhibitor)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
Ph1 in progress

JX09

 JX09 is a next-generation, highly selective aldosterone synthase inhibitor. Aldosterone plays an important role in the development of hypertension, heart failure and chronic kidney disease. Directly targeting the synthesis of aldosterone has been developed as a novel alternative strategy. Preclinical data suggest that JX09 offers strong potency inhibiting aldosterone synthase, with very little impact on the biological functions of a homologous enzyme that is key to cortisol synthesis. The high potency and selectivity make JX09 a potential best-in-class drug candidate. 

JX10

(formerly BIIB131,novel small molecule )
Preclinical
Phase 1
Phase 2
Phase 3
Approval
Ph2 results released

JX10

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.

In January 2024, JIXING acquired the global rights for JX10, excluding Japan, from Biogen. TMS regains Japan rights of JX10 and will join forces with JIXING to expeditiously develop and launch JX10, facilitated by the formation of a Joint Development Committee.

For further information please visithttps://www.tms-japan.co.jp/

Ophthalmology

Asset
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approval
  • China studies conducted by JIXING
  • Global studies conducted by partner

Varenicline Solution Nasal Spray

(nAChR agonist in preservative-free formulation, US brand name TYRVAYA®)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
China NDA accepted by NMPA
FDA Approval received October 2021

Varenicline Solution Nasal Spray

Varenicline solution nasal spray (US brand name TYRVAYA®)  is a highly selective cholinergic agonist that is U.S. FDA approved to treat signs and symptoms of dry eye disease as a multidose nasal spray. The parasympathetic nervous system controls tear film homeostasis partially via the trigeminal nerve, which is accessible within the nose. The efficacy of varenicline solution nasal spray in dry eye disease is believed to be the result of varenicline's activity at heteromeric sub-type(s) of the nicotinic acetylcholine (nACh) receptor where its binding produces agonist activity and activates the trigeminal parasympathetic pathway resulting in increased production of basal tear film as a treatment for dry eye disease. Varenicline binds with high affinity and selectivity at human α4β2, α4α6β2, α3β4, α3α5β4 and α7 neuronal nicotinic acetylcholine receptors. 

In February 2023, Varenicline solution nasal spray was approved in Macao SAR, and since April 2023, it has been available in Boao Lecheng International Medical Tourism Pilot Zone as an imported drug for patients with urgent clinical needs.In July 2023, the new drug application was formally accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of the PRC.

TYRVAYA® is a registered trademark of Oyster Point Pharma, Inc., a Viatris company. 

OC-02 Nasal Spray

(nAChR agonist)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
To be initiated
Ph2b completed

OC-02 Nasal Spray

OC-02 (simpinicline) nasal spray contains nicotinic agonists, that bind to specific receptors in the nasal mucosa to elicit production of natural tears. Delivering OC-02 directly to the target tissue via a nasal spray is an innovative approach to the treatment of DED. The nasal spray delivery platform can provide both preservative free and preserved product formulations.

OC-02 has been previously studied by our partner, Oyster Point Pharma in two Phase 2b clinical trials for dry eye disease. As part of the dry eye disease clinical development pathway in China, JIXING will initiate Phase 1 and Phase 3 clinical studies for OC-02 starting in 1H 2024. 

For further information about our partner, Oyster Point Pharma, please visit oysterpointrx.com

LNZ100/101

(parasympathomimetic miotic agent)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
First patient enrolled in phase 3 clinical study in China
Positive Topline Data from Ph3 CLARITY Presbyopia Trials

LNZ100/101

LNZ100 (aceclidine) eye drops and LNZ101 (aceclidine/brimonidine) eye drops are being developed by LENZ Therapeutics and JIXING acquired the Greater China rights for the development and commercialization of the products in April 2022. 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.

For more information, visit: LENZ-Tx.com.

JX08

(a pre-clinical ophthalmology program)
Preclinical
Phase 1
Phase 2
Phase 3
Approval
Pre-clinical program
Pre-clinical program

JX08

A pre-clinical ophthalmology program