Pipeline
We are focused on the development of novel drug candidates with compelling mechanisms of action, regulatory pathways, and commercial opportunities
LX-101: A New Approach to Targeting IGF-1R
Our lead product candidate, LX-101, is a novel, clinical-stage, “next generation”, precision-engineered targeted therapy directed to the insulin-like growth factor-1 receptor (IGF-1R). The IGF-1/IGF-1R pathway has been implicated in a host of malignancies and autoimmune diseases and we believe represents a scientifically and medically rational target.
LX-101 has a differentiated mechanism of action, and consists of a proprietary, precision-engineered IGF-1 variant, with high affinity to IGF-1R, coupled to a cytotoxic payload, methotrexate. Methotrexate is a cytotoxic inhibitor of DNA synthesis, repair, and cellular replication and has been used to treat patients with certain cancers and autoimmune diseases, including thyroid eye disease (TED).
Active payload
Optimized
IGF-1R variant
LX-101
IGF-1
receptor
Precision targeting: Optimized IGF-1 variant targeting IGF-1R, enabling precise payload delivery to IGF-1R+ cells
Rational payload: Delivers methotrexate, used to treat cancer and autoimmune disease (including TED), directly to diseased cells
Novel and differentiated: Offers a differentiated mechanism of action compared to past and present IGF-1R targeting agents
Positive clinical experience: Well-tolerated with single agent activity in Phase 1 trials of advanced, pretreated cancer patients
Large market opportunity: Development paths in a wide range of oncologic and autoimmune indications
LX-101, formerly 765IGF-MTX, has been clinically evaluated in Phase 1 trials conducted by the licensor of patients with advanced, pretreated cancer, where it was found to be well-tolerated and demonstrated single agent activity. We intend to further dose escalate and optimize the administration schedule given that no dose limiting toxicity or maximum tolerated dose was reached in these trials.
As a next step, we intend to build upon LX-101’s positive Phase 1 clinical experience by leveraging its novel and differentiated features, along with the well-documented involvement of IGF-1R in a multitude of diseases. We plan to advance LX-101 to key clinical milestones that offer the potential for expedited regulatory pathways and attractive market opportunities in oncology and autoimmune disease. With this approach, we believe that we can create a positive impact on patients with certain debilitating diseases.
Lirum Pipeline
Oncology
Pan-cancer
(IGF-1R )
low-high
Pediatric
(well-established IGF-1R pathway involvement)
Adult
(well-established IGF-1R pathway involvement)
3
2
3
1
Autoimmune
4
Thyroid Eye Disease
1. Some indications by virtue of certain factors (e.g., unmet medical need, etc.) could lend themselves to the possibility of pivotal phase 2 studies or other expedited development pathways, although we cannot be assured that LX-101 or any future products will qualify.
2. This trial, conducted by the licensor with 765IGF-MTX, the former name of LX-101, enrolled patients with multiple cancer types including colorectal, endometrial, pancreatic, breast, basal cell carcinoma, Hodgkin’s lymphoma, and others. IGF-1R expression was assessed on patient tumors via immunohistochemical staining and scored based on the proportion of cells that were positive (PS=proportion score; range 0%-100%) and Q score (range 0-7), which is the combination of PS and intensity score (IS).
3. Cancers with “well-established IGF-1/IGF-1R pathway involvement” include those tumor types with genetic alterations relating to the pathway and/or elevated IGF-1R expression.
4. Reviewing opportunities in other autoimmune diseases including rheumatoid arthritis, Graves’ disease, Cushing’s syndrome, lupus, Crohn’s disease, and others.