Partnering
At Enemsa Pharma, we believe that scientific innovation reaches patients faster and more effectively through strong, strategic partnerships. We are actively seeking collaborations with pharmaceutical companies, patient organizations, and clinical experts to advance the development of Icerguastat, our first‑in‑class modulator of the Integrated Stress Response (ISR), across multiple severe and underserved diseases.
Clinical development of Icerguastat in ALS
Icerguastat has demonstrated compelling clinical potential in Amyotrophic Lateral Sclerosis (ALS). Our exploratory Phase 2 clinical study in 51 bulbar‑onset ALS patients showed a favorable safety profile, clinical benefits across multiple validated outcomes, clear engagement of disease‑related molecular pathways, and improvement in key biomarkers, thereby validating our medical hypothesis.
Icerguastat has been granted Orphan Drug Designation to the FDA and EMA for the ALS indication, underscoring the relevance of our approach in an area of high unmet medical need.
Our ALS development strategy is strengthened by the active support of leading key opinion leaders (KOLs) in the ALS field, who recognize both the quality of our Phase 2 results and the therapeutic promise of Icerguastat.
Enemsa Pharma is committed to advancing Icerguastat through the next stages of clinical development, and we believe that partnering with established pharmaceutical companies is the most effective path forward.
We welcome collaborative discussions with industry partners and patient associations interested in accelerating therapeutic innovation for people living with ALS.
Clinical development of Icerguastat in CMT subtypes
Enemsa Pharma is also advancing Icerguastat toward clinical evaluation in Charcot‑Marie‑Tooth (CMT), one of the most common inherited neuromuscular disorders, characterized by chronic and progressive degeneration of peripheral nerves which leads to muscle weakness, motor impairment, sensory loss, and significant lifelong disability.
While many patients present with the more prevalent CMT1A subtype (PMP22 gene duplication), several rare and severe forms of CMT – including CMT1B (mutations in MPZ gene) and CMT2A (mutations in MFN2 gene)- are associated with earlier onset, faster progression, and greater clinical burden.
Preclinical studies with Icerguastat, that acts by fine‑tuning the integrated stress response (ISR) to restore cellular homeostasis, demonstrated its broad therapeutic potential across several CMT subtypes:
- CMT1A and CMT1B: Icerguastat has shown proven efficacy in animal models, improving myelination and peripheral nerve function.
- CMT2A: Icerguastat was shown to revert the pathological cellular phenotype of two different types of human CMT2A iPS cell-derived motor neurons bearing MFN2Arg94Gln and MFN2Arg707Trp mutations, and to prevent locomotor impairments in a mouse model of axonal CMT2A bearing MFN2Arg94Gln.
Together, these findings provide robust, cross‑subtype preclinical proofs of concept, supporting the advancement of Icerguastat into clinical development for CMT subtypes where no disease‑modifying therapies currently exist.
Icerguastat has received Orphan Drug Designation to the FDA and EMA for the CMT indication, recognizing the critical unmet need and the strength of our scientific rationale.
Given the rarity of some CMT subtypes, the development pathway may be eligible for accelerated approvals or equivalent expedited regulatory frameworks, provided that meaningful clinical and biomarker improvements are demonstrated.
Our program is supported by patient associations and key opinion leaders in neuromuscular diseases, who acknowledge the strong biological rationale behind Icerguastat’s mechanism of action and its potential to address the core cellular dysfunctions in these conditions.
Enemsa Pharma seeks partnerships with pharmaceutical companies committed to bringing transformative therapies to rare disease communities. We welcome discussions with industry partners and patient organizations wishing to advance therapeutic innovation for patients living with underserved forms of CMT.