Attractiveness of fusion proteins sparks financial investments and biopharmaceutical collaborations
In the realm of medical research, fusion proteins are making significant strides in the treatment of cancer and rare diseases. Two notable developments come from Larimar Therapeutics and Citius Oncology.
Larimar Therapeutics' nomlabofusp for Friedreich's Ataxia
Nomlabofusp, a fusion protein developed by Larimar Therapeutics, is making headway in the treatment of Friedreich's Ataxia (FA), a rare neurodegenerative disease characterised by a deficiency in the frataxin (FXN) protein. Recent clinical data shows that daily subcutaneous doses of 25 mg were generally well-tolerated up to 260 days in an open-label extension study, with increases in FXN levels detected in buccal and skin cells, indicating biological activity correlated with dosing [1].
Phase 1 and 2 studies have demonstrated that nomlabofusp not only raises FXN levels but also modifies gene expression and lipid profiles. Modelling suggests that 50 mg doses could be effective for most patients [1]. Larimar Therapeutics recently secured $60 million in funding to support Phase 3 trials and FDA-validated manufacturing efforts, aiming for accelerated approval. The FDA's acceptance of skin FXN as a surrogate endpoint could potentially speed regulatory timelines and secure orphan drug exclusivity [3][4][5].
Citius Oncology's Lymphir for T Cell Lymphoma
Lymphir, a fusion protein immunotherapy from Citius Oncology, is targeting abnormal T cells in relapsed or refractory cutaneous T-cell lymphoma (CTCL), an aggressive blood cancer. It binds to the IL-2 receptor on tumor and regulatory T-cells (Tregs), delivering a toxin payload to kill tumor cells and temporarily depleting immunosuppressive Tregs, thereby enhancing the patient's immune response against the cancer [2].
The FDA approved Lymphir about a year ago, making it the only immunotherapy of its kind for this indication. Citius Oncology recently secured $9 million in funding to support Lymphir’s commercial rollout and further clinical development, including studies exploring its use prior to CAR-T therapy in B-cell lymphoma and in combination with Keytruda in solid tumors [2].
These advancements in fusion protein technology are reshaping the landscape of both rare disease and oncology treatments, offering new hope for patients with unmet needs.
[1] Larimar Therapeutics Press Release [2] Citius Oncology Press Release [3] FierceBiotech Article [4] Endpoints News Article [5] BioPharma Dive Article
- The biopharma sector is witnessing transformative progress with fusion proteins, such as Larimar Therapeutics' nomlabofusp and Citius Oncology's Lymphir, advancing in drug development for medical-conditions like Friedreich's Ataxia and T Cell Lymphoma, respectively.
- In the realm of rare diseases, nomlabofusp, a fusion protein from Larimar Therapeutics, shows promising results in boosting frataxin protein levels and modifying gene expression in Friedreich's Ataxia patients, potentially setting the stage for accelerated approval in Phase 3 trials.
- Citius Oncology's Lymphir, another fusion protein immunotherapy, targets abnormal T cells in cutaneous T-cell lymphoma by binding to the IL-2 receptor and temporarily depleting immunosuppressive T-cells, rekindling the patient's immune response against the cancer.
- The advancements in fusion protein technology, as demonstrated by the success of nomlabofusp and Lymphir, are creating posiitive ripples across the health-and-wellness industry, offering new possibilities for patients facing unmet needs in treating various medical-conditions, including rare diseases and oncology.
References: Larimar Therapeutics Press Release, Citius Oncology Press Release, FierceBiotech Article, Endpoints News Article, BioPharma Dive Article
