On the 25th, Pharmosa Biopharm's General Manager Gan Pei shared during an investor conference that the company will focus on advancing two clinical trials for L608, completing the EU clinical development consultation for L608, producing and supplying clinical trial drugs and specialized nebulizers for L606, building a resilient supply chain for drug-device combination products, and receiving development milestone payments for L606 based on the regulatory and clinical progress with authorized partners. The company is optimistic about its operations for the entire year.
In addition, with ample cash reserves, Pharmosa is well-positioned to support its clinical activities and the establishment of its own filling production lines this year, contributing to a positive outlook for overall operations.
Gan Pei noted that L608, an inhalation drug encapsulating Iloprost in liposomes, is designed for the treatment of systemic sclerosis-related Raynaud phenomenon and digital ulcers (SSc-RP/DU), as well as the first rare pulmonary arterial hypertension (PAH) disease. The first phase of clinical trials for L608 was completed in Australia in Q4 of last year. Since L608 has received orphan drug designation from the US FDA for treating systemic sclerosis (also known as scleroderma), the company plans to aggressively push forward with late-stage clinical trials for L608 in SSc-RP/DU this year.
Scleroderma is a very rare autoimmune disease, and Raynaud phenomenon and digital ulcers are the primary vascular-related clinical manifestations of the condition. In Europe, there is only one marketed injectable drug, Ilomedin, which contains Iloprost as its active ingredient. However, there are no approved drugs for this indication in the United States.
Pharmosa pointed out that the existing drug, Ilomedin, requires intravenous infusion for six hours a day for five days in the hospital. In contrast, L608 is an inhalable formulation with a specialized nebulizer that allows patients to self-administer at home. Furthermore, inhaled L608 with liposomal Iloprost can directly reach deep alveoli and continuously release the drug into the bloodstream, enhancing the treatment effect in the peripheral circulation and improving blood circulation abnormalities in the hands and feet over long-term use.
There are more than 70,000 scleroderma patients in the US alone, and based on the current pricing of similar drugs in the market, a single course of treatment for L608 could cost around $100,000. The market opportunity is substantial. Pharmosa has completed preclinical consultation with the FDA and plans to submit applications for the Phase II/III clinical trials for SSc-RP/DU treatment this year and initiate patient recruitment.
Moreover, Pharmosa will accelerate the Phase I clinical trial of L608 in New Zealand, aiming to enroll 56 participants, with the goal of completing recruitment this year. The trial aims to establish safety, tolerability, and pharmacokinetics data for higher doses, which will provide more flexibility for long-term dose adjustments and speed up the overall development timeline for L608.
Regarding the nebulizer, considering the global commercialization needs of its new drug and the flexibility of drug-device combination regulations, Pharmosa has introduced its first self-branded nebulizer into the L608 clinical trial in Australia. A new generation of different specifications of mesh vibration nebulizers is being continuously optimized for functionality and convenience. Development and mass production have been completed, and these will be used to supply both L608 and L606 as delivery devices. Starting this year, the company expects its nebulizer products to contribute significantly to its operations.
Pharmosa has successfully established a unique nebulizer technology and product platform, greatly enhancing its ability to develop "drug-device combination" new drugs. To ensure a resilient supply chain, the company is also building its own sterile filling and packaging automated production line, along with an analytical laboratory. The laboratory, which meets European and American regulations, has already been completed and will allow for stability testing of pharmaceutical products. The filling plant is expected to be completed in the second half of this year, with a maximum annual capacity to meet the needs of tens of thousands of patients. The company will apply for inspections from the TFDA, FDA, and EMA, further positioning Taiwan as a primary production base to supply pharmaceuticals globally.
As for the licensed L606, Pharmosa will collaborate with its partners on the regulatory strategy and clinical progress, supplying clinical trial drugs and specialized nebulizers for L606, and receiving milestone payments based on the relevant progress. The company will also continue negotiations for licensing in regions such as China, South Korea, and Southeast Asia to complete the global licensing strategy for L606.
Looking ahead to this year, Gan Pei stated that both L606 and L608 are being developed to address unmet medical needs. After L606 enters pivotal Phase III clinical trials, it is nearing the final stage before commercialization and large-scale production. Pharmosa will continue to actively collaborate with partners. The company will also accelerate the clinical trials for L608 and simultaneously begin commercialization planning to enhance the market value of its existing products. Optimistic about the future development potential of both new drugs, Pharmosa maintains a positive outlook for its overall operations.
Resource: 國邑射五箭 今年展望正向
