Audentes Therapeutics Inc. (NASDAQ:BOLD) seems to have made good progress in 2018 according to a recent update report. The company made massive accomplishments and as per the Chief Executive Officer, Matthew R. Patterson, it was an outstanding year. Among the many accomplishments of Audentes were RMAT and PRIME designations from the FDA and EMA respectively. The company also shared with the FDA it ambitions to register pathways for AT132. From the ASPIRO Phase 1/2 study, the treatment for X-linked Myotubular Myopathy demonstrated a very promising clinical profile.
The preclinical studies for AT845 a treatment for Pompe disease were equally encouraging. The company is making plans to file investigational new drug (IND) application for it in Q3 2019. Audentes’ balance sheet did not disappoint either because cash, cash equivalents, and short-term investments totaled nearly $450 million. This includes net proceeds of $162.5 million.
Audentes says it is preparing for other multiple milestones
There is a lot aligned for 2019 as Patterson explains. He says that they have put systems and structures in place, which will support the company’s growing efforts. The progress from the Pompe disease program is one of its kind. Plans are underway to have AT720 advanced. The novel AAV-based therapeutic is a treatment option for a large neuromuscular disease.
In affirmation, Patterson says, “We are preparing for multiple milestones and inflection points across our portfolio of product candidates in the coming year, including the continuation of BLA and commercial readiness activities in our XLMTM program.”
All these are achievable courtesy of the company’s strong balance sheet. The CEO insists that they are well – positioned to offer transformative therapies to those suffering from rare diseases. They are passionate about forging strong, global relationships with the patients and they will also look out for medical communities.
Expansion of the company’s leadership team Audentes’ 2018 activities, projects, and vision had such strong support. The company recruited Eric B. Mosbrooker to take up the positions of a Senior VP and Chief Commercial Officer. Eric has substantial experience according to Patterson and is of great benefit in building the company’s vision. With his 15 years in the healthcare industry, he is more than Audentes would ask for.