“Tackling Unmet Medical Needs”

When we think about an accountant, we have always stereotyped them to be sitting in the office every day following the accounting standards and preparing reports. Being an accountant who loves exploring new opportunities and enjoys creative process , Sabrina Khan is the Chief Financial Officer and company secretary of Aptorum Group Limited. Aptorum is a Nasdaq and Euronext listed (APM) pharmaceutical company dedicated to developing and commercializing novel therapeutics to tackle unmet medical needs including orphan, infectious and metabolic diseases. Ms. Khan leads the company’s financial strategy and operations, such as the IPO and fund-raising activities, as well as investor relations.

" Aptorum is facilitating the development of medical innovations, particularly in those that able to meet current unmet medical needs and significantly improve clinical and surgical experience."

Aptorum is facilitating development of medical innovations, particularly those that able to meet current unmet medical needs and significantly improve clinical and surgical experience. “We have a group of very talented and experienced clinicians, pharmaceutical executives and scientists with diversified strengths and specialty areas,” Sabrina. “We brought them together and provided Aptorum Group’s platform and resources to allow these specialists to have an opportunity to explore and work together in developing new therapeutic innovations to tackle current unmet medical needs.” Aptorum Group focuses on diseases that are currently considered as unmet medical needs or do not have effective treatment available. “We believe that patients with these diseases often suffer from the lack of choices of effective drugs and face limited therapeutic options available. On this basis, I believe that our company’s objective is simple – to bring effective drugs to these patients and tackle these diseases,” she adds.

One of Aptorum’s major therapeutic development is in “orphan diseases” (also known as rare diseases), an exciting emerging market that has a huge growth potential. In US, there are over 7000 orphan diseases have been identified (compared to over 2500 identified back in 2015), and approximately over 90% of these diseases do not have effective treatment. On this basis, Smart-ACTTM was set up to focus on screening our library of over 2600 existing approved small molecule drugs against the selected orphan disease targets, with the aim to “discover and repurpose” drugs that can work for other orphan disease indications and to shorten drug development from on average of 10+ years to 3-4 years and cut short the total cost of drug development in some cases up to 90%. Drug repurposing is well regulated and has had numerous successful precedents (for example, better known ones are Viagra, Trastuzumab, Minoxidil etc) and the US FDA has a reg 505b(2) approval pathway which we can fast track repurposed drugs to late human clinical trials, because all these drugs have established safety and toxicity records when previously approved. “Our first proof of concept drug under this platform is SACT-1 which targets Neuroblastoma, a common form of pediatric solid tumor, which is expected to reach clinical stage in Q4 2020 this year and we are extremely excited about this drug, especially because on average only 40% of neuroblastoma patients on respond to treatment during the 5 year survival period under existing therapies,” adds Sabrina. “Another therapeutic focus is on infectious diseases under our Acticule (ALS) platform, especially focusing on diseases that have risks of triggering pandemics in the future.”

As a fast-growing biopharmaceutical company, Aptorum often faces new opportunities and new challenges. Hence, Sabrina motivates the staff to proactively participate in different projects, jump out of their comfort zone, take up the responsibility and be a key player. Aptorum Group has a rich pipeline of over 15 existing drugs under development and in addition.They are constantly investing in their drug discovery platforms to generate additional potential drug candidates for other diseases. For example, the Smart-ACTTM platform is currently developing 4 drug assets and we hope to generate 3-5 further clinical stage assets going forward, making Aptorum Group potentially one of the largest orphan disease pipeline company in the world and eclipse some of those in US. “Our metabolic disease platform which is called Claves has a very scalable macromolecule technology to target major classes of metabolic diseases such as obesity, renal, cardiovascular diseases (etc.) using the human’s gut microbiome as an extremely first-in-class approach,” adds Sabrina. IE