AI-discovered drug for ALS enters clinical trials

Patient enrollment complete for clinical trial of 4B Technologies’ AI-discovered ALS drug supported by Insilico Medicine.

Two years after the collaboration was announced between Insilico and 4B Technologies, the AI-discovered ALS drug, FB1006, completed patient enrollment for the IIT study. From target identification to efficacy assessment, the discovery and development of FB1006 was fully driven by AI, with Insilico’s PandaOmics platform used for target identification and drug repurposing.

Longevity.Technology: Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease or motor neurone disease) is a fatal type of motor neuron disease characterized by progressive degeneration of nerve cells in the spinal cord and brain. Relentlessly progressive, ALS results in muscle weakness and paralysis that can include the inability to walk and speak, or even swallow and breathe. It is universally terminal, and as with many neurodegenerative diseases, aging is the biggest risk factor [1]. The average life expectancy of an ALS patient after diagnosis is two to five years, and currently, there is no cure for or way to prevent ALS from progressing.

The ALS drug known as FB1006, was fully discovered and developed using AI, from target identification to efficacy assessment. The company leading the drug’s development, 4B Technologies, has now successfully completed enrollment of all 64 patients in the investigator-initiated clinical trial (IIT) for the treatment of ALS. The program was conducted at the Third Hospital of Peking University, and is expected to complete the double-blind dosing in August 2024 and the one-year clinical observation in February 2025.

Led by Professor Bai Lu of Tsinghua University, with Professor Dongsheng Fan, a renowned ALS expert from Peking University Third Hospital, as the Principal Investigator, and in collaboration with 4B Technologies, Insilico and other AI institutes, the IIT study integrates industry, academia, research and clinical medicine to drive new treatment possibilities for ALS patients.

AI-discovered drug for ALS enters clinical trials
ALS causes motor neurons to gradually deteriorate and then die

AI technology was applied to multiple stages of the drug’s discovery and development, including target identification, patient enrollment and efficacy assessment. Using AI allowed researchers to significantly shorten the patient screening and complete enrollment in under one year, as well maximizing the use of the clinical data of the subjects, solving the problem of insufficient sample size of rare diseases.

“The development of ALS medications calls for innovative approaches to accelerate clinical research,” said Dongsheng Fan, MD, PhD, from Peking University Third Hospital.

“In the clinical study of FB1006, we successfully adopted the innovative approach of Trial-ready Cohort (TRC), which is established through datasets comprising patient history, blood biochemistry results, EEG/EMG, imaging, genome and more. With the help of the ALS TRC database, we could accurately enroll patients upon clinical trial initiation, based on target and drug properties, clinical effects and side effects, thus shortening the time and reducing the cost of patient enrollment, while increasing the success rate of the Investigator-initiated Trial (IIT). We look forward to the promising results of FB1006.”

AI-discovered drug for ALS enters clinical trials
Bai Lu, PhD, is the founder of 4B Technologies

“We are pleased to see that 4B Technologies completed the whole development process of FB1006, from target identification and compound screening to patient enrollment, in less than 2 years with the joint efforts,” said Bai Lu, PhD, founder of 4B Technologies and professor at Tsinghua University.

“The discovery of FB1006 is powered by AI applications, and the ongoing clinical trial has utilized AI in processes including patient enrollment and efficacy assessment, potentially benefiting more patients. We expect that these explorations into innovative concepts, technologies and modes will help overcome the challenges of ALS and benefit patients worldwide.”

Insilico played a key role in the early drug development of the FB1006 program. In August 2021, Insilico and 4B Technologies embarked on a collaboration to leverage PandaOmics, Insilico’s proprietary AI target identification engine, to discover potential targets and to advance drug repurposing strategies for ALS treatment.

In this collaboration, Insilico identified 28 novel targets with potential for the treatment of ALS, three of which have approved and marketed drugs for other diseases. 4B Technologies, leveraged its research expertise in translational and clinical medicine in the field of neurological diseases to evaluate, select and manufacture one of these drugs as a candidate repurposed for ALS. An IIT clinical trial was then initiated.

AI-discovered drug for ALS enters clinical trials
Feng Ren, PhD, is co-CEO and Chief Scientific Officer of Insilico Medicine

“It is exciting that the research Insilico contributed to has advanced to the clinical stage as a potential drug for ALS in such a short period of time,” said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine.

“FB1006, as an innovative drug, was assisted by AI in both early drug discovery and clinical stage studies. It took less than 2 years from target identification to the completion of IIT enrollment, which once again proves the ability of AI to accelerate drug discovery and development. We look forward to the efficient progress of the IIT program and further research results.”


Photographs courtesy of Insilico Medicine