Prilenia Therapeutics Lead Product

Pridopidine is the lead company’s product. It is a highly selective sigma-1 receptor (S1R) agonist with an established safety profile and potential in multiple movement disorders and neurodegenerative diseases affecting adults and children.

Pridopidine demonstrated a maintained functional capacity in early Huntington’s Disease as measured by Total Functional Capacity (TFC). The product was recently selected from an international competition of over 30 potential therapeutics for inclusion in the first-ever ALS platform trial, led by the Healey Center for ALS at Massachusetts General Hospital. 

Pridopidine is currently in late-stage clinical development for HD and ALS.

Both trials, the global phase 3 clinical trial in HD and the platform trial in ALS, are currently active and recruiting. In the face of compelling results, these trials could potentially lead to the registration of pridopidine for more indications.  

Recent News from Prilenia Therapeutics

Prilenia Therapeutics announced that it has enrolled more than 120 patients in its Phase 3  trial of pridopidine in HD, reaching 25% of the total target enrollment. The study remains on target to complete enrollment by Q4 2021 as originally planned.

The study is being conducted in collaboration with the Huntington Study Group at 60 sites across North America and Europe.

Pridopidine Safety

To date, no safety signals of concern have emerged and none of the participants have dropped out from the study. This is consistent with the previously established favorable tolerability and safety profile of pridopidine. 

Dr. Y. Paul Goldberg Appointment

Prilenia Therapeutics has also announced the appointment of Dr. Y Paul Goldberg (MB ChB, Ph.D.) as Chief Scientific Officer and Head of Early Clinical Development. Dr. Goldberg will lead the scientific strategy and build the early clinical development plan for pridopidine in new indications including evaluation of opportunities for expanding Prilenia’s pipeline.  

Dr. Goldberg brings more than 20 years of industry experience, working across clinical development, drug discovery, and medical genetics. Most recently, he served as VP of Clinical Development at Ionis Pharmaceuticals where he led rare disease programs with a strong neurological focus. Prior to this, Dr. Goldberg served in various roles at Xenon Pharmaceuticals including Senior VP, Clinical Development. Dr. Goldberg obtained an MB ChB and Ph.D. at the University of Cape Town. He obtained his fellowship in medical genetics (FRCPC) from the University of British Columbia and practiced as a medical geneticist for many years with a focus on adult neurogenetic disorders.

Dr. Michael R. Hayden,  CEO and Founder of Prilenia, commented, “We are delighted that Dr. Y Paul Goldberg joins the Prilenia team. His proven track record in clinical development and his strong academic background will be of enormous value to our company”.

​Dr. Y Paul Goldberg, said, “Joining Prilenia presents a fantastic opportunity to help advance a new and promising therapy for severely undertreated neurological diseases. I look forward to supporting the team and helping to accelerate the development of pridopidine, to potentially provide a much-needed treatment option for patients and their families.” 

Huntington’s disease

Huntington’s disease is a progressive brain disorder. It is caused by a single defective gene on chromosome 4. This defect is “dominant. The disorder is named after George Huntington, M.D., who first described this condition in the late 1800s.

Symptoms of the disease develop between ages 30 and 50, but they can appear as early as age 2 or as late as 80. The hallmark symptom of Huntington’s disease is uncontrolled movement of the arms, legs, head, face and upper body.

Huntington’s disease also causes a decline in thinking and reasoning skills, including memory, concentration, judgment and ability to plan and organize.

Huntington’s disease brain changes lead to depression, anxiety, uncharacteristic anger, irritability and obsessive-compulsive behavior.

Amyotrophic Lateral Sclerosis

ALS is also known as Lou Gehrig’s Disease or Motor Neuron Disease; it is a chronic progressive neurodegenerative disease. This deadly disease affects about 350,000 people worldwide. The majority of ALS cases (~90%) are sporadic. However, ~10% of ALS cases are caused by genetic mutations. 

Several genes have been discovered, which cause ALS, including the gene encoding the S1R, which explains the use of pridopidine as it is a highly selective S1R agonist.

In patients with ALS, motor neurons in the brain and spinal cord that convey messages to the muscles degenerate, which affects the ability of the brain to communicate with muscles. Muscle wasting could occur and progressive paralysis could start. Patients rapidly lose their ability to walk, speak, eat and breathe and become fully dependent on their caretakers.

Prohost Observations 

With the advancement of biotechnology we see biotech and biopharma companies coming up with treatments for deadly hereditary or nonhereditary genetic diseases. We believe that Prilenia Therapeutics will be among the most important creative and successful firms in bringing products that treat CNS diseases and genetic diseases. 

Though you are not be able to invest in this firm now, we believe that Prilenia will become a publicly traded firm that can be invested in directly. There is also the possibility, however, that this firm might be acquired by a deep-pocketed biopharma company that can be invested in. 

Read more at www.prohostbiotech.com.