Global Players Making Waves in MDD, Sanfilippo syndrome and SCLC

Global Health

Across the globe, biopharma companies are continuously working to develop, test and bring new treatments to the market to make a difference in patients' lives. Here are three clinical updates announced this week, spanning three different continents. 

American major leaguer AbbVie has submitted a supplemental New Drug Application for Vraylar (cariprazine), a drug first approved 6.5 years ago for schizophrenia and bipolar disorder. AbbVie is hoping to add in a new indication for the drug as an add-on treatment for patients with major depressive disorder (MDD) who are already taking an antidepressant ongoing. 

In the supporting studies, cariprazine showed statistically significant changes from baseline by 6-8 weeks. Patients had a significant improvement in their MADRS score, a scale used to rate depression, compared to those on the placebo. Long-term safety and tolerability were established over 26 weeks. 

Even with all the antidepressants on the market, many living with MDD are too familiar with the struggle to find something that helps. AbbVie continues to push the envelope to offer options that could be the right fit to help one more suffering person. 

Across the pond, Parisian gene therapy platform Lysogene has gone to bat for a rare genetic MPS IIIA, also known as Sanfilippo syndrome. The severe, progressive disorder targets the central nervous system causing neurological symptoms in early childhood such as progressive dementia, aggressive behavior, seizures, loss of vision and more. Most patients do not survive past the second decade of life. 

Lysogene has an ongoing phase II/III trial for its investigational gene therapy to treat Sanfilippo syndrome. Since the disorder is caused by mutations to the SGSH gene, the treatment is intended to be a one-time intracerebral delivery of a working copy of that gene. Once functional, the brain would be able to secrete the enzyme needed to break down heparan sulfate. 

So far, positive biomarker data has been seen, reducing the buildup of the harmful molecule in cerebrospinal fluid. Currently, there is no treatment specified for MPS IIA. Lysogene will continue to analyze data to examine the treatment's ability to improve the patient's cognitive development, behavior and quality of life. 

To the east of Paris, Hong Kong's Simcere Pharmaceutical Group is excited to announce its phase III study of a drug co-developed with G1 Therapeutics for extensive-stage small cell lung cancer (ES-SCLC) has met its primary endpoint. According to Simcere, Trilaciclib is the world's first and only anti-tumor drug with a myeloprotective effect. 

In trial patients, Trilaciclib was able to significantly decrease the duration of severe neutropenia, a condition where white blood cells called neutrophils, which the body utilizes to fight bacterial infections, are depleted. This condition occurs due to the toxic side effects of the first-line treatment for ES-SCLC, chemotherapy. For chemo patients, Trilaciclib has a "bone marrow protection effect" to fill a clinical gap in cancer treatment. 

Simcere's CMO hopes the data will support an accelerated NDA approval in China to benefit Chinese small cell lung cancer patients as soon as possible. 

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