NeuExCell Therapeutics, Inc.

SUZHOU, China, March 21, 2024 /PRNewswire/ -- NeuExcell Therapeutics, a leading biotechnology company focused on in vivo neural regenerative therapies, announces the successful dosing of the first patient with the first-in-class NeuroD1 gene therapy product NXL-004. This milestone represents significant progress in the development of new treatment for malignant glioma patients. Led by Professor Yulun Huang and his team at the Fourth Affiliated Hospital of Soochow University in Suzhou, China, the investigator-initiated trial (IIT) will evaluate the safety and tolerability of NeuExcell's AAV-NeuroD1 gene therapy product NXL-004 in patients with glioblastoma. This clinical study marks the first clinical use of AAV-NeuroD1 gene therapy to treat patients worldwide. Glioblastoma is a highly aggressive form of brain cancer with limited treatment options and poor prognosis. NXL-004, based on NeuExcell's revolutionary in vivo trans-differentiation technology platform, aims to inhibit tumor cell proliferation and promote their transformation into neural cells through the overexpression of the NeuroD1 transcription factor. The advantage of this glioma cell trans-differentiation approach is minimal side-effect on other brain cells, which is distinct from radiation or chemotherapy that has collateral damage on healthy cells. Promising preclinical results from animal model studies have paved the way for this groundbreaking clinical trial, offering hope for many patients suffering from debilitating brain disorders such as malignant glioma. "We are very excited about completing the dosing of the first patient using NeuroD1 gene therapy product NXL-004 in this exploratory clinical study," said Dr. Yulun Huang, the study's lead investigator. "This milestone underscores the dedication of our team to try innovative approaches in addressing unmet medical needs such as glioblastoma. We are committed to closely monitor the patient and evaluate the safety and efficacy of NXL-004 in this trial." Professor Gong Chen, the inventor of in vivo neural regenerative gene therapy and the founder of NeuExcell Therapeutics, expressed his gratitude: "We are grateful to Professor Yulun Huang and his team for their outstanding work. This first-in-human trial sets an important milestone for our first-in-class NeuroD1 gene therapy that has the potential to treat millions of patients suffering from severe neurological disorders. We are dedicated to developing game-changing treatments for neurodegenerative diseases using in vivo neural regenerative therapies." About NeuExcell Therapeutics: NeuExcell Therapeutics is a leading biotechnology company focused on developing innovative neural regenerative therapies to address unmet medical needs in the field of neurodegenerative diseases and cancers. NeuExcell is committed to revolutionizing the field of CNS drug development and improving outcomes for patients worldwide.

PHILADELPHIA, Dec. 18, 2023 /PRNewswire/ -- On December 7, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation ("ODD") to NXL-004, an investigational AAV gene therapy product being developed by NeuExcell Therapeutics Inc. ("NeuExcell"), for the treatment of malignant glioma. This is the world's first AAV gene therapy product for malignant glioma to receive FDA ODD designation. Malignant gliomas are the most common primary malignant brain tumors in adults, about 60% of which are glioblastoma (GBM). Characterized by high malignancy and mortality, the current standard-of-care of GBM, encompassing surgery, radiotherapy, and chemotherapy, results in a median overall survival of merely 15-18 months and a 5-year survival rate below 10%. Recurrence is virtually inevitable for malignant glioma and there is no clear standard salvage therapy. NXL-004 is an innovative treatment based on the groundbreaking astrocyte-to-neuron ("AtN") conversion platform developed by Professor Gong Chen. It has shown good efficacy and safety in preclinical studies and will enter first-in-human clinical trials in early 2024. According to the U.S. Orphan Drug Act, drugs that have received ODD in the U.S. can enjoy a series of regulatory support, including but not limited to tax credits for clinical trial expenses, exemption of new drug application fees, and 7-year market exclusivity after product approval. ODD can help accelerate development of NLX-004 and provide new hope for many GBM patients around the world. NeuExcell is committed to restore health and improve the quality of life for millions of patients worldwide suffering from neurodegenerative diseases and neurological injuries. NeuExcell's disruptive AtN technology has the potential to regenerate neuronal tissues and restore neurological functions, with robust preclinical efficacy data in a wide variety of disease models including non-human primates. NeuExcell's lead clinical programs include Alzheimer's disease, ischemic stroke and GBM, all expected to enter the clinic in 2024, with pipeline expansion opportunities in numerous indications such as Huntington's disease and Amyotrophic Lateral Sclerosis. SOURCE NeuExcell Therapeutics

Renovacor wanted to be in the clinic already. The biotech expected an $11 million Series A in summer 2019 would bankroll an IND filing for its cardiac gene therapy, but as it goes with drug R&D, the path took some twists. Fifty-four weeks after shipping up to New York City for a blank check merger, Renovacor is turning off the lights on $RCOR and going the way of $RCKT , or Rocket Pharmaceuticals, in an all-stock deal that adds to the latter’s gene therapy ambitions, which includes two likely BLA filings next year. Touting 100% OS rate in pivotal rare disease trial, Rocket Pharma ready to head to FDA For an implied value of $2.60 per share, Rocket is snapping up the preclinical-stage biotech by issuing its own shares to Renovacor stockholders. The acquisition holds an equity deal value of about $53 million and is expected to close sometime by the first quarter of 2023, the companies said early Tuesday. Renovacor said last month that it expects to be in the clinic later this year for its rare cardiac gene therapy, targeting BAG3-associated dilated cardiomyopathy, but that guidance is now out the door. Magdalene Cook “As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team,” Renovacor CEO Magdalene Cook said in a statement. After that 2019 Series A, the Bay State-based biotech ended up securing additional funds by landing on the Nasdaq in a blank-check tie-up with Chardan’s second SPAC last September. Between the deal announcement and ringing the bell on Wall Street, the Temple University spinout lined up a C-suite flush with execs from MyoKardia , BioMarin , PassageBio , NeuExcell Therapeutics and Lung Biotechnology . Biotech targeting rare heart mutation with gene therapy scores $11M in Series A haul For its part, Cranbury, NJ-based Rocket says the deal will add $38 million to the cash heap, which would extend runway into the second quarter of 2024. As any M&A deal should tell you, the gene therapy companies said the combination will create “strong synergies.” This includes assets, employees, capabilities, IP, and scientific and clinical collaborators, they noted. Just this summer, Renovacor added another AAV gene therapy program to its pipeline through a research collaboration with the University of Utah, going after “multiple genetic segments of arrhythmogenic cardiomyopathy.” “By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible,” Rocket CEO Gaurav Shah said in a statement. The recent gene therapy approvals over at bluebird bio — some of the costliest treatments ever approved in the US — “[bode] well” for companies making lentivirus-mediated gene therapies, like Rocket, according to SVB Securities analysts earlier this week. The late Friday Skysona approval hasn’t directly translated into a stock bump for Rocket just yet. Rocket has three such LV programs across Fanconi anemia (FA), leukocyte adhesion deficiency-I (LAD-I) and pyruvate kinase deficiency (PKD). Rocket expects to ask the FDA for approval of the LAD-I gene therapy in the first half of next year. The company could also ship its papers on the FA asset to the regulator next year. Meanwhile, Rocket is picking up an AAV-focused biotech, a space that has caught the watchful eyes of the FDA in recent years with safety concerns. Nonetheless, new AAV gene therapy players continue to crop up.