NEW YORK – Taysha Gene Therapies on Monday said it has entered into a securities purchase agreement for a private placement (PIPE) financing, which it expects will bring in gross proceeds of approximately $150 million.
In the PIPE, Taysha is selling 122,412,376 shares of its common stock at $.90 per share, and instead of offering common stock to certain investors, it is offering pre-funded warrants to purchase up to 44,250,978 shares of common stock at $.899 per pre-funded warrant.
The PIPE was led by new investor RA Capital Management and included participation from an unnamed large institutional investor, as well as PBM Capital, RTW Investments, Venrock Healthcare Capital Partners, TCGX, Acuta Capital Partners, Kynam Capital Management, Octagon Capital, Invus, GordonMD Global Investments, and B Group Capital.
Dallas-based Taysha said that the $150 million expected from this PIPE financing plus the $45.1 million it has in cash and cash equivalents as of June 30, 2023, will extend its cash runway into the third quarter of 2025. The company will use the proceeds to advance its investigational gene therapy TSHA-102 in Rett syndrome and TSHA-120 in giant axonal neuropathy, as well as for working capital and general corporate expenses.
In parallel with announcing the PIPE financing, Taysha reported its Q2 2023 financial performance and provided updates on TSHA-102 and TSHA-120.
The company said it has received the go-ahead from an independent data monitoring committee to continue dosing adult Rett syndrome patients into the Phase I/II REVEAL study of TSHA-102, after the first patient dosed in the study appeared to tolerate the treatment and experience symptom improvements. The US Food and Drug Administration has also cleared Taysha's investigational new drug application for TSHA-102, allowing it to test the gene therapy in pediatric Rett syndrome patients. The firm has also submitted a clinical trial application to the Medicines and Healthcare Products Regulatory Agency, seeking permission to start a pediatric study for TSHA-102 in the UK.
TSHA-102, which is administered into the spinal canal fluid, uses adeno-associated virus 9 to deliver a shorter but functional version of the MECP2 gene, mutations in which cause the rare neurodevelopmental disorder. The gene therapy is designed to use Taysha's miRARE platform to adjust levels of the MECP2 protein and avoid adverse side effects.
The first adult patient who received TSHA-102 in the REVEAL study experienced no treatment-emergent serious adverse events six weeks after dosing. In a seven-point scale assessing clinical outcomes improvements, this patient had a score of 2, or "much improved." In a seven-point scale gauging disease severity, this patient had a one-point improvement four weeks after receiving the gene therapy. The patient didn't report any "quantifiable seizure events" through the fifth week after treatment, and after six weeks, investigators noted improvements in breathing patterns, sleep quality, and the ability to sit and hold objects.
"The patient was able to sit unassisted for the first time in over a decade, and she demonstrated the ability to unclasp her hands and hold an object steadily for the first time since infancy," Elsa Rossignol, a principal investigator in REVEAL and an associate professor of neuroscience and pediatrics at the Centre Hospitalier Universitaire Sainte-Justine in Montreal, said in a statement. "I believe that the patient achieving these milestones so early in treatment, coupled with the improvements in breathing patterns and quality of sleep that we have observed, are highly encouraging and support the potential of TSHA-102."
Taysha said it will dose the second patient with TSHA-102 in the REVEAL trial in Q3 and treat additional patients with the gene therapy in the second half of the year.
TSHA-120, Taysha's gene therapy for giant axonal neuropathy, an extremely rare genetic neurodegenerative disorder, hit a snag earlier this year, when the FDA said the firm needed to address patient heterogeneity in the progression of the disease. The agency also expressed concerns about the firm's use of the motor function measure 32 scale as an endpoint. However, Taysha developed a disease progression model using functional, electrophysiological, and biological endpoints and used it to conduct a new analysis within a Phase I study of the drug.
Taysha has submitted this data as part of a meeting request with the FDA and said it is expecting feedback on a regulatory path forward for TSHA-120 in Q3 2023.
Taysha currently has no marketed therapeutic products. In Q2 2023, the company reported $2.4 million in service revenue; it had no revenues in the comparable quarter in 2022. Taysha recorded a net loss of $24.6 million, or $.38 per share, compared to a net loss of $34.1 million, or $.85 per share, in Q2 2022.