| Over a week ago | ||||
Taysha Gene Therapies… Taysha Gene Therapies presents preclinical data from neonatal mouse models on TSHA-102 for Rett syndrome, including new data in wild-type mice, at the American Society of Gene and Cell Therapy 26th Annual Meeting. TSHA-102 utilizes a miniMECP2 gene and a novel miRNA-Responsive Auto-Regulatory Element technology designed to regulate cellular MECP2 expression. In a Taysha-sponsored study, the safety and efficacy of TSHA-102 were explored in both neonatal wild-type and Mecp2-/Y knockout mice, respectively. Preclinical data in neonatal wild-type mice suggest miRARE suppressed toxic overexpression after early intervention with TSHA-102: In wild-type mice treated with TSHA-102, new data showed no deleterious impact on survival, neurobehavioral functions and overall health, suggesting miRARE regulated expression of MeCP2 with the below results from the study: No toxicity relative to vehicle treatment No reduction in survival over 36-weeks; No treatment effect on Bird Score (a measure of Rett syndrome-like behaviors and pathologies) analysis relative to vehicle treatment; No impact on overall growth over the course of the study. This builds on prior preclinical data in neonatal Mecp2-/Y knockout mice showing miRARE regulated MECP2 expression levels in deficient CNS cells with early intervention of TSHA-102: In Mecp2-/Y knockout mice treated with TSHA-102 with the below results from the study: 47% survived the 36-week study vs a median survival of 8.1 weeks with vehicle-treated knockout mice, representing a significant greater than4-fold extension of their lifespan; Restoration of normal and faster-than-normal growth; Aggregate Bird Score was significantly improved at several time points, with a significant delay in the onset of severe Rett syndrome-like phenotypes, including the delayed average age of onset for severe clasping from approximately 7 to 21 weeks and severely abnormal gait from approximately 8 to 20 weeks | ||||
Reports Q1 revenue… Reports Q1 revenue $4.706M, consensus $70,000. "We continue to make significant progress with our two lead clinical programs and remain on track to deliver on multiple key milestones, including the generation of first-in-human clinical data for TSHA-102 in adult patients with Rett syndrome, the submission of a CTA to the MHRA to initiate expansion of TSHA-102 in pediatric patients, the submission of an IND application to the FDA for TSHA-102, and obtaining further clarity from the FDA on the regulatory path forward for TSHA-120 in GAN," said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. "Screening is completed, and dosing is now scheduled for our first potential patient in the adult Rett syndrome study. For GAN, our comprehensive analyses of the totality of data for TSHA-120 continues to be encouraging and includes compelling findings with potential to further support a regulatory path forward." | ||||
| Over a month ago | ||||
Taysha Gene Therapies… Taysha Gene Therapies "announced that an abstract related to its TSHA-102 program in Rett syndrome was accepted for presentation at the upcoming American Society of Gene and Cell Therapy 26th Annual Meeting, taking place in Los Angeles, CA from May 16-20, 2023. The abstract includes new preclinical data from a Taysha-sponsored study for TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome, a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene." | ||||
Cantor Fitzgerald analyst… Cantor Fitzgerald analyst Kristen Kluska lowered the firm's price target on Taysha Gene Therapies (TSHA) to $2 from $13 and keeps an Overweight rating on the shares. The firm believes the stock has a significant amount of near-term risk, and strongly believes Taysha could become an M&A candidate in the near-term, potentially from Astellas (ALPMY), the analyst tells investors in a research note. While there is strong proof-of-concept on-hand for TSHA-120 in giant axonal neuropathy, at this stage it's possible the company may have to run an additional trial or wait a few months before a meeting with the FDA to discuss the regulatory path/receive minutes. | ||||
Chardan lowered the… Chardan lowered the firm's price target on Taysha Gene Therapies to $5 from $6 but keeps a Buy rating on the shares. The analyst is updating the firm's valuation while incorporating increased dilution from a modeled 2023 equity raise for the company. | ||||
Truist analyst Joon Lee… Truist analyst Joon Lee lowered the firm's price target on Taysha Gene Therapies to $3 from $4 to reflect the company's program delays but keeps a Buy rating on the shares. Given the recent positive commentaries from CBER's Peter Marks regarding the path to approving drugs for orphan diseases, there may be a "regulatory tailwind" for Taysha Gene Therapies' TSHA-120, the analyst tells investors in a research note. The firm adds that there "isn't much not to like" about the stock's current valuation. | ||||
Reports Q4 revenue $2.5M,… Reports Q4 revenue $2.5M, consensus $8.18M .Net loss for the three months ended December 31 was $55.7M, or 99c per share, as compared to a net loss of $50.4M or $1.32 per share, for the three months ended December 31, 2021. "The actions taken early this year to improve execution and expedite progress on our two lead clinical programs in Rett syndrome and GAN are having a positive effect," said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. "We recently initiated screening of the first potential adult subject for the REVEAL Rett syndrome trial and remain on track to dose the first patient and deliver initial available first-in-human adult data, primarily on safety, for TSHA-102 in the first half of the year. Additionally, we recently submitted a protocol amendment to allow patients as young as 15 years old to be included in the study, which we believe will further expedite enrollment. We remain on track to submit a CTA to the MHRA in mid-2023 to conduct a pediatric Rett syndrome trial, and plan to submit an IND to the FDA for Rett syndrome in the second half of 2023. For TSHA-120 in GAN, based on the constructive feedback recently received from the FDA in response to our follow up questions to the formal Type B end-of-Phase 2 meeting minutes, coupled with the positive preliminary assessment of the ongoing comprehensive data analyses, we plan to submit a formal meeting request to the Agency in the second quarter of 2023 to discuss the potential regulatory pathway forward for this ultra-rare disease with no approved treatment." | ||||
| Over a quarter ago | ||||
BTIG analyst Yun Zhong… BTIG analyst Yun Zhong downgraded Taysha Gene Therapies to Neutral from Buy without a price target. The analyst cites uncertainties in the regulatory pathway for the company's lead program in giant axonal neuropathy that could eventually result in program discontinuation in the downgrade. In addition, the delay in Taysha's Rett syndrome program due to operational issues increase the possibility that no meaningful clinical catalysts could be available by the end of the company's current cash runway into Q1 of 2024, the analyst tells investors in a research note. | ||||
Goldman Sachs analyst… Goldman Sachs analyst Salveen Richter suspended the firm's rating on Taysha Gene Therapies saying it has "insufficient information" on which to base an investment view. | ||||
