Tonix Pharmaceuticals trading resumes 17:1007/2307/23/21
Tonix Pharmaceuticals to stop enrollment in Phase 3 RALLY study of TNX-102 SL » 16:4107/2307/23/21
Tonix Pharmaceuticals Holding Corp. announced that the company has decided to stop enrollment in the Phase 3 RALLY study of TNX-102 SL 5.6 mg for the management of fibromyalgia following an unblinded, pre-planned interim analysis by the Independent Data Monitoring Committee of the RALLY study. Based on interim analysis results of the first 50% enrolled participants, the IDMC recommended stopping the trial for futility as TNX-102 SL is unlikely to demonstrate a statistically significant improvement in the primary endpoint of overall change from baseline in daily diary pain severity scores between those treated with TNX-102 SL 5.6 mg and those receiving placebo. Tonix remains blinded to the detailed interim analysis results and only received the recommendation made by the IDMC. Preliminary blinded safety data from these participants did not reveal any new safety signals, and the decision to discontinue enrolling new participants is not related to safety. The company intends to continue studying those participants currently enrolled until completion and then proceed with a full analysis of the unblinded data, with the topline results expected to be reported in the fourth quarter of 2021, to determine the next steps in this program. "We are surprised and disappointed that the interim analysis did not support continued enrollment in this Phase 3 RALLY study, especially considering the previous Phase 3 RELIEF study, which had a similar design and achieved statistical significance on the primary endpoint. After the currently enrolled participants complete the study, we will proceed with a full analysis of the unblinded data from the study to determine the next steps in this program," commented Seth Lederman, M.D., President and Chief Executive Officer. "These results underscore the difficulty in managing and treating fibromyalgia. We thank the patients, caregivers and investigators who participated in the RALLY study."
Tonix Pharmaceuticals trading halted, news pending 16:4007/2307/23/21
Incorrect headline could be cause of Selecta weakness, says Cantor Fitzgerald » 16:0207/2207/22/21
Cantor Fitzgerald analyst…
Cantor Fitzgerald analyst Kristen Kluska notes that Seleta Biosciences (SELB) shares have been trading down on Thursday, despite the company not reporting any news-related items. The analyst thinks shares are trading down as a result of a Bloomberg article highlighting the setback reported in the microbiome space from Seres (MCRB). Kluska says "the article incorrectly lists Selecta Biosciences as a related ticker." as Selecta is "not a microbiome-based company." Overall, the analyst sees "no fundamental reasons" for the weakness in the shares and reiterates an Overweight rating and a price target of $9 on the stock.
Catalyst Biosciences announce first patient screened for CFI deficiency in study » 08:2107/2207/22/21
Catalyst Biosciences announced the screening of the first patient in its CFI-deficiency study in the CB 4332 program, its wholly-owned, first-in-class, enhanced Complement Factor I, intended for prophylactic subcutaneous administration in individuals with CFI deficiency. "The findings from the CFI deficiency screening and natural history of disease studies will be instrumental in identifying patients for the Phase 1/2 trial of CB 4332, planned for mid-year 2022," said Nassim Usman, Ph.D., president and chief executive officer of Catalyst. "Following the disease manifestations and biomarkers of this complement disorder will be important in unlocking the full therapeutic potential of CB 4332." The ConFIrm screening study will measure CFI levels and activity in patients who have diseases related to a CFI deficiency and who may potentially benefit from CB 4332 treatment. The ConFIdence natural history of disease study will follow these CFI-deficient subjects, who often present with repetitive bacterial infections, immune-related diseases, and/or glomerulopathies, for clinical biomarkers and safety of current treatments. The findings from these studies will identify opportunities to potentially develop CB 4332 for treatment in multiple indications.
Annexon advances ANX009 into Lupus Nephritis, ANX005 into MMN » 07:2107/2207/22/21
Annexon announced the…
Annexon announced the broadening of its autoimmune franchise through advancement of its third clinical-stage product candidate, ANX009, into Lupus Nephritis and expansion of the ANX005 clinical program into a second neuromuscular condition, Multifocal Motor Neuropathy, or MMN. Annexon's third clinical-stage product candidate, ANX009, is a subcutaneous C1q inhibitor developed for antibody-mediated autoimmune diseases of blood and vascular tissues. In a recently completed Phase 1 first-in-human study of ANX009 in healthy volunteers, the dose-escalation study demonstrated: ANX009 was well-tolerated at all dose levels and no drug-related safety signals were observed; A clear dose response in single ascending dose cohorts, with robust C1q inhibition at higher doses; Sustained C1q inhibition with multiple doses, supporting the potential for twice weekly subcutaneous administration with the current formulation. No serious adverse events, discontinuations related to treatment or dose limiting toxicity were observed. Some participants reported mild, transient localized subcutaneous injection site reactions. The company now plans to advance ANX009 into a Phase 1b study in Lupus Nephritis patients in early 2022. Annexon also plans to advance ANX005 into a Phase 2 trial in MMN patients in early 2022. In a preclinical model of MMN, C1q inhibition blocks nerve damage induced by IgM autoantibodies from patient sera. Moreover, MMN is mechanistically related to Guillain-Barre Syndrome, an antibody-mediated autoimmune disorder that causes acute neuromuscular paralysis driven also by IgM autoantibodies targeting peripheral nerve axons and myelin sheaths that spur nerve destruction and impaired conduction. ANX005 demonstrated proof-of-concept in a placebo-controlled Phase 1b trial in GBS, and Annexon is leveraging those learnings and the prominent overlap in pathology between GBS and MMN in the upcoming MMN Phase 2 trial.
Zogenix's FINTEPLA recognized by clinicians in editorial » 08:1007/2107/21/21
Zogenix announced that…
Zogenix announced that its product FINTEPLA oral solution has been recognized by two distinguished clinicians in an Epilepsy & Behavior editorial titled "Raising the Bar: Fenfluramine Sets New Treatment Standards for Dravet Syndrome." In the editorial, the authors, Joseph Sullivan, M.D., of the UCSF Benioff Children's Hospitals, and Helen Cross, M.B.,Ch.B. Ph.D., of the UK's UCL Institute of Child Health, note that the treatment and diagnosis of severe, rare epilepsies such as Dravet syndrome have improved in the U.S. and Europe with earlier accurate diagnosis, better access to a network of specialized medical care, and availability of new medications. In particular, they note that FINTEPLA has achieved responder rates for seizure reduction at the greater than/=75% level that were previously only observed at the greater than/= 50% level. In their assessment, the authors write that future Dravet syndrome treatments "should be evaluated against the demonstrated efficacy of fenfluramine." FINTEPLA was approved in the U.S. and European Union in 2020 for the treatment of seizures associated with Dravet syndrome patients aged two years and older. The authors write that the novel outcomes reported with FINTEPLA treatment, such as effects on executive functions, have effectively raised the bar for assessment of future therapies. Their opinion is based on the body of previously released data from the clinical trials that supported the U.S. and EU regulatory reviews, as well as data from patients treated up to three years in an ongoing open-label study and in early access programs. Across studies, the data demonstrate that FINTEPLA provides safe, effective, and durable seizure reduction for a majority of treated Dravet syndrome patients, including those who, prior to treatment with FINTEPLA, had continued to experience a high seizure burden despite treatment with one or more other antiepileptic medicines.Across studies, FINTEPLA has been generally well-tolerated, with no observed cases of pulmonary arterial hypertension or valvular heart disease. The most common adverse events were reported as decreased appetite, fatigue, diarrhea, and pyrexia.
Aytu BioScience management to meet virtually with Cantor Fitzgerald » 04:5507/2007/20/21
Virtual Meetings to be…
Virtual Meetings to be held on July 19-20 hosted by Cantor Fitzgerald.
Piper remains Overweight on Catalyst Biosciences following R&D day » 16:0607/1907/19/21
Piper Sandler analyst…
Piper Sandler analyst Christopher Raymond keeps an Overweight rating on Catalyst Biosciences shares following the company's R&D day focused on its complement platform and particularly CB 4332, noting that its Phase 1/2 development is expected to start next year. Raymond continues to recommend purchasing Catalyst Biosciences shares to levels approaching his $15 per share price target given what he sees "as an ongoing valuation disconnect based on the overall stage of this company," the analyst said.
Quidel not commenting on report claiming OraSure rejected takeover approach » 14:2307/1907/19/21
Contacted by The Fly…
Contacted by The Fly after Street Insider said it was told by a source that OraSure (OSUR) rejected a recent takeover approach made by Quidel (QDEL), a spokesperson for the latter said the company does "not respond to rumors regarding M&A." In afternoon trading, shares of Quidel have gained almost 8% to $134.60, while OraSure's stock has advanced more than 4% to $10.52.