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Praxis Precision Medicines (PRAX) shares cratered 75% on Monday, to $1.88 from $8.59 on the previous trading day of June 3, after the company announced plans to cut an unspecified number of jobs and pivot its R&D toward movement disorders and epilepsy. The moves followed Praxis’ acknowledgement that its lead PRAX-114 failed a Phase II/III trial.
PRAX-114 failed the ARIA Study (NCT04832425) by missing its primary endpoint of statistically significant change from baseline in the 17-item Hamilton Depression Rating Scale total score at Day 15, as well as all its secondary endpoints. The company did not disclose its data, saying it intends to share detailed results “in an upcoming medical presentation or publication.”
Praxis also said it was pulling back from three other clinical trials related to PRAX-114, by:
- Closing screening in its Phase II Acapella Study (NCT04969510), evaluating PRAX-114 for treatment of MDD at doses up to 60 mg, with the intent of reading out results from approximately 100 patients in the third quarter.
- Halting enrollment in another Phase II study (NCT05260541) assessing PRAX-114 for the treatment of post-traumatic stress disorder, with plans to review safety data once available.
- Also ending its Phase II crossover study (NCT05387642) designed to gauge the safety, pharmacokinetics and efficacy of daytime dosing of PRAX-114 for the treatment of essential tremor (ET).
Cutting operations and costs, Praxis said, will extend its cash runway into 2024, from the third quarter of 2023. The company didn’t quantify its current available cash and investments. But at the end of the first quarter on March 31, Praxis reported having $77.854 million in cash and cash equivalents—down 44% from $138.704 million at the end of Q4 2021. However, the company’s marketable securities rose 4%, to $144.662 million at end of Q1, from $137.207 million at the close of last year.
Praxis’ cash has shriveled from $296.608 million at the end of 2020, two months after the company went public through an initial public offering that generated approximately $200.3 million in net proceeds.
The near freefall of Praxis shares came just over two years after the company was launched with more than $100 million in financing from big-name sources like founding investor Blackstone Life Sciences (via prior Clarus funds in 2016) and Novo Holdings, as well as Vida Ventures, Eventide and other prominent funds.
PRAX-114 is an extrasynaptic GABAA receptor preferring positive allosteric modulator (PAM) under development for the potential treatment of MDD as well as perimenopausal depression.
PRAX-114 is one of two lead pipeline candidates for Praxis; the other is PRAX-944, a novel selective T-type calcium channel blocker designed to block abnormal neuron burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. PRAX-944 is under development for ET and Parkinson’s disease.
Through its strategic alignment, Praxis said, it will prioritize generating results from the Phase IIb Essential1 Study (NCT05021991) assessing PRAX-944 in ET; advancing its preclinical pipeline; and carrying out a proof-of-concept study for PRAX-562 in epilepsy. PRAX-562 is a preferential persistent sodium channel blocker currently in development for the potential treatment of rare adult cephalgias and Developmental and Epileptic Encephalopathies (DEEs).
Bluejay Diagnostics (BJDX)
Shares of Bluejay soared 26% on Thursday, from $1.29 to $1.63, the day it announced that the FDA had agreed to consider the company’s plan to pursue a De Novo submission for its Symphony IL-6 Test, designed to provide quantitative measurements of specific biomarkers to determine the need for additional patient care and monitoring.
Symphony IL-6 is based on Bluejay’s Symphone System™, which does not require any sample prep or dedicated staff and was shown in published clinical studies to deliver results in approximately 20 minutes. Bluejay expects to submit an application to market Symphony IL-6 “by year-end 2022,” CEO Neil Dey said in a statement.
The FDA can designate tests for De Novo review rather than require standard 510(k) clearance if the agency concludes there’s no legally marketed class I or class II device upon which to base a determination of substantial equivalence, and the device is considered to be of low to moderate risk.
Mirati Therapeutics (MRTX)
Shares of Mirati jumped 36% on Tuesday, from $43.16 to $58.89, the day after the company presented positive Phase Ib data at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting showing that its small-molecule KRASG12C inhibitor adagrasib (MRTX849) generated an intracranial (IC) response in about one-third of patients with neuro-oncology-brain metastases (modified RANO-BM) metastases—three complete responses and three partial responses out of 19 radiographically evaluable patients.
The data came from the Phase Ib cohort of the KRYSTAL-1 trial (NCT03785249), designed to assess IC responses of adagrasib in patients with KRASG12C-mutated non-small cell lung cancer (NSCLC) with active and untreated central nervous system (CNS) metastases.
Mirati released those results about 10 days after announcing initial results showing a 43% objective response rate (ORR) by blinded independent central review, an 80% disease control rate (DCR), an 8.5-month median duration of response (DOR), and a 6.5-month median progression-free survival (PFS).
Mirati said its efficacy results from KRYSTAL-1 were essentially identical to those of Amgen’s marketed drug Lumakras™ (sotorasib), which won FDA approval last year as a treatment for adults with KRASG12C -mutated locally advanced or metastatic NSCLC. In April, Amgen reported two-year analysis results for Lumakras showing an (ORR) of 40.7%, a DCR of 83.7%, a median DOR of 12.3 months, and median PFS of 6.3 months. Amgen also reported that five patients achieved complete responses and 65 patients achieved partial responses among 174 patients studied.
Adagrasib is being evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including NSCLC, colorectal cancer and pancreatic cancer.
Rigel Pharmaceuticals (RIGL)
Rigel shares plunged 60% on Wednesday, from $1.76 to 70 cents, after the company acknowledged that its lead pipeline drug fostamatinib failed the Phase III FORWARD trial (NCT03764618) in patients with warm autoimmune hemolytic anemia (wAIHA). Fostamatinib failed to show statistical significance in the primary efficacy endpoint of durable hemoglobin response response rate (16 of 45 fostamatinib patients vs. 12 of 45 placebo patients).
The company cited a post-hoc regional analysis of U.S., Canadian, Australian, and Western European trial sites, in which patients treated with fostamatinib showed a favorable durable hemoglobin response compared to placebo. However, patients in Eastern European sites did not. In a statement, Rigel President and CEO Raul Rodriguez said the results “were impacted by a large placebo response rate from Eastern European clinical sites.”
“We will continue to analyze the data and look forward to discussing our findings with the FDA,” Rodriguez added.
Yumanity Therapeutics (YMTX)
Shares of Yumanity jumped 40% on Monday, from $1.42 to $1.99, after the company announced a reverse merger with Kineta that will turn the privately-held Seattle biotech into a wholly-owned subsidiary of Boston-based Yumanity.
Kineta will hold 85% ownership of the combined company, which will take Kineta’s name upon the expected closing of the deal in the second half of this year. The new Kineta will also continue Yumanity’s collaboration with Merck & Co. to develop treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia—with the combined company standing to gain up to $500 million in milestone payments.
Kineta’s lead asset is KVA12.1, a VISTA blocking immunotherapy designed to address immunosuppression in the tumor microenvironment. The companies reason that KVA12.1 may effectively treat several types of cancer that include non-small cell lung cancer (NSCLC), colorectal, renal cell carcinoma, head and neck, and ovarian.
Also on Monday, Yumanity announced it will sell its first clinical-stage pipeline candidate, the SCD inhibitor YTX-7739 for Parkinson’s disease, as well as Yumanity’s unpartnered discovery-stage neuroscience product candidates and targets to Johnson & Johnson’s Janssen Pharmaceutica for $26 million cash.
Yumanity announced plans in February to pursue “strategic alternatives” and restructure its operations, in part by eliminating about 60% of its workforce by April. The moves came less than a month after the FDA imposed a partial hold on multi-dose clinical trials of YTX-7739, for reasons not disclosed in a January 19 announcement.