BioCryst Pharmaceuticals announced on January 7, 2022, that enrollment has begun in its Redeem-1 clinical trial.
BioCryst Begins Patient Enrollment in REDEEM-1 Pivotal Trial Evaluating BCX9930 as Oral Monotherapy for Patients with PNH
On January 7, 2022, BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced the enrollment of the first patient in the REDEEM-1 pivotal trial with its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
REDEEM-1 is a randomized, open-label, active comparator-controlled comparison of the efficacy and safety of BCX9930 (500 mg bid) monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. In part 1 of this trial, patients who have not had an adequate response to a C5 inhibitor will be randomized 2:1 to discontinue their C5 inhibitor and receive BCX9930 as monotherapy or to continue receiving their C5 inhibitor for 24 weeks. All patients will receive BCX9930 in part 2 (weeks 25-52) to assess the long-term safety, tolerability and effectiveness of BCX9930. Patients who are randomized to C5 inhibitor therapy in part 1 will discontinue that therapy at the week 24 visit and start BCX9930 for part 2. The primary endpoint of REDEEM-1 is change from baseline in hemoglobin, as assessed at weeks 12 to 24.
“On the heels of recently beginning enrollment in our REDEEM-2 pivotal trial, today’s announcement marks another important milestone as we advance BCX9930 closer to registration for patients living with PNH,” said Dr. William Sheridan, chief medical officer of BioCryst. “Given the unmet need patients have related to the current standard of care, we aim to demonstrate in the REDEEM-1 pivotal trial the potential of BCX9930 as an oral monotherapy that could represent a significant improvement for patients compared to their experiences with C5 inhibitor therapies.”
In a dose-ranging trial of BCX9930 in C5 inadequate response patients, the company previously reported that BCX9930 (at doses of 400 mg or 500 mg bid) increased hemoglobin from baseline by a mean of 2.7 g/dL through weeks 12 to 24 with 80 percent of patients being transfusion-free over the same period. BCX9930 was safe and generally well-tolerated in the trial.
BioCryst recently announced it had begun enrolling patients in the REDEEM-2 pivotal trial, a randomized, placebo-controlled trial to evaluate the efficacy and safety of BCX9930 (500 mg bid) as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. Additionally, the company is initiating a proof-of-concept trial of BCX9930 in renal complement-mediated diseases including C3 glomerulopathy (C3G), IgA nephropathy (IgAN) and primary membranous nephropathy (PMN).
The U.S. Food and Drug Administration has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. For more information about REDEEM-1, visit ClinicalTrials.gov and search NCT number NCT05116774.
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States, the European Union, Japan, the United Arab Emirates and the United Kingdom. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever. RAPIVAB® (peramivir injection) has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea.
Oppenheimer starts ‘underappreciated’ BioCryst at Outperform
On December 9, 2021, Oppenheimer analyst Justin Kim initiated coverage of BioCryst with an Outperform rating and $16 price target, calling it an “underappreciated commercial-stage company with pipeline growth potential.” He views BioCryst being a “near-term attractive bolt-on” given the medium-term commercial potential of Orladeyo and its long-run pipeline optionality, including with BCX9930, Kim added.
BioCryst Begins Patient Enrollment in REDEEM-2 Pivotal Trial Evaluating BCX9930 as Oral Monotherapy for Patients with PNH
On November 29, 2021, BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) announced the enrollment of the first patient in the REDEEM-2 pivotal trial with its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
REDEEM-2 is a randomized, placebo-controlled trial evaluating the efficacy and safety of BCX9930 (500 mg bid) as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. In part 1 of this trial, patients will be randomized 2:1 to receive BCX9930 or placebo under double-blind conditions for 12 weeks. All patients will receive BCX9930 in part 2 (weeks 13-52) to assess the long-term safety, tolerability and effectiveness of BCX9930, with patients randomized to placebo in part 1 switching to BCX9930 at the week 12 visit. The primary endpoint of REDEEM-2 is change from baseline in hemoglobin, as assessed at week 12.
“There are important unmet needs among patients living with PNH based on the current standard of care, specifically related to efficacy and burden of therapy. As an oral monotherapy with proof-of-concept data showing control of both intravascular and extravascular hemolysis, BCX9930 has the potential to help patients live their lives with more freedom from their disease,” said Dr. William Sheridan, chief medical officer of BioCryst.
“Beginning patient enrollment in our REDEEM-2 pivotal trial marks a critical step forward as BCX9930 advances closer to registration for patients living with PNH,” Sheridan added.
REDEEM-2 is powered at 90 percent to detect a difference in mean change from baseline of hemoglobin of ≥ 2.15 g/dL at 12 weeks. In a dose-ranging trial of BCX9930 in treatment-naïve patients, the company previously reported that BCX9930 (at doses of 400 mg or 500 mg bid) increased hemoglobin from baseline by a mean of 3.7 g/dL at week 12 and eliminated transfusions. BCX9930 was safe and generally well-tolerated in the trial.
BioCryst also is preparing to enroll patients in the REDEEM-1 pivotal trial, a randomized, open-label, active, comparator-controlled comparison of the efficacy and safety of BCX9930 (500 mg bid) monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. Additionally, the company is initiating a proof-of-concept trial of BCX9930 in renal complement-mediated diseases.
The U.S. Food and Drug Administration has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. For more information about REDEEM-2, visit ClinicalTrials.gov and search NCT number NCT05116787.
📺 BioCryst Pharmaceuticals 4th Annual Evercore ISI HealthCONx Conference
[embedyt] https://www.youtube.com/watch?v=cBTMNXXwXyk[/embedyt]📈 BCRX Stock Technical Analysis
Both the short-term and long-term trends are neutral. BCRX is one of the better-performing stocks in the Biotechnology industry, it outperforms 92% of 628 stocks in the same industry. BCRX is currently trading in the middle of its 52-week range. The S&P500 Index however is trading in the upper part of its 52-week range, so BCRX is lagging the market slightly.
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