CYP2D6

Fiscal year 2023 net product sales from prescriptions totaled approximately $5.2M, a 107% increase from 2022 Company projects $14M in net revenue for 2024 SOLANA BEACH, Calif., March 14, 2024 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ: EVOK), a specialty pharmaceutical company focused primarily on treatments for gastrointestinal (GI) diseases with an emphasis on GIMOTI® (metoclopramide) nasal spray, today announced its financial results for the fourth quarter and full year ended December 31, 2023, and recent corporate developments. "In 2023, we focused on operational excellence to expand GIMOTI’s market share and ensure its availability for patients requiring an improved gastroparesis treatment. Our commercial team’s dedicated efforts yielded a 107% increase in year-over-year revenue,” stated Dave Gonyer, R.Ph., CEO of Evoke Pharma. “The new Healthcare Resource Utilization (HCRU) data has only been available to our commercial team since near the end of the year. We believe it is just starting to drive growth beginning with the addition of new prescribers, a rise in prescription fills, and an uptick in patient enrollments, solidifying 2023 as an important year for our product’s market presence. As of December 31, 2023, we’ve seen a 66% rise in cumulative prescribers from the previous year, totaling 1,689. Prescription fills and patient enrollments also climbed significantly by 100% and 88% respectively, year-over-year,” Mr. Gonyer added. Fourth Quarter and Full Year 2023 Developments and Recent Highlights: Continued Advocacy for GIMOTI as the Standard of Care for Diabetic Gastroparesis Treatment Healthcare resource utilization data presented at DDW 2023 revealed that there is statistically signficantly less burden on healthcare resources and facilities such as hospitical admissions, emergency department and physician office visits with patients who are being prescirbed GIMOTI versus those on oral metoclopramide.Based on the reduced utilization data, additional compelling cost data presented in a distinguished plenary session at ACG 2023 demonstrated financial savings benefits of GIMOTI for patients and payors compared to oral metoclopramide.Abstract focused on the HCRU of diabetic gastroparesis care in women using nasal metoclopramide to be presented at DDW 2024. Transitioned Pharmacy Service Partnership to ASPN Pharmacies Aiming to enhance patient prescription process and enhance revenues through ASPN’s extensive network of partners with broader PBM agreements. Fortified Patent Estate for GIMOTI Two patents issued in 2023 covering the methods of use for GIMOTI including two Orange Book listings.Teva Pharmaceuticals determination not to pursue a paragraph 4 certification against Gimoti without financial or other consideration further enhanced Evoke’s intellectual property position and eliminated 180-day exclusivity opportunity for later possible generic seeking entities for the future. Improved Cash Position In February 2024, the Company closed a $7.5M public offering with fundamental, healthcare-oriented institutional investors providing the company runway into the fourth quarter of 2024 with up to an additional $22.5M available if common stock warrants are exercised in full. Mr. Gonyer concluded, “2023 was a milestone year for GIMOTI with new real-world evidence demonstrating its superiority over traditional treatments and data showing a marked decrease in hospitalization and ER visits for GIMOTI over oral metoclopramide which translated into savings of over $15,000 per patient in only six months. Our results at major gastroenterology conferences including Digestive Disease Week 2023 have strengthened market trust in GIMOTI, bolstering our goal to make it the standard treatment for diabetic gastroparesis. Current and new IP protections, coupled with new inquiries about the prevalence GLP-1 associated diabetic gastroparesis, and the recent capital boost from our public offering further position us to scale our operations and achieve sustained business growth.” Fourth Quarter and Full Year 2023 Financial Review and Outlook For the fourth quarter of 2023, net product sales were approximately $1.7 million compared with $0.8 million during the fourth quarter of 2022, and the net loss was approximately $2.0 million, or $0.59 per share compared with $1.8 million, or $0.54 per share, for the fourth quarter of 2022. For the year ended December 31, 2023, net product sales were approximately $5.2 million compared with approximately $2.5 million for the year ended December 31, 2022, and the net loss was approximately $7.8 million, or $2.33 per share, compared with a net loss of $8.2 million, or $2.62 per share, for the year ended December 31, 2022. The year-over-year increase in revenue was due to higher net product sales in 2023, resulting from: Prescription sales through pharmacy service partnership with ASPN Pharmacy;Recapture of prescriptions sent to retail pharmacies without ability to order product; andMarketing of head-to-head real-world data comparing Gimoti to oral metoclopramide showing improvements in fewer hospitalizations and ER visits with GIMOTI. Research and development expenses totaled approximately $23,000 for the fourth quarter of 2023 compared with $27,000 for the fourth quarter of 2022. For the full year of 2023, research and development expenses were approximately $0.2 million compared with approximately $0.3 million for the prior year. For the fourth quarter of 2023 selling, general and administrative (SG&A) expenses were approximately $3.5 million compared with $2.3 million for the fourth quarter of 2022. For the year ended December 31, 2023, selling, general and administrative expenses were approximately $12.2 million versus approximately $9.6 million for the year ended December 31, 2022. The increase in SG&A costs year-over-year resulted primarily from higher marketing and Eversana profit sharing costs. We expect that selling, general and administrative expenses will increase in the future as we continue to progress with the commercialization of GIMOTI. Total operating expenses for the fourth quarter of 2023 were approximately $3.6 million compared with $2.3 million for the same period of 2022. For the year ended December 31, 2023, total operating expenses were approximately $12.6 million compared with approximately $10.3 million for the full year of 2022. As of December 31, 2023, cash and cash equivalents were approximately $4.7 million. We believe, based on our current operating plan, that our existing cash and cash equivalents, including the proceeds of approximately $6.1 million from the public offering closed in February 2024, as well as future cash flows from net product sales of Gimoti, will be sufficient to fund our operations into the fourth quarter of 2024. Evoke projects net revenue in 2024 of approximately $14 million. Evoke’s 2024 guidance is dependent on its current business and expectations, including recent growth rates in net sales, assumptions regarding reimbursements and prescription fills, as well as factors that are outside of our control, such as the global macroeconomic and geopolitical environment, continued supply chain constraints and inflationary pressures. About Evoke Pharma, Inc. Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The company developed, commercialized and markets GIMOTI, a nasal spray formulation of metoclopramide, for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adults. Diabetic gastroparesis is a GI disorder affecting millions of patients worldwide, in which the stomach takes too long to empty its contents resulting in serious GI symptoms as well as other systemic complications. The gastric delay caused by gastroparesis can compromise absorption of orally administered medications. Prior to FDA approval to commercially market GIMOTI, metoclopramide was only available in oral and injectable formulations and remains the only drug currently approved in the United States to treat gastroparesis. Visit www.EvokePharma.com for more information.Follow GIMOTI on FacebookFollow Evoke Pharma on FacebookFollow Evoke Pharma on LinkedInFollow Evoke Pharma on Twitter About Gimoti® (metoclopramide) nasal spray GIMOTI is indicated for the relief of symptoms in adults with acute and recurrent diabetic gastroparesis. Important Safety InformationWARNING: TARDIVE DYSKINESIA Metoclopramide can cause tardive dyskinesia (TD), a serious movement disorder that is often irreversible. The risk of developing TD increases with duration of treatment and total cumulative dosage. Discontinue GIMOTI in patients who develop signs or symptoms of TD. In some patients, symptoms may lessen or resolve after metoclopramide is stopped. Avoid treatment with metoclopramide (all dosage forms and routes of administration) for longer than 12 weeks because of the increased risk of developing TD with longer-term use. GIMOTI is not recommended for use in: Pediatric patients due to the risk of developing tardive dyskinesia (TD) and other extrapyramidal symptoms as well as the risk of methemoglobinemia in neonates. Moderate or severe hepatic impairment (Child-Pugh B or C), moderate or severe renal impairment (creatinine clearance less than 60 mL/minute), and patients concurrently using strong CYP2D6 inhibitors due to the risk of increased drug exposure and adverse reactions. GIMOTI is contraindicated: In patients with a history of tardive dyskinesia (TD) or a dystonic reaction to metoclopramide. When stimulation of gastrointestinal motility might be dangerous (e.g., in the presence of gastrointestinal hemorrhage mechanical obstruction, or perforation). In patients with pheochromocytoma or other catecholamine-releasing paragangliomas. Metoclopramide may cause a hypertensive/pheochromocytoma crisis, probably due to release of catecholamines from the tumor. In patients with epilepsy. Metoclopramide may increase the frequency and severity of seizures. In patients with hypersensitivity to metoclopramide. Reactions have included laryngeal and glossal angioedema and bronchospasm. Potential adverse reactions associated with metoclopramide include: Tardive dyskinesia (TD), other extrapyramidal effects (EPS), parkinsonism symptoms, motor restlessness, neuroleptic malignant syndrome (NMS), depression, suicidal ideation and suicide, hypertension, fluid retention, hyperprolactinemia, effects on the ability to drive and operate machinery. Most common adverse reactions (≥5%) for GIMOTI are: dysgeusia, headache, and fatigue. These are not all of the possible side effects of GIMOTI. Call your doctor for medical advice about whether you should take GIMOTI and the possible risk factors and side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Safe Harbor Statement Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negatives of these terms or other similar expressions. These statements are based on the company’s current beliefs and expectations. These forward-looking statements include statements regarding: guidance regarding 2024 net product sales; potential future prescribing trends for GIMOTI based on Evoke’s or EVERSANA’s marketing efforts; Evoke’s commercialization plans, including the potential that GIMOTI could become the standard of care for gastroparesis; the potential for additional funds from the exercise of outstanding warrants and Evoke’s expected cash runway. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke’s business, including, without limitation: Evoke may not be able to achieve it’s guidance for 2024 including as a result of decreased demand for GIMOTI; Evoke’s and EVERSANA’s ability to successfully drive market demand for GIMOTI; Evoke’s ability to obtain additional financing as needed to support its operations; Evoke may use its capital resources sooner than expected; warrant holders may choose not to exercise any of the outstanding warrants; Evoke’s dependence on third parties for the manufacture of GIMOTI; Evoke is entirely dependent on the success of GIMOTI; inadequate efficacy or unexpected adverse side effects relating to GIMOTI that could result in recalls or product liability claims; Evoke’s ability to maintain intellectual property protection for GIMOTI; and other risks and uncertainties detailed in Evoke’s prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor & Media Contact:Daniel Kontoh-Boateng DKB PartnersTel: 862-213-1398dboateng@dkbpartners.net Financial Statements to Follow Evoke Pharma, Inc.Balance Sheet Evoke Pharma, Inc.Statement of Operations The Financial Statements accompanying this announcement are available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/1505239a-5694-4a7e-99c2-8d96e50a84a3 https://www.globenewswire.com/NewsRoom/AttachmentNg/b26e2618-665c-4966-acc6-c45d683cb7e0

SOLANA BEACH, Calif., March 07, 2024 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc., (NASDAQ: EVOK), a specialty pharmaceutical company focused on treatments for gastrointestinal (GI) diseases with an emphasis on Gimoti® (metoclopramide) nasal spray, today announced that the Company is scheduled to release its fourth quarter and full year 2023 on Thursday, March 14, 2024, after the market closes. Management will host a conference call and webcast with accompanying slides on Thursday, March 14, 2024, at 4:30 p.m. ET to discuss the results. The dial-in numbers for the conference call are (800) 267-6316 and (203) 518-9783 for international callers. The conference ID number is EVOKQ423. The webcast details can be found in the events section of Evoke’s investor relations page. In addition, a telephonic replay of the call will be available until March 21, 2024. The replay dial-in numbers are (888) 562-3381 for domestic callers and (402) 220-1189 for international callers. About Evoke Pharma, Inc. Evoke is a specialty pharmaceutical company focused primarily on the development of drugs to treat GI disorders and diseases. The company developed, commercialized and markets GIMOTI, a nasal spray formulation of metoclopramide, for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adults. Diabetic gastroparesis is a GI disorder affecting millions of patients worldwide, in which the stomach takes too long to empty its contents resulting in serious GI symptoms as well as other systemic complications. The gastric delay caused by gastroparesis can compromise absorption of orally administered medications. Prior to FDA approval to commercially market GIMOTI, metoclopramide was only available in oral and injectable formulations and remains the only drug currently approved in the United States to treat gastroparesis. Visit for more information. Follow GIMOTI on Facebook Follow Evoke Pharma on Facebook Follow Evoke Pharma on LinkedIn Follow Evoke Pharma on Twitter About Gimoti® (metoclopramide) nasal spray GIMOTI is indicated for the relief of symptoms in adults with acute and recurrent diabetic gastroparesis. Important Safety Information WARNING: TARDIVE DYSKINESIA Metoclopramide can cause tardive dyskinesia (TD), a serious movement disorder that is often irreversible. The risk of developing TD increases with duration of treatment and total cumulative dosage. Discontinue GIMOTI in patients who develop signs or symptoms of TD. In some patients, symptoms may lessen or resolve after metoclopramide is stopped. Avoid treatment with metoclopramide (all dosage forms and routes of administration) for longer than 12 weeks because of the increased risk of developing TD with longer-term use. GIMOTI is not recommended for use in: Pediatric patients due to the risk of developing tardive dyskinesia (TD) and other extrapyramidal symptoms as well as the risk of methemoglobinemia in neonates. Moderate or severe hepatic impairment (Child-Pugh B or C), moderate or severe renal impairment (creatinine clearance less than 60 mL/minute), and patients concurrently using strong CYP2D6 inhibitors due to the risk of increased drug exposure and adverse reactions. GIMOTI is contraindicated: In patients with a history of tardive dyskinesia (TD) or a dystonic reaction to metoclopramide. When stimulation of gastrointestinal motility might be dangerous (e.g., in the presence of gastrointestinal hemorrhage mechanical obstruction, or perforation). In patients with pheochromocytoma or other catecholamine-releasing paragangliomas. Metoclopramide may cause a hypertensive/pheochromocytoma crisis, probably due to release of catecholamines from the tumor. In patients with epilepsy. Metoclopramide may increase the frequency and severity of seizures. In patients with hypersensitivity to metoclopramide. Reactions have included laryngeal and glossal angioedema and bronchospasm. Potential adverse reactions associated with metoclopramide include: Tardive dyskinesia (TD), other extrapyramidal effects (EPS), parkinsonism symptoms, motor restlessness, neuroleptic malignant syndrome (NMS), depression, suicidal ideation and suicide, hypertension, fluid retention, hyperprolactinemia, effects on the ability to drive and operate machinery. Most common adverse reactions (≥5%) for GIMOTI are: dysgeusia, headache, and fatigue. These are not all of the possible side effects of GIMOTI. Call your doctor for medical advice about whether you should take GIMOTI and the possible risk factors and side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. Safe Harbor Statement Evoke cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negatives of these terms or other similar expressions. These statements are based on the company’s current beliefs and expectations. These forward-looking statements include statements regarding: potential future prescribing trends for GIMOTI based on Evoke’s or EVERSANA’s marketing efforts; Evoke’s commercialization plans, including its plans to increase awareness of and access to GIMOTI; and Evoke’s future capital requirements. The inclusion of forward-looking statements should not be regarded as a representation by Evoke that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Evoke’s business, including, without limitation: Evoke’s and EVERSANA’s ability to successfully drive market demand for GIMOTI; Evoke’s ability to obtain additional financing as needed to support its operations; the COVID-19 pandemic may continue to disrupt Evoke’s and EVERSANA’s business operations impairing the ability to commercialize GIMOTI and Evoke’s ability to generate product revenue; Evoke’s dependence on third parties for the manufacture of GIMOTI; Evoke is entirely dependent on the success of GIMOTI; inadequate efficacy or unexpected adverse side effects relating to GIMOTI that could result in recalls or product liability claims; Evoke’s ability to maintain intellectual property protection for GIMOTI; and other risks and uncertainties detailed in Evoke’s prior press releases and in the periodic reports it files with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Evoke undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor & Media Contact: Daniel Kontoh-Boateng DKB Partners Tel: 862-213-1398 dboateng@dkbpartners.net

RESEARCH TRIANGLE PARK, N.C., Feb. 23, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new analyses of real-world use of oral, once-daily ORLADEYO® (berotralstat) that showed patients who initiated ORLADEYO experienced rapid, substantial and sustained reductions in attack rates through 18 months of treatment regardless of the severity of their disease, their history of prior prophylaxis or their C1-inhibitor (C1-INH) level and function. The data are being presented in five posters at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting, which is being held at the Walter E. Washington Convention Center in Washington, D.C., from February 23-26, 2024. “These additional analyses of real-world use of ORLADEYO show that any person living with HAE has the potential to experience a rapid, substantial and sustained reduction in their monthly attack rate with ORLADEYO. From patients who live with severe disease to well-controlled patients and those who have a history of being treated with other long-term prophylaxis that carry a therapeutic burden, these data demonstrate that once patients begin oral, once-daily ORLADEYO, they can experience attack control over the duration of their treatment,” said Jonathan Bernstein, M.D., professor of medicine, department of internal medicine, division of allergy & immunology at the University of Cincinnati and partner of the Bernstein Allergy Group and Bernstein Clinical Research Center. Rapid, Substantial and Sustained HAE Attack Rate Reduction After Beginning ORLADEYO Across Multiple Patient Types Three posters (#012; #008; #281) highlight data collected through BioCryst’s sole-source pharmacy that show real-world effectiveness outcomes for patients aged 12 and above with HAE who initiated ORLADEYO in the United States. These analyses present the overall attack rate progression and attack rate progression stratified by severity (i.e., number of attacks at baseline), prior prophylaxis and C1-INH level and function. “We are continuing to see strong disease control with ORLADEYO in the real world, including in patients with HAE who report differing baseline disease severities. These findings further demonstrate that ORLADEYO can help maintain disease control in patients with lower baseline attack rates and further reduce attack rates in patients with more active disease. We continue to be encouraged by the consistent, building body of real-world evidence demonstrating the significant benefit that our oral, once-daily prophylactic therapy can provide to people living with HAE,” said Dr. Ryan Arnold, chief medical officer of BioCryst. Berotralstat Prophylaxis Reduces HAE Attack Rates Regardless of Baseline Attacks: Real-World Outcomes; Poster #012; Friday, February 23, 3:15-4:15 p.m. ET Patients with C1-INH deficiency who received long-term prophylaxis with ORLADEYO achieved rapid reduction in patient-reported monthly HAE attack rates from baseline, regardless of the severity of their disease (n=335).Median attack rates decreased below baseline (1.33 median monthly attack rate) in the first 90 days of ORLADEYO treatment and remained below baseline across all additional 90-day intervals for up to 18 months (540 days) (0.50 median monthly attack rate).Additionally, when stratified by baseline attacks, median monthly attack rates decreased and remained below baseline for up to 540 days on ORLADEYO regardless of their attack rate at baseline. This was observed across patients who reported a 90-day baseline of 0 attacks (0 attacks/month at all time points except for 0.33 at Days 271-360) to those who reported a 90-day baseline of ≥10 attacks (>3.33 attacks/month at baseline to 1.58 attacks/month at Days 1-90, concluding at 1.50 attacks/month at Days 451-540). Consistently Low Hereditary Angioedema Attack Rates with Berotralstat Regardless of Prior Prophylaxis: Real-World Outcomes; Poster #008; Friday, February 23, 3:15-4:15 p.m. ET Patients who initiated ORLADEYO and were previously on at least one other prophylactic therapy at some point during their lifetime (n=216) also experienced rapid, substantial and sustained reductions in HAE attack rates regardless of prior prophylactic therapy, including lanadelumab, subcutaneous (SC) C1-INH and androgens.Of note, the median monthly attack rate for patients who were previously treated with lanadelumab decreased from 1.00 at baseline (n=66) to 0.33 at Days 1-90 (n=71) after initiating ORLADEYO and remained below baseline through Days 451-540 (0.50; n=21). Real-World Effectiveness of Berotralstat in HAE With and Without C1-Inhibitor Deficiency; Poster #281; Saturday, February 24, 9:45-10:45 a.m. ET Similarly, a reduction in monthly HAE attack rates was observed in patients with HAE who have normal C1-INH level and function (n=302) and those with C1-INH deficiency (n=402) upon initiating ORLADEYO.Patients with normal C1-INH had a median attack rate at baseline of 3.00 attacks/month (n=249), which was reduced to 1.00 at Days 1-90 (n=277) and remained below baseline through Days 451-540 (1.50; n=79), while patients with C1-INH deficiency had a median attack rate at baseline of 1.33 attacks/month (n=335), which was reduced to and remained at 0.50 through Days 451-540 (n=119). Methods These posters highlight outcomes collected through BioCryst’s sole-source pharmacy and include patients who actively received ORLADEYO 110 or 150 mg QD from December 16, 2020 to June 15, 2023.Patient-reported attack rates were collected at baseline and at each refill (approximately every 30 days).The baseline 30-day average was calculated based on each patient’s self-reported attack rate for the 90 days prior to initiating ORLADEYO and by dividing that value by three.Monthly attack rates were calculated by taking the average of the reported attacks across each 90-day period.Not all patients reported a baseline attack rate or attack rates during each 90-day period. Real-world Data from French Cohorts Support Safety, Effectiveness and Adherence to ORLADEYO Two additional posters (#028; #023) highlight findings from real-world studies in patients with HAE aged 12 and above in France, including an observational study (BeroLife) and the ongoing prospective MATCH study (Monitoring HAE Treatment Compliance by the community pHarmacist). “These new findings from the BeroLife and MATCH studies indicate an association between adherence to therapy and effectiveness of ORLADEYO. Of note, the results from MATCH show that the number of patients who had high adherence to ORLADEYO after one month of treatment was comparable to the adherence rate among patients who take medications for chronic diseases in France, but this number increased the longer they remained on treatment. Additionally, patients who participated in these studies had a low discontinuation rate, which supports our previously reported findings that the longer patients remain on ORLADEYO, the better their outcomes are,” continued Dr. Ryan Arnold. Assessment of the Tolerability and Effectiveness of Berotralstat for Long-term Prophylaxis in Hereditary Angioedema: BeroLife Study Interim Analysis; Poster #028; Friday, February 23, 3:15-4:15 p.m. ET An interim analysis that shows long-term prophylaxis with ORLADEYO was generally well tolerated and safety was consistent with what has previously been reported from clinical trials. Only 15.6 percent (n=10) experienced a drug-related treatment-emergent adverse event (TEAE), and fewer than 10 percent discontinued ORLADEYO due to a TEAE (n=6).From an effectiveness standpoint, this analysis shows a decrease in HAE attack rates among patients, the majority of whom had already been treated with previous long-term prophylaxis such as attenuated androgens, tranexamic acid and lanadelumab, which is consistent with what was observed in clinical trials with long-term prophylaxis with ORLADEYO. Final data from the BeroLife study are expected later this year. Evaluation of Adherence to Berotralstat in Patients with Hereditary Angioedema: A Prospective Survey in Community Pharmacies; Poster #023; Friday, February 23, 3:15-4:15 p.m. ET An association between adherence and effectiveness of ORLADEYO was observed when taken by patients who received follow-up engagements from a pharmacist each month for six months (n=23).No association was found between adherence to ORLADEYO and the incidence of adverse events. Additionally, no serious drug-related TEAEs occurred and only 12.8 percent of patients (n=6) discontinued treatment during the study. Methods The BeroLife study assesses the safety and effectiveness of ORLADEYO 150 mg QD in a French cohort of patients ages 12 years and older with HAE Type I or Type II (n=64). Data were collected by 18 physician investigators associated with the Reference Centre for Angioedema (CREAK) Expert Network in France from November 2021 to May 2023.The MATCH study assesses the impact of monthly follow-up and therapeutic monitoring by community pharmacists in France on patient adherence to ORLADEYO 150 mg QD (n=47). Adherence is measured using the 8-item Morisky Medication Adherence Scale (MMAS-8), a validated method of determining adherence to treatment through a self-reported measurement of medication-taking behavior. All posters are available to meeting registrants and will be on display in the poster hall in the Walter E. Washington Convention Center (Level 2, Hall D) during the meeting. About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein. U.S. Indication and Important Safety Information INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older. Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation. IMPORTANT SAFETY INFORMATION An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent. The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease. A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C). Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO. ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO. The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established. There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production. To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please see full Prescribing Information. About BioCryst Pharmaceuticals BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: the ongoing COVID-19 pandemic, which could create challenges in all aspects of BioCryst’s business, including without limitation delays, stoppages, difficulties and increased expenses with respect to BioCryst’s and its partners’ development, regulatory processes and supply chains, negatively impact BioCryst’s ability to access the capital or credit markets to finance its operations, or have the effect of heightening many of the risks described below or in the documents BioCryst files periodically with the Securities and Exchange Commission; BioCryst’s ability to successfully implement its commercialization plans for, and to commercialize, ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; risks related to the international expansion of BioCryst’s business; and actual financial results may not be consistent with expectations, including that revenue, operating expenses and cash usage may not be within management's expected ranges. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements. BCRXW Contact:John Bluth+1 919 859 7910jbluth@biocryst.com