CYP2D6

Analysis of real-world data compared patients on GIMOTI (n=51) to Oral Metoclopramide (n=41), both taking GLP-1s, showing significant statistical improvement for GIMOTI over Oral Metoclopramide in All Cause Emergency Department Visits (-91%, p=0.001), All Cause Office Visits (-41%, p=0.027) and All Cause Hospital Outpatient Visits (-89%, p=0.032) within 6-month index period Data presented at American College of Gastroenterology (ACG) 2024; the submission garnered both the Presidential Poster Award as one of the top 5% of data accepted to the conference and selected as the Outstanding Research Award in the Stomach Category First study to show Gimoti’s potential as supportive care for GLP-1 therapy SOLANA BEACH, Calif., Oct. 28, 2024 (GLOBE NEWSWIRE) -- Evoke Pharma, Inc. (NASDAQ: EVOK), a specialty pharmaceutical company focused on developing treatments for gastrointestinal (GI) diseases, with a particular emphasis on GIMOTI® (metoclopramide) nasal spray, together with EVERSANA, a leading provider of global commercial services to the life sciences industry, today announced the presentation of data for GLP-1 users with diabetic gastroparesis using GIMOTI at the American College of Gastroenterology (ACG) 2024 Annual Meeting. The real-world retrospective study evaluated the impact of GIMOTI (metoclopramide nasal spray) in patients with diabetic gastroparesis (DGP) who were concurrently using GLP-1 receptor agonists. GLP-1 drugs are commonly prescribed for type 2 diabetes, and with the approval of GLP-1 medications, there have been increasing reports of these drugs exacerbating GI symptoms, specifically gastroparesis. GLP-1 medications function, in part, by causing delayed gastric emptying. Delayed gastric emptying, in the absence of mechanical obstruction, is the definition of gastroparesis. In a recent National Institute of Health (NIH) study (https://pubmed.ncbi.nlm.nih.gov/38399414/), an estimated 5.1% of patients in their study (n=10,328) on GLP-1 medications had gastroparesis. The GIMOTI study compared healthcare resource utilization (HRU) between patients using GIMOTI nasal metoclopramide (NMCP) and those on oral metoclopramide (OMCP), specifically focusing on individuals with a prior GLP-1 prescription. Significant reductions across a collection of outcomes were observed in patients treated with GIMOTI versus oral metoclopramide. Key Study Findings Presented at ACG 2024: Patients with prior GLP-1 history had reduced HRU after taking NMCP In NMCP patients, all-cause emergency department (ED) visits decreased by 55% (mean [SD]: 0.25 [1.13] post-index vs. 0.55 [1.30] pre-index; p=0.063); andDGP-related ED visits decreased by 28% (mean [SD]: 0.18 [0.99] post-index vs. 0.25 [1.28] pre-index; p=0.203). In patients taking GLP-1, those that took NMCP had fewer healthcare visits compared to those taking OMCP All-cause and DGP-related ED visits were 91% lower (cIRR: 0.09, 95% CI: 0.01, 0.42; p=0.001) and 89% lower (cIRR: 0.11, 95% CI: 0, 0.93; p=0.046) for NMCP vs. OMCP; andAll-cause Hospital Outpatient visits were 89% lower (cIRR = 0.11, 95% CI: 0.01, 0.73; p = 0.032)All-cause and DGP-related office visits were 41% lower (cIRR: 0.59, 95% CI: 0.37, 0.94; p=0.027) and 66% lower (cIRR: 0.34, 95% CI: 0.017, 0.65; p=0.001) for NMCP vs. OMCP. NMCP can be used to effectively treat patients with gastroparesis taking GLP-1 treatment and avoid costly healthcare visits All-cause clinic, outpatient, and inpatient visits showed similar trends favoring NMCP vs. OMCP.In DGP patients with a prior claim for GLP-1, NMCP use was associated with numerically and significantly reduced all-cause and DGP-related HRU compared to pre-treatment utilization and OMCP-treated controls. "This data is particularly encouraging given the rising number of patients using GLP-1 agonists for diabetes, many of whom also suffer from gastroparesis," said Matt D’Onofrio, CEO of Evoke Pharma. "Our goal is to provide data regarding Gimoti as supportive care for diabetic gastroparesis for those also on GLP-1 agonists. This study, combined with the earlier real-world data presented at key conferences, reinforces GIMOTI's potential to improve outcomes for patients and reduce the overall financial burden on the healthcare system." The study's specific cohort consisted of 92 total patients between the nasal metoclopramide (NMCP, N=51) and oral metoclopramide (OMCP, N=41) groups. The NMCP group had a slightly older average age (55.1 years) compared to the OMCP group (53.1 years). A notable portion of the NMCP group (31.4%) had experienced hospitalizations or emergency department visits before treatment, compared to 19.5% in the OMCP group. In a separate study, a post-hoc analysis focused on safety data for a female subgroup from a previous Phase 3 study that included 36 women using GLP-1 drugs during the 28-day study. Among these, 13 were treated with GIMOTI and 23 with placebo. The majority (94%) of the participants completed the study, with no serious adverse events reported in either group. This analysis indicated no series safety issues and a trend toward nausea improvement when GLP-1 drugs were used with NMCP. "Our continued focus is helping patients that have gastroparesis and diabetes. The fact that up to 20 million patients (KFF Health Tracking Poll (April 23-May 1, 2024) (kff.org/KFF Health Tracking Poll)) may have taken or are currently taking GLP-1s for their diabetes, indicates this is an important patient constituency the medical community needs to monitor and address," said Chris Quesenberry, Chief Commercial Officer of Gimoti. "Evoke Pharma, through its clinical development program and real-world evidence studies, is uniquely positioned to provide clinicians with information that may inform treatment decisions. This study provides insight that clinicians who treated their patients with diabetic gastroparesis, who were also taking a GLP-1, benefitted in taking GIMOTI®, an innovative, non-oral therapy that does not rely on a dysfunctional gut to work." The results presented at ACG 2024 build on a growing body of real-world data that underscores GIMOTI's ability to significantly reduce HRU in patients with diabetic gastroparesis. Earlier studies presented at Digestive Disease Week (DDW) 2024 and ACG 2023 demonstrated that patients treated with GIMOTI showed fewer hospitalizations, emergency department visits, and physician office visits compared to those using oral metoclopramide. Also, these studies highlighted that GIMOTI reduced healthcare costs by over $15,000 per patient in a six-month period compared to oral metoclopramide​​. "These studies further confirm the important role of GIMOTI in managing diabetic gastroparesis and supports its use for patients concurrently on GLP-1 agonists," said Dr. Michael Cline, Medical Director of the Gastroparesis Clinic at Cleveland Clinic, an author of the study. "In my practice, I’ve treated patients arriving with gastroparesis while on a GLP-1. This data demonstrates a significant reduction in emergency room visits, office visits, and overall healthcare resource utilization, which not only improves patient outcomes but also reduces the strain on healthcare systems. The nasal delivery of metoclopramide offers a distinct advantage for patients with delayed gastric emptying, ensuring they receive the relief they need without the complications or unknown absorption associated with oral medications." EVERSANA was named Evoke’s commercialization partner to support commercialization efforts of GIMOTI, first in 2020 and then an extended partnership was announced in February 2022. Slides referencing data to the studies can be found under news and presentations of the Evoke Pharma investor relations website. 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 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 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 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: GIMOTI’s potential to reduce HRU by diabetic gastroparesis patents taking GLP-1 therapies; the possibility that increased use of GLP-1 agonists could increase the need for treatment of gastroparesis or increase the patient base for GIMOTI; and Evoke’s belief that GIMOTI can improve treatment of gastroparesis in patients taking GLP-1 agonists. 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 and EVERSANA may not be able to successfully drive market demand for GIMOTI; the results of market research studies may not predict acceptance by patients, healthcare providers or payors; GLP-1 agonists may not increase the number of patients diagnosed with gastroparesis, which remains speculative; alternative treatments for gastroparesis may be developed or approved and may be shown to be superior to GIMOTI; Evoke’s ability to obtain additional financing as needed to support its operations; Evoke may use its capital resources sooner than expected; Evoke’s ability to maintain compliance with Nasdaq’s stockholder’s equity requirements; 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. About EVERSANA EVERSANA® is a leading independent provider of global services to the life sciences industry. The company’s integrated solutions are rooted in the patient experience and span all stages of the product life cycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 650 organizations, including innovative start-ups and established pharmaceutical companies, to advance life sciences solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and X. Investor & Media Contact: Daniel Kontoh-Boateng DKB Partners Tel: 862-213-1398 dboateng@dkbpartners.net A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/cfc46127-a976-498b-8e19-d5c879171b81

Additional real-world studies show sustained HAE attack rate reduction after initiating ORLADEYO, regardless of C1-INH status, and patient preference for an oral LTP therapyRESEARCH TRIANGLE PARK, N.C., Oct. 24, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world comparative research on the use of oral, once-daily ORLADEYO® (berotralstat) that found high rates of adherence and persistence for ORLADEYO, similar to the rates observed with two other long-term prophylactic (LTP) therapies for hereditary angioedema (HAE). The company also announced new real-world evidence showing statistically significant and sustained HAE attack rate reductions after initiating ORLADEYO in patients with HAE, regardless of their C1-inhibitor (C1-INH) deficiency status, and new findings from an HAE patient survey confirming patient preference for an oral LTP therapy. “These results build on previous research that highlights how the real-world effectiveness and convenience of our oral, once-daily prophylactic therapy are differentiators for patients. The strong adherence, persistence and preference for ORLADEYO we report here provide further evidence that enables physicians to optimize individualized treatment recommendations for each of their patients. We continue to generate real-world evidence demonstrating that ORLADEYO is a powerful, potentially life-changing treatment for many people with HAE,” said Dr. Donald S. Fong, chief medical officer of BioCryst. The data are being presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is taking place in Boston from October 24-28, 2024. High Adherence and Persistence Rates for ORLADEYO and Other LTP Therapies Poster #R072 explores adherence and persistence rates among patients with HAE following initiation of ORLADEYO (n=90) compared with lanadelumab (n=189) and subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH) (n=78). “Our findings reveal that among HAE patients managed with LTP, high adherence and persistence rates were demonstrated for ORLADEYO. There was a striking consistency between first line therapies with comparable results for lanadelumab and SC-pdC1-INH. ORLADEYO rates were equivalent to – and in some cases better than – these LTP counterparts. The outcomes of our study provide further support for a shared decision-making approach within the physician-patient partnership to inform LTP selection, incorporating effectiveness, safety, treatment burden and patient preference for each individual,” said Sandra Christiansen, MD, professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego. Adherence And Persistence Among Hereditary Angioedema Patients Treated With Berotralstat, Lanadelumab, And Subcutaneous Plasma-Derived C1-Inhibitor; ePoster #R072; Friday, October 25, 2:30-2:45 p.m. ET; Monitor #19, Hall A Eighty-six percent of patients who took ORLADEYO (n=77), 91 percent who took lanadelumab (n=172) and 83 percent who took SC-pdC1-INH (n=65) filled at least two prescriptions during the 12-month follow-up period.Adherence rates among patients with at least two fills were high at 12 months of follow-up. Seventy-seven percent of patients taking ORLADEYO were adherent compared to 76 percent of patients taking lanadelumab and 80 percent of patients taking SC-pdC1-INH.Persistence rates among patients with at least two fills were high at 12 months of follow-up, with 71 percent of patients taking ORLADEYO persistent compared to 63 percent of patients taking lanadelumab and 63 percent of patients taking SC-pdC1-INH.No statistically significant differences in adherence or persistence were observed between the LTP patient cohorts. Methods Electronic health records linked to claims data were used to select mutually exclusive cohorts of patients aged 12 and above who initiated ORLADEYO, lanadelumab or pdC1-INH between June 22, 2017, and September 12, 2023.Adherence was defined as having a proportion of days covered greater than or equal to 0.80.Persistence was defined as fewer than 45 consecutive days without the index LTP treatment. Sustained HAE Attack Rate Reduction After Beginning ORLADEYO Regardless of C1-INH Status Posters #R092 and #R093 evaluate self-reported HAE attacks prior to and following ORLADEYO initiation among patients with and without HAE with C1-inhibitor deficiency (HAE-nl-C1-INH), respectively. “ORLADEYO was associated with statistically significant and sustained reductions in attack rates through 18 months (540 days) following ORLADEYO initiation, regardless of patients’ C1-INH status. Importantly, in this real-world study, patients with zero attacks at baseline were included, compared to the minimum of two attacks that was required at baseline in APeX-2. These data confirm that many already well-controlled HAE patients enjoy continued or improved attack control once they initiate ORLADEYO,” Dr. Fong added. Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency; ePoster #R092; Friday, October 25, 3:30-3:45 p.m. ET; Monitor #20, Hall A Patients (n=466) had significantly lower attack rates at each follow-up interval, which ranged from 0.62 attacks per month at days 361-450 (n=185) to 0.79 at days 271-360 (n=217) versus baseline (2.62 at days 361-450 to 2.50 at days 271-360).Mean monthly attack rate reduction was -1.71 (p<0.05) at days 271-360 (n=217) and -1.96 (p<0.05) at days 451-540 (n=157). Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema without C1-Inhibitor Deficiency; ePoster #R093; Friday, October 25, 3:45-4:00 p.m. ET; Monitor #20, Hall A Patients (n=353) had significantly lower attack rates at each follow-up interval, which ranged from 1.63 attacks per month at days 181-270 (n=191) to 2.04 at days 1-90 (n=304) versus baseline (4.33 at days 181-270 to 4.63 at days 1-90).Mean monthly attack rate reduction was -2.91 (p<0.05) at days 271-360 (n=157) and -2.53 (p<0.05) at days 451-540 (n=108). Methods Outcomes were collected through BioCryst’s sole-source pharmacy and included U.S. patients who actively received ORLADEYO 110 or 150 mg QD from December 15, 2020, to January 8, 2024.Patient-reported HAE attack rates were collected at baseline and each refill (approximately every 30 days).To be included in the analysis of a given follow-up period, patients were required to have responded to a follow-up assessment within the period of interest and to the onboarding self-assessment of attacks. Approximately 96 percent of patients met these criteria at each interval. Patients with HAE Prefer Oral LTP Poster #R081 examines the LTP treatment preferences of patients with HAE, as collected through an online survey. “We are encouraged to see both effectiveness and convenience are drivers of treatment preference with patients preferring oral therapy over injectable therapy when efficacy is assumed equal. Given the benefits of treatment with ORLADEYO for patients who remain on therapy over the long term, we believe we will continue to see the preference towards oral therapy reflected in the strong commercial performance of our oral, once-daily prophylactic therapy for HAE,” Dr. Fong noted. Long-Term Prophylactic Treatment Preferences of Patients With Hereditary Angioedema; ePoster #R081; Friday, October 25, 4:45-5:00 p.m. ET; Monitor #19, Hall A LTP treatment attributes rated most important were effectiveness in attack prevention and effectiveness in reducing attack severity.When presented with the option of an equally effective oral or injectable LTP, 54 percent preferred an oral daily therapy over a biweekly or monthly subcutaneous injection.More than half (52 percent) preferred oral over injectable LTP as infrequently as every three months. Methods Results from an online survey of U.S. patients with HAE who were at least 18 years of age, self-reported their diagnosis and were receiving an HAE treatment (LTP, on-demand or both) or had experienced at least one HAE attack in the preceding three months (n=150). In addition to displaying in the exhibit hall at the noted times, ePosters are accessible online and on demand to registered attendees on ACAAI’s website. 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 and statements relating to ORLADEYO performance and patient preferences. 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: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve sustained 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; and risks related to the international expansion of BioCryst’s business. 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

RESEARCH TRIANGLE PARK, N.C., Oct. 14, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world evidence on the use of oral, once-daily ORLADEYO® (berotralstat) demonstrating that patients with hereditary angioedema (HAE) in the United States experience significant reductions in healthcare resource utilization (HRU), including significant reductions in hospitalizations, emergency room visits and use of on-demand therapies, after beginning treatment with ORLADEYO. The study will be presented in a poster at the Academy of Managed Care Pharmacy (AMCP) Nexus 2024 national meeting, which is being held in Las Vegas from October 14-17, 2024. “Our interrogation of claims data builds upon previously reported findings demonstrating that ORLADEYO not only has a favorable impact on the lives of patients with HAE – but the broader healthcare system, as well. Here, our analysis reveals that significant reductions in healthcare resource utilization are achieved across multiple outcomes – such as reductions in hospitalizations and medical visits, including those related to HAE attacks. Decreases in on-demand treatment were also observed. Taken together, our investigation provides promising real-world evidence which supports ORLADEYO’s clinical and financial value as a prophylactic therapy for HAE,” said Sandra Christiansen, MD, professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego. The poster Healthcare Resource Utilization among Patients Initiating Berotralstat for the Long Term Prophylaxis of Hereditary Angioedema in the United States (#D20) detailed findings from a retrospective pre-post study that featured analysis of administrative U.S. claims data of patients with HAE in the United States. The analysis focused on eligible patients enrolled in commercial and public health plans who initiated ORLADEYO between December 2020 and December 2022 who had a baseline of at least six months of continuous health plan enrollment prior to starting ORLADEYO (n=260). Significant reductions in HRU were observed in the overall study population following initiation of ORLADEYO (p<0.05), including in: All-cause hospitalizations (34 percent reduction) and outpatient or emergency room visits (14 percent reduction).Angioedema-related hospitalizations (52 percent reduction) and outpatient or emergency room visits (44 percent reduction). Significant reductions in HAE attack-related HRU were also observed (p<0.05), including in: HAE attack-related visits (51 percent reduction), driven by significant decreases in hospitalization (60 percent) and outpatient or emergency room visits (50 percent).Reduction in HAE attack-related visits were observed when stratified by body location of the attack, including in the head and upper airways (48 percent reduction), gastrointestinal system (58 percent reduction) and unspecified locations (52 percent reduction).Additionally, a decrease in use of on-demand therapies administered by a healthcare professional was observed (39 percent reduction) among patients who previously received on-demand treatment. The poster will be on display in the expo hall of the MGM Grand Hotel and Convention Center on Tuesday, October 15, from 5:00-7:00 pm PT and will be presented on Wednesday, October 16, from 11:30 am-1:00 pm PT. 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 INFORMATIONAn 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 and statements relating to ORLADEYO performance, including with respect to ORLADEYO’s impact on healthcare resource utilization. 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: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve sustained 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; and risks related to the international expansion of BioCryst’s business. 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