CELEBRATION, Fla., Oct. 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that members of Zevra’s executive leadership team will participate in a fireside discussion at Guggenheim's 2 nd Annual Healthcare Innovation Conference in Boston, MA on Wednesday, November 12,... Read More

Company will host conference call at 4:30 p.m. ET on Wednesday, November 5, 2025 CELEBRATION, Fla., Oct. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced it will report corporate and financial results for the third quarter 2025 on Wednesday, November 5, 2025, via a news... Read More

CELEBRATION, Fla., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that the Company has granted options to purchase an aggregate of 38,000 shares of the Company’s common stock (the “Inducement Awards”) to two new employees pursuant to the Company’s 2023 Employment... Read More

MIPLYFFA, the only therapy for Niemann-Pick disease type C (NPC) shown to stop disease progression at 12 months, has been administered to more than 270 patients, representing the largest data set of NPC clinical trial patients OLPRUVA offers the established efficacy and safety of sodium phenylbutyrate in a novel and convenient formulation for the treatment of certain urea cycle disorders CELEBRATION, Fla., Sept. 18, 2025... Read More

New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targets underlying pathology of NPC CELEBRATION, Fla., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the... Read More

CELEBRATION, Fla., Aug. 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced four posters on MIPLYFFA ® (MY-PLY-FAH) (arimoclomol) will be presented at the International Congress of Inborn Errors of Metabolism (ICIEM) , taking place September 2-6, 2025, in Kyoto, Japan.... Read More

CELEBRATION, Fla., Aug. 26, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that members of Zevra’s executive leadership team will participate in the following September events: Cantor Global Healthcare Conference on Wednesday, Sept. 3, 2025, at 11:30 a.m. ET. H.C.... Read More

Q2 2025 net revenue of $25.9 million, driven by product net revenue of $21.8 million Completed sale of PRV for $150.0 million, bolstering the balance sheet as the Company executes on its commercial launches and development programs Submitted a Marketing Authorisation Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C Company to host conference call... Read More

CELEBRATION, Fla., Aug. 06, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that members of Zevra’s executive leadership team will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference in Boston, MA, on Wednesday, August 13, 2025, at 1:00... Read More

Company will host conference call at 4:30 p.m. ET, on Tuesday, August 12, 2025 CELEBRATION, Fla., Aug. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced it will report corporate and financial results for the second quarter 2025 on Tuesday, August 12, 2025, via a news... Read More

CELEBRATION, Fla., July 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare diseases, announced the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC). NPC is... Read More

CELEBRATION, Fla., July 18, 2025 (GLOBE NEWSWIRE) - - Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced that three posters on MIPLYFFA ® (MY-PLY-FAH) (arimoclomol) and one on OLPRUVA ® (sodium phenylbutyrate) are being presented at the 42 nd Annual Meeting of the Southeastern Regional Genetics Group... Read More

CELEBRATION, Fla., July 16, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced the publication of “ Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial ” in the peer-reviewed... Read More

CELEBRATION, Fla., July 11, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced an oral presentation and two poster presentations on MIPLYFFA ® (MY-PLY-FAH) (arimoclomol) are being featured at the National Niemann Pick Disease Foundation (NNPDF) Conference, taking place July... Read More

Zevra Stockholders Re-Elect Wendy L. Dixon, Ph.D. and Tamara A. Favorito to the Company’s Board of Directors Board and Management Reaffirm Commitment to Driving Continued Growth and Progress Against Strategic Plan CELEBRATION, Fla., May 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra,” or the “Company”), a commercial-stage company focused on providing therapies to people living with rare... Read More

ISS, Glass Lewis, and Egan-Jones Unanimously Conclude Mangless Has Not Made a Compelling Case for Change Board and Management Urge Stockholders to Follow Proxy Advisors’ Recommendation and Vote “FOR” Wendy L. Dixon, Ph.D. and Tamara A. Favorito on the WHITE Proxy Card and “WITHHOLD” for Mangless Nominees CELEBRATION, Fla., May 21, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra,” or the “Company”),... Read More

Q1 2025 net revenue of $20.4 million, driven by product net revenue of $17.2 million Completed sale of PRV for gross proceeds of $150 million, positioning balance sheet to drive the Company’s commercial launches and development programs Company to host conference call and webcast today, May 13, 2025, at 4:30 p.m. ET CELEBRATION, Fla., May 13, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the... Read More

CELEBRATION, Fla., May 06, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced it will report corporate and financial results for the first quarter 2025 on Tuesday, May 13, 2025. The Company will issue a news release after the market closes and host a conference call/audio... Read More

CELEBRATION, Fla., May 01, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that members of Zevra’s executive leadership team will participate in a fireside chat at the Citizens Life Science Conference in New York, NY, on Wednesday, May 7, 2025, at 11:30 a.m. ET. Additionally, management will be available for... Read More

Highlights 54.8% Total Stockholder Return Under Refreshed Board and Management Team Zevra Continues Growth Trajectory into a Global Commercial Rare Disease Company with Consistent Execution, New Product Launches, and Significant Financial Flexibility to Execute Strategic Plan Urges Stockholders to Defend Board Independence... Read More

CELEBRATION, Fla., April 17, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announces the publication of “Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease” in Molecular Genetics and Metabolism. “This elucidation of... Read More

CELEBRATION, Fla., April 07, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. “We have reached another key milestone with the closing on the sale of our... Read More

Confirms Receipt of Two Director Nominees from Daniel J. Mangless Stockholders Are Not Required to Take Action at This Time CELEBRATION, Fla., March 31, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (Nasdaq:GS ZVRA) (“Zevra,” or the “Company”), a commercial-stage company focused on providing therapies to people living with rare disease, today announced it has filed its preliminary proxy statement with the U.S. Securities... Read More

FY 2024 net revenue of $23.6 million, driven by net revenue of $12.0 million during fourth quarter of 2024 Entered into asset purchase agreement for sale of PRV for $150 million Company to host conference call and webcast today, Mar. 11, 2025, at 4:30 p.m. ET CELEBRATION, Fla., March 11, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing... Read More

New website launched in honor of Rare Disease Day, Feb. 28, 2025 Awareness campaign includes educational resources and testing information for individuals with suspected NPC. CELEBRATION, Fla., Feb. 28, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company... Read More

CELEBRATION, Fla., Feb. 27, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million upon the closing of the... Read More

CELEBRATION, Fla., Feb. 26, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced it will report corporate and financial results for the fourth quarter and full year 2024 on Tuesday, Mar. 11, 2025. The Company will issue a news release after the market closes and host a... Read More

CELEBRATION, Fla., Feb. 24, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced that members of Zevra’s executive leadership team will participate at the following March events: TD Cowen 45 th Annual Health Care Conference on Wednesday, Mar. 5, 2025, at 10:30 a.m. ET. 37 th... Read More

Zevra to receive a 2025 New Treatment Award for MIPLYFFA™ at the 21 st Annual WORLDSymposium Eight abstracts discussing data and clinical experience associated with MIPLYFFA accepted for poster presentation, with one poster selected for oral presentation by Dr. Eugen Mengel CELEBRATION, Fla., Jan. 30, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease... Read More

CELEBRATION, Fla., Jan. 29, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that members of Zevra’s executive leadership team will participate in the following February events. Guggenheim Securities SMID Cap Biotechnology Conference on Wednesday, Feb. 5, 2025, at 2:30 p.m. ET. Oppenheimer & Co. 35 th Annual... Read More

CELEBRATION, Fla., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced its upcoming addition to the Nasdaq Biotechnology Index (Nasdaq: NBI) (“NBI”) as part of the annual reconstitution of the 2024 Nasdaq indexes. Zevra’s inclusion in the NBI will be effective after... Read More

CELEBRATION, Fla., Dec. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced organizational changes designed to accelerate its transformation into a leading rare disease therapeutics company. The company announced that it is consolidating its development and scientific... Read More

CELEBRATION, Fla., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced that Neil F. McFarlane, President and Chief Executive Office of Zevra, will participate in the following December investor events. Piper Sandler 36 th Annual Healthcare Conference, on Wednesday,... Read More

MIPLYFFA, the first FDA-approved treatment for Niemann-Pick disease type C, is available at Zevra’s specialty pharmacy for dispense AmplifyAssist™, Zevra’s comprehensive patient support program, in place to address access barriers CELEBRATION, Fla., Nov. 21, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that... Read More

FDA approval of MIPLYFFA™ and product launch cap third quarter filled with multiple commercial and clinical development milestones Zevra received rare pediatric disease Priority Review Voucher Company ends quarter with cash, cash equivalents and investments of $95.5 million Company to host conference call and webcast today, Nov. 12, 2024, 4:30 p.m. ET CELEBRATION, Fla., Nov. 12, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics,... Read More

Data show that the NPCCSS swallow score reflects the patient’s level of dysfunction Study indicates that a change in score reflects actual improvement or worsening in a patient’s swallowing function CELEBRATION, Fla., Nov. 11, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc . (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today... Read More

CELEBRATION, Fla., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced that Neil F. McFarlane, President and Chief Executive Office of Zevra, will participate in a fireside chat at the Guggenheim Securities Healthcare Innovation Conference Virtual on Wednesday, Nov.... Read More

CELEBRATION, Fla., Oct. 31, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc . (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases, today announced it will report third quarter financial results and provide a corporate update on Tuesday, Nov. 12, 2024. The Company will issue a news release after the market closes and host a conference... Read More

CELEBRATION, Fla., Oct. 10, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Office of Zevra, will participate in a Rare Disease Panel at the 2024 Maxim Healthcare Virtual Summit, presented by Maxim Group LLC on Thursday, October 17, 2024 at 2 p.m. ET. Maxim Group 2024 Annual... Read More

PASADENA, Calif. and CELEBRATION, Fla., Oct. 09, 2024 (GLOBE NEWSWIRE) -- The National Urea Cycle Disorders Foundation (NUCDF) and Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) are teaming up to launch an awareness campaign during the month of October to improve the diagnosis of urea cycle disorders (UCDs). Their goal is to highlight the critical importance of promptly checking blood ammonia levels and using proper testing... Read More

KP1077 demonstrates clinically meaningful benefits for key IH symptoms Top-line data provide key information for the design of a Phase 3 study CELEBRATION, Fla., Sept. 24, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a commercial-stage rare disease therapeutics company, today announced that Rene Braeckman, Ph.D., Zevra’s Senior Vice President of Clinical Development, will present... Read More

MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older Zevra receives rare pediatric disease priority review voucher in conjunction with approval Company launches... Read More

New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting... Read More

CELEBRATION, Fla., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Office of Zevra, will present at upcoming investor conferences during the month of September 2024. Additionally, management will be available for one-on-one meetings with registered attendees at each... Read More

FDA advisory committee voted favorably that the data support arimoclomol as effective treatment for patients with NPC; PDUFA date of September 21, 2024 Pro forma June 30, 2024, cash, cash equivalents, investments and net proceeds from underwritten public offering total $113.8 million following the closing on August 12, 2024 Conference call scheduled for today, August 13, 2024, at 4:30 p.m. ET CELEBRATION, Fla., Aug. 13, 2024... Read More

CELEBRATION, Fla., Aug. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (Nasdaq: ZVRA), a rare disease therapeutics company (“Zevra”), today announced the pricing of an underwritten public offering of 9,230,770 shares of its common stock at an offering price of $6.50 per share, for total gross proceeds of approximately $60.0 million, before deducting underwriting discounts and commissions and offering expenses payable... Read More

CELEBRATION, Fla., Aug. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (Nasdaq: ZVRA), a rare disease therapeutics company (“Zevra”), today announced the commencement of a proposed underwritten public offering of its common stock. All shares of common stock to be sold in the proposed offering will be sold by Zevra. Zevra also expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the... Read More

CELEBRATION, Fla., Aug. 07, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Officer of Zevra, will present at Canaccord Genuity’s 44 th Annual Growth Conference in Boston, MA, on Wednesday, August 14, 2024, at 10:30 a.m. ET. Members of the executive team will be available for... Read More

CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC).... Read More

CELEBRATION, Fla., July 30, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Tuesday, August 13, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the second quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations... Read More

CELEBRATION, Fla., July 09, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to review the New Drug Application (NDA) for arimoclomol as an... Read More

Rahsaan W. Thompson appointed as Chief Legal Officer, Secretary and Compliance Officer Alison Peters appointed as Chief People Officer CELEBRATION, Fla., June 25, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the appointments of Rahsaan W. Thompson as Chief Legal Officer, Secretary and Compliance Officer, and Alison Peters as... Read More

CELEBRATION, Fla. and ELK GROVE VILLAGE, Ill. , June 18, 2024 /PRNewswire/ -- Orsini Specialty Pharmacy (Orsini), and Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra) today announced that Orsini is now the pharmacy partner for OLPRUVA ® (sodium phenylbutyrate) for oral suspension. OLPRUVA ® is a prescription medicine used along with certain therapies, including changes in diet, for long-term management of certain adult and... Read More

KP1077 was well tolerated and showed meaningful clinical improvements in patient-reported assessments of daytime sleepiness, sleep inertia, and brain fog Additional data presented on pharmacokinetics of morning and nighttime doses of KP1077 European Commission has granted Orphan Drug Designation for KP1077 for the treatment of idiopathic hypersomnia CELEBRATION, Fla., June 03, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics,... Read More

CELEBRATION, Fla., May 09, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Officer of Zevra, will present at several upcoming investor events in May 2024. Additionally, management will be available for one-on-one meetings with registered attendees. Mr. McFarlane will present at... Read More

Full commercial launch of OLPRUVA at the end of January 2024, with market access growing to ~75% of covered lives as of May 1, 2024 Arimoclomol NDA review underway with PDUFA date of September 21, 2024 Reported positive topline results from KP1077 Phase 2 Trial in idiopathic hypersomnia, with full data to be presented at SLEEP 2024 in early June 2024 Conference call scheduled for today, May 8, 2024, at 8:00 a.m. ET... Read More

CELEBRATION, Fla., May 01, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Wednesday, May 8, 2024, at 8:00 a.m. ET, to review its corporate and financial results for the first quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations... Read More

CELEBRATION, Fla., April 29, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, Zevra’s President and Chief Executive Officer, has issued a Letter to Stockholders providing a review of key accomplishments and outlook for 2024. Dear Stockholders, It is hard for me to believe that April 2024 marks six months... Read More

The oral presentation highlighted data from Zevra’s Expanded Access Program demonstrating a consistent safety profile CELEBRATION, Fla., April 15, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that new long-term real-world data from the arimoclomol expanded access program (EAP: NCT04316637 ) for the treatment of Niemann-Pick... Read More

CELEBRATION, Fla., April 10, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that it has entered into a new credit facility provided by leading biotech investors. Led by Perceptive Advisors and Healthcare Royalty, the new credit facility provides up to $100M in committed capital in three tranches: an initial draw of $60M at... Read More

Launch of OLPRUVA ® underway Topline results from KP1077 phase 2 study demonstrate clinically meaningful benefits for key IH symptoms Q4 2023 net revenue of $13.2M, and FY 2023 net revenue of $27.5M Conference call scheduled for today, March 28, 2024, at 4:30 p.m. ET CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company,... Read More

KP1077 demonstrates clinically meaningful benefits for key IH symptoms Top-line data provide key information for the design of a Phase 3 study CELEBRATION, Fla., March 26, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced top-line data from its placebo-controlled, double-blind Phase 2 clinical trial ( NCT05668754 ) evaluating the... Read More

CELEBRATION, Fla., March 25, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that it will participate in the upcoming 23 rd Annual Needham Virtual Healthcare Conference being held virtually on April 8-9, 2024. The Company’s presentation will take place on Monday, April 8, 2024, at 3:45 p.m. ET. Additionally, members of Zevra’s... Read More

CELEBRATION, Fla., March 18, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and live audio webcast on Thursday, March 28, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the fourth quarter and full year 2023. The link to the audio webcast will be accessible via the... Read More

The new Prescription Drug User Fee Act (PDUFA) action date set by the FDA is September 21, 2024 CELEBRATION, Fla., March 04, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for arimoclomol, an investigational orally... Read More

CELEBRATION, Fla., Feb. 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Officer of Zevra, will present at upcoming investor conferences in March 2024. Additionally, management will be available for one-on-one meetings with registered attendees at each of the conferences.... Read More

CELEBRATION, Fla., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that it will present at the upcoming Oppenheimer 34 th Annual Healthcare Life Sciences Conference being held virtually on Tuesday, February 13, 2024, at 9:20 a.m. ET. Additionally, members of Zevra’s management team will be available for one-on-one... Read More

CELEBRATION, Fla., Jan. 23, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the appointment of Alvin Shih, MD, MBA, to the Company’s Board of Directors (Board) effective as of January 20, 2024. Dr. Shih has extensive experience in biotechnology leadership and rare disease therapeutics development and currently serves as President... Read More

Arimoclomol NDA has been assigned a PDUFA action date of June 21, 2024 CELEBRATION, Fla., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the resubmission of the New Drug Application (NDA) for arimoclomol as an orally-delivered, first-in-class... Read More

CELEBRATION, Fla., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the appointment of Adrian Quartel, M.D., FFPM, as Chief Medical Officer. Dr. Quartel brings more than 20 years of experience in the international pharmaceutical industry with senior roles in clinical development, pharmacovigilance and medical affairs. "It... Read More

CELEBRATION, Fla., Dec. 27, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) a rare disease therapeutics company, today announced it resubmitted its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based on standard NDA resubmission review timelines,... Read More

Acquisition includes OLPRUVA ® , an FDA-approved treatment for urea cycle disorders (UCDs), which propels Zevra into commercial stage, diversifying its revenue and providing scale Settlement of Acer’s warrant obligations through direct transaction with a healthcare focused investment fund adds quality healthcare investor to Zevra’s shareholder base CELEBRATION, Fla., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc.... Read More

Arimoclomol NDA resubmission remains on track for end of year filing Proposed acquisition of Acer Therapeutics on track to close Q4 2023 and will diversify Zevra’s revenue with FDA-approved OLPRUVA ® for urea cycle disorders (UCDs) Net revenue of $2.9M for Q3 2023 Ended Q3 2023 with $83.4M in cash, cash equivalents, and investments, supporting our forecasted cash runway into 2026 Conference call and live audio webcast... Read More

CELEBRATION, Fla., Oct. 31, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and live audio webcast on Tuesday, November 7, 2023, at 8:00 a.m. ET, to review its corporate and financial results for the third quarter of 2023. The link to audio webcast will be accessible via the Investor Relations... Read More

CELEBRATION, Fla., Oct. 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a rare disease therapeutics company, proudly supports the Niemann-Pick community on Global Niemann-Pick Disease Awareness Day and throughout Niemann-Pick Disease Awareness Month. Patient organizations like the International Niemann-Pick Disease Alliance (INPDA) and the National Niemann-Pick Disease Foundation (NNPDF) provide... Read More

Christal Mickle transitions back to Chief Development Officer CELEBRATION, Fla., Oct. 10, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane has been named President and Chief Executive Officer of Zevra and appointed to the Board of Directors, effective October 10, 2023. Neil succeeds Christal M.M. Mickle who... Read More

Presentation to focus on the arimoclomol program for the treatment of Niemann-Pick Disease type C and related early access programs CELEBRATION, Fla., Oct. 05, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that it will present data related to clinical study outcomes and its early access programs via both oral and poster... Read More

Data generated to date indicate SDX is well-tolerated across all doses and both dosing regimens studied, laying the groundwork for future Phase 3 trial Topline data from completed trial expected in the first half 2024, with potential to initiate Phase 3 trial by the end of 2024 CELEBRATION, Fla., Oct. 02, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics... Read More

CELEBRATION, Fla., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that executive management will participate in a fireside chat at the Cantor Global Healthcare Conference on Wednesday, September 27, 2023, at 3:00 p.m. ET. Members of Zevra’s management team will be available for one-on-one investor meetings with... Read More

CELEBRATION, Fla., Sept. 09, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced a poster presentation featuring study data that underscores the cardiovascular safety profile of serdexmethylphenidate (SDX), the sole active pharmaceutical ingredient (API) in KP1077, Zevra’s investigational candidate for the treatment for idiopathic... Read More

CELEBRATION, Fla., Sept. 05, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that executive management will present at the H.C. Wainwright 25 th Annual Global Investment Conference on Wednesday, September 13, 2023, at 8:30 a.m. ET. Members of Zevra’s management team will be available for one-on-one investor meetings with... Read More

Proposed acquisition of Acer for $15M in Zevra stock plus Contingent Value Rights (CVRs) and Zevra’s purchase of Acer’s secured debt in capital efficient structure Zevra to assume commercialization efforts of OLPRUVA™, recently approved for the treatment of urea cycle disorders (UCDs) FDA-approved commercial asset expected to increase and diversify Zevra’s revenues OLPRUVA™ commercial operations provide Zevra scale and cost... Read More

Completed collaborative and productive pre-submission meeting with FDA for arimoclomol NDA in August 2023; filing expected in Q4 2023 Net revenue of $8.5M for Q2 2023, which includes $5 million milestone payment earned under the AZSTARYS® license agreement Ended Q2 2023 with $87.4 million in cash, cash equivalents, and investments, supporting our forecasted cash runway into 2026 Conference call and live audio webcast with... Read More

CELEBRATION, Fla., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics (NasdaqGS: ZVRA) ("Zevra" or “the Company"), a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options, proudly welcomes Tom Anderson, a highly accomplished biopharma veteran, to its Board of Directors (the "Board"). Concurrently, the Company announces the... Read More

CELEBRATION, Fla., Aug. 02, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, has announced that it will host a conference call and live audio webcast on Monday, August 14, 2023, at 8:30 a.m. ET, to review its corporate and financial results for the second quarter of 2023. The link to audio webcast with a slide... Read More

CELEBRATION, Fla., July 19, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, today announced their support of the upcoming National Niemann-Pick Disease Foundation’s (NNPDF) Family Support and Medical Conference. Members of the Zevra team will attend this event which is being held in Orlando, Florida, from July 20 through... Read More

CELEBRATION, Fla., June 20, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, announced its expected addition to the broad-market Russell 3000 ® Index and small-cap Russell 2000 ® Index in accordance with the 2023 Russell indexes annual reconstitution. Zevra anticipates that its inclusion in the Russell indexes will be... Read More

CELEBRATION, Fla., June 13, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, today announced executive management will participate in the Healthcare Virtual Conference Part II, presented by Maxim Group LLC and hosted by M-Vest, Tuesday, June 20 through Thursday, June 22, 2023. Zevra will participate in a fireside chat on... Read More

Zevra is a corporate sponsor of the Hypersomnia Foundation and Beyond Sleepy Conference CELEBRATION, Fla., June 03, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly Zevra.), a rare disease therapeutics company, today announced that an oral presentation featuring the Phase 2 clinical trial design evaluating KP1077 as a treatment for idiopathic hypersomnia (IH), a rare... Read More

Christal Mickle, Co-Founder and Chief Development Officer, appointed to serve as interim President and CEO effective June 1, 2023 Conference call and live audio webcast with slide presentation scheduled for today, May 15, 2023, 4:30 p.m. ET CELEBRATION, Fla., May 15, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, today... Read More

CELEBRATION, Fla., May 10, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that R. LaDuane Clifton, the Company’s Chief Financial Officer, will present at the RBC Capital Markets Global Healthcare Conference on Tuesday, May 16, 2023, at 3:05 p.m. ET. In addition, members of Zevra’s management team... Read More

CELEBRATION, Fla., May 09, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company, formerly KemPharm, Inc.), a rare disease therapeutics company, has announced that it will host a conference call and live audio webcast on Monday, May 15, 2023, at 4:30 p.m. ET, to review its corporate and financial results for the first quarter of 2023. The link to audio webcast with a slide presentation... Read More

CELEBRATION, Fla., May 08, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced changes to the Company’s Board of Directors (“Board”) and leadership. At the Company’s recent 2023 Annual Meeting of Stockholders (“Annual Meeting”), Zevra shareholders elected John B. Bode, Douglas W. Calder and Corey Watton... Read More

Zevra plans to expand KP1077 clinical program to address multiple rare sleep disorders with trials initiating in 2023 CELEBRATION, Fla., May 03, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced the acceptance of an Investigational New Drug (“IND”) application by the U.S. Food and Drug Administration... Read More

Glass Lewis Recommends a Vote “FOR” Zevra’s Three Nominees Both ISS and Glass Lewis Recommend Shareholders DO NOT Vote “FOR” Any of the Mangless Nominees Zevra Urges Shareholders to Vote on the WHITE Proxy Card CELEBRATION, Fla., April 17, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that leading... Read More

Zevra seeks to expand KP1077 clinical program to address multiple rare sleep disorders CELEBRATION, Fla., April 04, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced the submission of an Investigational New Drug (IND) application seeking authorization from the U.S. Food and Drug Administration (“FDA”)... Read More

CELEBRATION, Fla., March 30, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that it has appointed Wendy L. Dixon, Ph.D., to the Company’s Board of Directors (the “Board”), effective immediately following the conclusion of the 2023 Annual Meeting of Stockholders (“2023 Annual Meeting”), to fill the... Read More

New Patent Issued Covering Serdexmethylphenidate (SDX), the Sole Active Pharmaceutical Ingredient in KP1077, Zevra’s Product Candidate for the Treatment of Idiopathic Hypersomnia (IH) CELEBRATION, Fla., March 17, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that it has joined the Sleep Data Initiative as a founding member.... Read More

Urges Shareholders to Vote "FOR" All Zevra Director Nominees on the WHITE Proxy Card CELEBRATION, Fla., March 15, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced it has filed its definitive proxy statement with the U.S. Securities and Exchange Commission (the “SEC”) in connection with the Company’s 2023... Read More

Conference Call and Live Audio Webcast with Slide Presentation Scheduled for Today, March 7, 2023, 8:30 a.m. ET Newly rebranded Zevra Therapeutics well positioned to become a commercially focused rare disease company Arimoclomol NDA resubmission on track to be filed as early as Q3 2023 KP1077 Phase 2 Trial on track to deliver preliminary data as early as Q3 2023 and top-line data as early as year-end 2023 AZSTARYS ® royalty... Read More

CELEBRATION, Fla., March 06, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that Richard W. Pascoe, the Company’s Chief Executive Officer, will participate in a fireside chat at the 35 th Annual Roth Conference on Monday, March 13, 2023, at 2:00 p.m. PT. In addition, Zevra management will be... Read More

Company’s common stock will begin trading on the Nasdaq Global Select Market under the new ticker symbol “ZVRA” starting today, March 1, 2023 New Company name, Zevra, is Greek for zebra, the internationally recognized symbol for the rare disease community CELEBRATION, Fla., March 01, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics (NasdaqGS: ZVRA) (“Zevra” or the “Company” and formerly KemPharm, Inc.), a rare disease... Read More

New name, Greek for “zebra,” is the internationally-recognized symbol for the rare disease community Branding reflects Company’s commitment to making rare disease therapies available through its research, development, and commercialization efforts The Company’s common shares will trade on The Nasdaq Global Select Market under the new ticker symbol “ZVRA” starting on or about March 1, 2023 CELEBRATION, Fla., Feb. 22, 2023... Read More

Daniel Gallo, Ph.D., Appointed Senior Vice President of Medical Affairs and Advocacy Abbi Maher, J.D., Named Vice President of Legal Affairs CELEBRATION, Fla., Jan. 31, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS), neurodegenerative diseases, lysosomal storage disorders... Read More

CELEBRATION, Fla., Jan. 24, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced that Richard W. Pascoe, Chief Executive Officer of KemPharm, has issued a Letter to... Read More

CELEBRATION, Fla., Jan. 18, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas, announced its partnership with the Hypersomnia Foundation , which engages, informs and champions the... Read More

Expanded roles for Guenther and Mickle support broader evolution into a leading rare disease company CELEBRATION, Fla., Jan. 11, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the development of treatments for rare central nervous system (CNS) disorders, neurodegenerative diseases, lysosomal storage disorders and related treatment areas,... Read More

Matthew R. Plooster named Chairman of the Board of Directors Richard W. Pascoe appointed Chief Executive Officer Travis C. Mickle, Ph.D. transitions to President Joshua Schafer appointed Chief Commercial Officer and Executive Vice President of Business Development CELEBRATION, Fla., Jan. 09, 2023 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics company focused on the... Read More

Trial designed as a multi-center, dose-optimizing, double-blind, placebo-controlled, randomized-withdrawal study to evaluate safety and efficacy of KP1077, as well as to assess the symptoms and severity of “brain fog” Interim efficacy and safety data expected as early as Q3 2023 CELEBRATION, Fla., Dec. 21, 2022 (GLOBE NEWSWIRE) - - KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a rare disease therapeutics... Read More