Zevra Therapeutics, Inc. - ZVRA STOCK NEWS

Zevra Therapeutics announced FDA approval of MIPLYFFA™ (arimoclomol) as the first treatment for Niemann-Pick disease type C (NPC), an ultra-rare neurodegenerative disease. MIPLYFFA is indicated for use with miglustat to treat neurological manifestations in patients 2 years and older. The approval is based on a 12-month trial showing MIPLYFFA halted disease progression. Zevra received a rare pediatric disease priority review voucher and launched AmplifyAssist™, a patient support program.

MIPLYFFA will be commercially available in 8-12 weeks. The drug is administered orally three times daily, with dosage ranging from 47-124 mg based on body weight. Zevra estimates 900 people in the U.S. have NPC, with about one-third diagnosed. The approval marks a significant milestone for NPC patients, offering the first FDA-approved treatment option for this progressive and fatal disease.

Zevra Therapeutics (NASDAQ: ZVRA) presented new data for arimoclomol and OLPRUVA at the SSIEM 2024 Annual Symposium. Four posters focused on arimoclomol's efficacy and safety for treating Niemann-Pick disease type C (NPC). Key findings include:

1. Statistically significant treatment effect using the 4DNPCCSS scale
2. Long-term safety and efficacy in a 48-month open-label extension trial
3. Stable disease progression in real-world settings
4. Consistent safety profile in expanded access program

One poster on OLPRUVA showed increased drug exposure under fasting conditions, potentially allowing for lower effective dosages when taken without food.

Zevra Therapeutics (NASDAQ: ZVRA), a rare disease therapeutics company, has announced its participation in two upcoming investor conferences in September 2024. Neil F. McFarlane, President and CEO of Zevra, will present at:

1. The H.C. Wainwright 26th Annual Global Investment Conference in New York on September 9, 2024, at 2:00 p.m. ET.

2. The Cantor Annual Healthcare Conference in New York on September 17, 2024, at 2:30 p.m. ET.

Management will also be available for one-on-one meetings with registered attendees at both conferences. Live webcasts of the presentations will be accessible through the 'Events & Presentations' section of Zevra's investor website at investors.zevra.com.

Zevra Therapeutics (NASDAQ: ZVRA) reported Q2 2024 financial results and corporate highlights. Key points include:

1. FDA advisory committee voted favorably on arimoclomol's effectiveness for NPC treatment; PDUFA date set for September 21, 2024.

2. Pro forma cash position of $113.8 million as of June 30, 2024, including recent public offering.

3. Q2 2024 net revenue of $4.4 million, down from $8.5 million in Q2 2023.

4. Net loss of $19.9 million ($0.48 per share) in Q2 2024, compared to $2.6 million ($0.08 per share) in Q2 2023.

5. Cash runway extended into Q1 2027, excluding potential arimoclomol revenue.

Zevra Therapeutics (Nasdaq: ZVRA) has announced the pricing of an underwritten public offering of 9,230,770 shares of its common stock at $6.50 per share. This offering is expected to generate gross proceeds of approximately $60.0 million. The company has also granted underwriters a 30-day option to purchase up to an additional 1,384,615 shares. Cantor and William Blair are acting as joint book-running managers, with Citizens JMP and H.C. Wainwright & Co. as co-managers. The offering, subject to customary closing conditions, is anticipated to close on August 12, 2024. This public offering is being conducted under a registration statement filed with the SEC on June 4, 2024, which became effective on June 13, 2024.

Zevra Therapeutics (Nasdaq: ZVRA) has announced a proposed underwritten public offering of its common stock. The company plans to offer all shares and grant underwriters a 30-day option to purchase up to an additional 15% of shares at the public offering price. Cantor and William Blair are acting as joint book-running managers. The offering is subject to market conditions, with no assurance of completion or final terms. It will be made through a prospectus supplement and accompanying prospectus filed with the SEC. This announcement does not constitute an offer to sell or solicitation of an offer to buy securities.

Zevra Therapeutics (NASDAQ: ZVRA), a rare disease therapeutics company, has announced its participation in Canaccord Genuity's 44th Annual Growth Conference in Boston, MA. Neil F. McFarlane, President and CEO of Zevra, will present on Wednesday, August 14, 2024, at 10:30 a.m. ET.

The executive team will be available for one-on-one investor meetings with registered attendees throughout the conference. Interested parties can access live webcasts of the presentation through the 'Events & Presentations' section of Zevra's website at investors.zevra.com.

Zevra Therapeutics (NasdaqGS: ZVRA) announced that the FDA's Genetic Metabolic Diseases Advisory Committee voted 11-5 in favor of arimoclomol's effectiveness in treating Niemann-Pick disease type C (NPC). The committee's recommendation will be considered by the FDA as it reviews the arimoclomol New Drug Application (NDA), with a PDUFA action date of September 21, 2024. Zevra's CEO, Neil F. McFarlane, expressed confidence in arimoclomol's clinical benefit based on data from the pivotal trial, long-term open-label extension study, and expanded access programs. The GeMDAC's decision, while not binding, is a positive step towards potential FDA approval for this rare disease treatment.

Zevra Therapeutics (NASDAQ: ZVRA), a rare disease therapeutics company, has announced it will host a conference call and audio webcast on August 13, 2024, at 4:30 p.m. ET to review its corporate and financial results for the second quarter of 2024. The audio webcast will be accessible through the Investor Relations section of the company's website. Investors can join the meeting via conference call using the provided dial-in numbers. An archive of the webcast will be available for 90 days starting approximately at 5:30 p.m. ET on the same day.