Zevra Therapeutics to Present Multiple Analyses on MIPLYFFA® at the 22nd Annual WORLDSymposium™

Rhea-AI Summary

Zevra Therapeutics (Nasdaq: ZVRA) will present four abstracts on MIPLYFFA® (arimoclomol) at the 22nd Annual WORLDSymposium™ in San Diego, Feb 2-6, 2026. Four-year real-world safety and effectiveness data in Niemann-Pick disease type C (NPC) will be presented orally by Caroline Hastings, MD on Thursday, Feb 5, 2026 at 2:30 p.m. PT.

Three poster presentations on Feb 5 at 3:30 p.m. PT cover the U.S. Early Access Program (EAP), multi-year subgroup analyses, and efficacy from a randomized, placebo-controlled trial including arimoclomol plus miglustat. Zevra will host a non-CE satellite symposium on NPC diagnostics and therapeutics on Feb 5 at 12:15 p.m. PT. E-posters available via the WORLDSymposium app beginning Feb 3, 2026 at 5:00 p.m. PT, with on-demand access Feb 11–Mar 13, 2026. Zevra staff will be at booths #213 and #507 during the meeting.

Key Figures

Number of abstracts: 4 abstracts Real-world data duration: 4 years Poster number: 41 +5 more

8 metrics

Number of abstracts 4 abstracts MIPLYFFA data and real-world experience accepted at WORLDSymposium

Real-world data duration 4 years U.S. Early Access Program safety and effectiveness data for NPC

Poster number 41 Real-world Safety and Effectiveness outcomes from U.S. EAP

Poster numbers 193, 250, 273 Additional subgroup, efficacy, and long-term safety posters

Conference dates February 2–6, 2026 22nd Annual WORLDSymposium meeting window in San Diego

E-Poster access start February 3, 2026, 5:00 p.m. PT E-Posters available to registered attendees via mobile app

E-Poster on-demand window February 11–March 13, 2026 Period when e-Posters remain accessible on demand

Booth numbers 213 and 507 Zevra exhibit locations at WORLDSymposium

Market Reality Check

Price: $9.02 Vol: Volume 1,434,765 is above...

normal vol

$9.02 Last Close

Volume Volume 1,434,765 is above the 20-day average of 1,068,571 (relative volume 1.34). normal

Technical Price $8.97 is trading below the 200-day MA at $9.22 and 31.84% below the 52-week high.

Peers on Argus

Only one peer from the momentum scanner, ERAS, showed notable action, moving -6....

1 Down

Only one peer from the momentum scanner, ERAS, showed notable action, moving -6.73% without same-day news, suggesting today’s setup looks more stock-specific than sector-driven for ZVRA.

Historical Context

5 past events · Latest: Jan 08 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 08	Conference appearance	Positive	+1.8%	Presentation at major J.P. Morgan healthcare conference and investor meetings.
Dec 29	Commercial expansion	Positive	-0.6%	Exclusive distribution agreement to broaden access to MIPLYFFA outside Europe.
Dec 12	Equity inducement grants	Neutral	-1.9%	Stock option inducement awards to new employees under inducement plan.
Dec 02	Board changes	Neutral	-2.0%	Appointment of new director and retirement of existing board member.
Nov 20	CFO transition	Negative	-6.5%	CFO resignation and launch of search for a new chief financial officer.

Pattern Detected

Recent company updates often saw modest price moves, with leadership and corporate news sometimes met by negative reactions despite constructive business developments.

Recent Company History

Over the last few months, ZVRA has reported conference participation, commercial expansion for MIPLYFFA, equity inducement grants, and finance team changes. The Dec 29, 2025 distribution deal for MIPLYFFA outside Europe drew a slight negative move, while the Jan 8, 2026 J.P. Morgan conference appearance saw a +1.77% reaction. CFO transition headlines in November 2025 coincided with a -6.51% move, highlighting sensitivity to leadership changes relative to routine corporate updates like today’s scientific presentation news.

Market Pulse Summary

This announcement spotlights expanded clinical and real‑world evidence for MIPLYFFA in Niemann‑Pick ...

Analysis

This announcement spotlights expanded clinical and real‑world evidence for MIPLYFFA in Niemann‑Pick disease type C, including 4 abstracts and 4-year Early Access Program data at the 22nd WORLDSymposium. It reinforces Zevra’s focus on rare disease and builds on prior commercialization and distribution updates. Investors may focus on the depth of safety and efficacy outcomes, subgroup analyses, and how long‑term data in both pediatric and adult NPC patients shape future clinical and regulatory discussions.

Key Terms

niemann-pick disease type c, double-blind, randomized, placebo-controlled

3 terms

niemann-pick disease type c medical

"advances in Niemann-Pick disease type C (NPC)"

A rare inherited disorder in which cells cannot move cholesterol and other fats properly, causing them to build up like a clogged waste line and leading to progressive neurological problems, liver dysfunction, and variable symptoms that can appear from infancy to adulthood. It matters to investors because effective treatments are limited, so drug candidates can attract strong regulatory incentives, premium pricing and focused patient populations, but clinical development is small, risky and uncertain.

double-blind medical

"Double-blind, Randomized, Placebo-controlled NPC002 Trial"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

randomized, placebo-controlled medical

"Double-blind, Randomized, Placebo-controlled NPC002 Trial"

A randomized, placebo-controlled trial is a medical study where people are assigned by chance (like flipping a coin) to receive either the experimental treatment or an inactive substitute (a placebo), and neither participants nor often the researchers know who got which. For investors, this design is the gold standard for showing whether a therapy truly works beyond chance or expectation, which reduces uncertainty about regulatory approval, market adoption, and the commercial value of a drug or device.

AI-generated analysis. Not financial advice.

Four abstracts highlighting clinical data and real-world experience with MIPLYFFA accepted for presentation, including an oral presentation by Caroline Hastings, MD

Company to host satellite symposium on diagnostic and therapeutic advances in Niemann-Pick disease type C (NPC)

CELEBRATION, Fla., Jan. 26, 2026 (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 four abstracts discussing data and clinical experience associated with MIPLYFFA^® (arimoclomol) have been accepted for presentation at the 22^nd Annual WORLDSymposium™. Dr. Caroline Hastings will present four-year real-world safety and efficacy data of MIPLYFFA in patients with Niemann-Pick Disease Type C (NPC) during a podium presentation.

The poster presentations include outcomes from the U.S. Early Access Program (EAP), multi-year subgroup analyses, and efficacy results from a randomized, placebo-controlled clinical trial, further expanding the body of evidence characterizing MIPLYFFA in pediatric and adult NPC patients. In addition, Zevra will host a non-continuing education (Non-CE) sponsored satellite symposium focused on diagnostic and therapeutic challenges in NPC.

Podium Presentation Details

Poster Number:	41
Title:	Real-world Safety and Effectiveness of Arimoclomol in Patients with NPC: Outcomes from the U.S. Early Access Program (EAP) Over a 4-Year Period
Date/Time:	Thursday, February 5, 2026, at 2:30 p.m. PT
Presenter:	Caroline Hastings, MD, UCSF Benioff Children’s Hospital

Satellite Symposium Details

Title:	Charting a Path in Niemann-Pick Disease Type C: Diagnostic Challenges, Therapeutic Innovations, and Real-World Patient Cases
Date/Time:	Thursday, February 5, 2026, at 12:15 p.m. PT
Presenter:	Caroline Hastings, MD, UCSF Benioff Children’s Hospital

Poster Presentation Details

Poster Number:	193
Title:	Multi-year Subgroup Analyses of Niemann-Pick Disease Type C Participants Treated with Arimoclomol in the U.S. Early Access Program
Date/Time:	Thursday, February 5, 2026, at 3:30 p.m. PT
Presenter:	Kristina Julich, MD, Dell Children’s Medical Center

Poster Number:	250
Title:	Efficacy of Arimoclomol Combined with Miglustat at Months 3, 6, 9, and 12 of the Double-blind, Randomized, Placebo-controlled NPC002 Trial
Date/Time:	Thursday, February 5, 2026, at 3:30 p.m. PT
Presenter:	Eugen Mengel, MD, SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany

Poster Number:	273
Title:	Long-term Safety and Effectiveness of Arimoclomol in Adult and Pediatric Niemann- Pick disease type C Patients in the US Early Access Program (EAP)
Date/Time:	Thursday, February 5, 2026, at 3:30 p.m. PT
Presenter:	Damara Ortiz, MD, UPMC Children’s Hospital of Pittsburgh

E-Posters will be available to all registered attendees via the WORLDSymposium mobile app beginning at 5:00 p.m. PT on Tuesday, February 3, 2026, and will remain accessible throughout the live meeting. Registered attendees may continue to access e-Posters on demand from February 11 through March 13, 2026.

Members of the Zevra team will be available during the meeting, taking place February 2-6, 2026, in San Diego, CA. Attendees are invited to visit Zevra at booths #213 and #507 for more information.

About Zevra Therapeutics, Inc.

Zevra Therapeutics, Inc. is a purpose-driven, commercial-stage company focused on bringing life-changing therapeutics to people living with rare diseases. The company’s commercialization of its lead product, marketed in the U.S. for Niemann-Pick disease type C (NPC), a rare, progressive neurodegenerative disorder, provides a strong corporate foundation and validates its ability to advance therapies. In addition, the company is broadening access through geographic expansion opportunities and has a pipeline of rare disease programs. Zevra is a patient-centric organization guided by our values of accountability, integrity, innovation and courage, with the goal of creating long-term value for patients, partners, and shareholders.

For more information, please visit www.zevra.com or follow us on X and LinkedIn.

Caution Concerning Forward-Looking Statements

This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation statements regarding the Company’s expanded access program. Forward-looking statements are based on information currently available to Zevra and its current plans or expectations. They are subject to several known and unknown uncertainties, risks, and other important factors that may cause our 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 and other important factors are described in detail in the "Risk Factors" section of Zevra’s Annual Report on Form 10-K for the year ended December 31, 2024, filed on March 12, 2025, Quarterly Report on Form 10-Q for the three and nine months ended September 30, 2025, filed on November 5, 2025, as well as Zevra’s other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we cannot assure that such expectations will prove correct. These forward-looking statements should not be relied upon as representing our views as of any date after the date of this press release.

Investor Contact

Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com

Media Contact

Julie Downs
+1 (508) 246-3230 
jdowns@zevra.com

FAQ

What will Zevra (ZVRA) present about MIPLYFFA at WORLDSymposium 2026?

Zevra will present four abstracts on MIPLYFFA including a 4-year real-world safety and effectiveness oral presentation and three posters covering EAP data, subgroup analyses, and randomized trial efficacy.

When and where is the Caroline Hastings oral presentation on MIPLYFFA (ZVRA)?

Caroline Hastings will give the oral presentation on Thursday, February 5, 2026 at 2:30 p.m. PT in San Diego during WORLDSymposium.

What trial results on arimoclomol will be presented by Zevra (ZVRA) at WORLDSymposium?

Poster #250 reports efficacy of arimoclomol combined with miglustat from the double-blind, randomized, placebo-controlled NPC002 trial with results at months 3, 6, 9, and 12.

How can attendees access Zevra's MIPLYFFA e-posters at WORLDSymposium 2026?

E-posters are available to registered attendees via the WORLDSymposium mobile app starting Feb 3, 2026 at 5:00 p.m. PT and on demand Feb 11–Mar 13, 2026.

When is Zevra's satellite symposium on Niemann-Pick disease type C during WORLDSymposium 2026?

The non-CE satellite symposium 'Charting a Path in Niemann-Pick Disease Type C' is scheduled for Thursday, February 5, 2026 at 12:15 p.m. PT.

Where can investors or attendees find Zevra representatives at the WORLDSymposium event?

Zevra team members will attend the meeting Feb 2–6, 2026 in San Diego and will be available at booths #213 and #507 for more information.