RegulationBreakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (United States)

Structure/Sequence

Molecular FormulaC18H24ClN3O7

InChIKeyOHUSJUJCPWMZKR-FEGZNKODSA-N

CAS Registry289893-26-1

Clinical Trials associated with Arimoclomol

NL-OMON49378

/ CompletedNot Applicable

Interventional, randomised, partial double-blind, placebo- and positive controlled, multiple-dose, 4-way-cross-over trial Investigating the effect of arimoclomol on cardiac repolarization in healthy men. - CS0344 Orphazyme TQT

Start Date02 Jun 2020

Sponsor / Collaborator-

NCT03836716

/ TerminatedPhase 3

Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial

A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.

Start Date19 Sep 2019

Sponsor / Collaborator-

NCT04049097

/ TerminatedPhase 3

An Open-label, Non-randomized Trial to Investigate the Efficacy and Safety of Early Versus Delayed Start of Arimoclomol in Patients With Sporadic Inclusion Body Myositis Who Have Completed the IBM4809 Trial

A multicenter, nonrandomized, open-label, uncontrolled clinical extension trial designed to compare the efficacy and safety of early versus delayed start of arimoclomol in the treatment of Inclusion Body Myositis (IBM)

Start Date20 May 2019

Sponsor / Collaborator

University College London

[+1]

100 Clinical Results associated with Arimoclomol

100 Translational Medicine associated with Arimoclomol

100 Patents (Medical) associated with Arimoclomol

Literatures (Medical) associated with Arimoclomol

01 Aug 2025·MOLECULAR GENETICS AND METABOLISM

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

Article

Author: Harmatz, Paul ; Dali, Christine Í ; Himmelstrup, Louise ; Grønborg, Sabine Weller ; Héron, Bénédicte ; Hennermann, Julia B ; Grunewald, Stephanie ; Mengel, Eugen ; Maier, Esther M ; Sharma, Reena ; Guenther, Sven ; Gautschi, Matthias ; Cording, Malene ; Da Riol, Rosalia M ; Deodato, Federica ; Tylki-Szymanska, Anna ; Santra, Saikat ; Del Toro, Mireia

BACKGROUND:

This paper presents efficacy and safety outcomes from the 48-month open-label extension (OLE) of the phase 2/3 NPC-002 trial (NCT02612129) which evaluated arimoclomol treatment in patients with Niemann-Pick disease type C (NPC). Arimoclomol was recently approved by the US Food and Drug Administration for treatment of NPC in combination with miglustat.

METHODS:

Patients with NPC who completed the double-blind (DB) phase of the randomized controlled NPC-002 trial were eligible to continue in the OLE, during which all patients received arimoclomol in addition to routine clinical care. Primary efficacy outcomes were the 5-domain NPC Clinical Severity Scale (5DNPCCSS), and the rescored 4-domain NPCCSS (R4DNPCCSS), which was introduced post-hoc. Additional outcomes included NPC-specific measures (full scale NPCCSS, and NPC clinical database [NPC-cdb] score), and safety evaluations.

RESULTS:

Of the 50 patients who started the DB phase, 41 entered the OLE phase, with 29 completing 48 months. During the OLE, mean (SD) 5DNPCCS and R4DNPCCSS scores increased by 3.2 (4.8) and 2.7 (4.2) over 48 months, respectively. Among patients switching from placebo to arimoclomol after the DB phase, mean annual change in 5DNPCCSS decreased from 2.0 (on placebo) to 0.1 in the first year of receiving arimoclomol and mean annual change in R4DNPCCSS decreased from 1.9 to 0.2, indicating slowing of disease progression. Annual scores for both endpoints remained numerically smaller throughout the OLE than during the DB phase. The score pattern in the subset of patients who received miglustat as part of their standard care regime in addition to arimoclomol (N = 33) was similar to that seen in the total population. 17-domain NPCCSS (excluding hearing domains) and NPC-cdb results further supported sustained efficacy of arimoclomol. Arimoclomol was well-tolerated over 48 months, with no new safety concerns identified.

CONCLUSION:

The OLE of the NPC-002 trial provides evidence for a sustained reduction in disease progression for at least 5 years in a heterogeneous population of NPC patients receiving arimoclomol in addition to routine clinical care, with no new safety concerns. These results align with the statistically significant and clinically meaningful reduction in disease progression observed over 12-months in the DB phase, further highlighting the potential of arimoclomol as an effective and well tolerated disease modifying treatment for NPC.

01 Jul 2025·NEUROLOGICAL SCIENCES

Safety and efficacy of arimoclomol in amyotrophic lateral sclerosis: a systematic review and meta-analysis

Review

Author: Fiaz, Muhammad Hamza ; Zafar, Hafiz Muhammad Qasim ; Masood, Musa ; Minhas, Umm E Aimen ; Tahira, Sameen ; Almas, Muhammad Sameer ; Masood, Saad ; Hassan, Syed Saad Ul

OBJECTIVES:

Amyotrophic Lateral Sclerosis (ALS) is a debilitating motor neuron disorder characterized by muscle weakness, atrophy, and spasticity. This meta-analysis aims to assess the safety and efficacy of Arimoclomol in patients with ALS.

METHOD:

A comprehensive literature search was conducted on 3 databases to discover articles published up to August 2024. Included studies were randomized controlled trials (RCTs). Data was analysed using Review Manager (v5.4). Cochrane Risk of Bias-2 (RoB-2) was adopted to assess the quality of RCTs.

RESULTS:

A total of 359 patients were analysed, with 239 individuals in the Arimoclomol group and 120 individuals in the placebo group. The pooled analysis of the primary outcome, change in Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score from baseline, did not demonstrate a statistically significant difference favoring the Arimoclomol group (MD = 0.4495; 95% CI: -0.39, 1.27; p = 0.30). Similarly, secondary outcomes, including the Combined Assessment of Function and Survival (CAFS) rank score (MD = 1.00; 95% CI: -2.68, 4.67; p = 0.60), increase in transaminases (RR = 1.05; 95% CI: 0.19, 5.70; p = 0.95), mortality rate (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), and adverse events (RR = 0.86; 95% CI: 0.55, 1.34; p = 0.50), showed no statistically significant differences between the groups.

CONCLUSION:

This study does not conclusively demonstrate that Arimoclomol has beneficial effects on ALS patients' physical functionality but shows promise for safety. Further clinical trials are needed to explore the neuroprotective effects of Arimoclomol in the treatment of ALS.

01 Jun 2025·Molecular Genetics and Metabolism Reports

Efficacy results from a 12-month double-blind randomized trial of arimoclomol for treatment of Niemann-Pick disease type C (NPC): Presenting a rescored 4-domain NPC Clinical Severity Scale

Article

Author: Harmatz, Paul ; Héron, Bénédicte ; Roubertie, Agathe ; LaGorio, Lisa ; Porter, Forbes D ; Grunewald, Stephanie ; Mickle, Travis ; Maier, Esther M ; Dali, Christine Í ; Mengel, Eugen ; Berry-Kravis, Elizabeth ; Gautschi, Matthias ; Deodato, Federica ; Guenther, Sven ; Del Toro, Mireia ; Da Riol, Rosalia M ; Tylki-Szymanska, Anna ; Grønborg, Sabine Weller ; Santra, Saikat ; Patterson, Marc C ; Solomon, Beth ; Himmelstrup, Louise ; Hennermann, Julia B

Background:

In the 12-month, randomized, double-blind, placebo-controlled Phase 2/3 NPC-002 study (NCT02612129), arimoclomol significantly reduced annual disease progression versus placebo, measured by the 5-domain NPC Clinical Severity Scale (5DNPCCSS). Arimoclomol has been approved in the US for treatment of Niemann-Pick disease type C (NPC) in combination with miglustat. This paper introduces the rescored 4-domain NPCCSS (R4DNPCCSS) as a post-hoc primary endpoint in NPC-002, discusses its validation, and presents the results of the post-hoc primary analysis.

Methods:

To more accurately assess changes in disease course over a 12-month time period in a heterogeneous group of patients, the Cognition domain was removed from the 5DNPCCSS and the Swallow domain was rescored to reflect linearity in disease progression. Rescoring of the Swallow domain was based on input from clinical NPC and swallow experts from a qualitative interview-based study (N = 12), resulting in the R4DNPCCSS. To supplement prior validation analyses, data supporting the overall validity and reliability of the R4DNPCCSS was gathered through additional analyses of construct and convergent validity. The NPC-002 prespecified primary efficacy endpoint analysis based on the 5DNPCCSS score change from baseline to 12 months was repeated with R4DNPCCSS.

Results:

Construct validity analysis demonstrated high agreement between the R4DNPCCSS domain scores and the Clinical Global Impression Scale of Severity (CGI-S) and NPC Clinical Database (NPC-cdb) scores. Convergent validity was confirmed by strong correlations between the R4DNPCCSS domains and corresponding items on the Scale for Assessment and Rating of Ataxia (SARA), 9-hole peg test (9-HPT), and Video Fluoroscopic Swallowing Study (VFSS) performance tests. The NPC-002 post-hoc primary analysis showed a mean standard error (SE) change in R4DNPCCSS score of 0.35 (0.40) with arimoclomol (N = 34) versus 2.05 (0.54) with placebo (N = 16), and a treatment effect in favor of arimoclomol over placebo of -1.70 (p = 0.0155). In the miglustat subgroup analysis, mean (SE) change in R4DNPCCSS score was -0.23 (1.02) with arimoclomol (N = 22) versus 1.92 (3.37) with placebo (N = 12), representing a treatment effect of -2.21 (p = 0.0077).

Conclusion:

The R4DNPCCSS is a valid and reliable measure of disease progression demonstrating consistent outcomes with the prespecified 5DNPCCSS endpoint. Arimoclomol significantly slowed disease progression through 12 months as measured by the R4DNPCCSS versus placebo.

187

News (Medical) associated with Arimoclomol

13 Aug 2025

·www.globenewswire.com

XOMA Royalty Reports Second Quarter and Year to Date 2025 Financial Results and Highlights Recent Business Achievements

Business development: Purchased mezagitamab royalty and milestone rights held by BioInvent International and will secure royalty economic interests in two early-stage partnered assets through XOMA Royalty’s recently announced acquisition of LAVA Therapeutics. Company acquisitions: Announced XOMA Royalty’s acquisitions of Turnstone Biologics, LAVA Therapeutics, and HilleVax; acted as structuring agent and provided financing for XenoTherapeutics’ acquisition of ESSA Pharma; completed the sale of Kinnate pipeline assets and distributed upfront proceeds to Kinnate contingent value right (CVR) holders. Key Pipeline advancements: Rezolute completed enrollment in Phase 3 sunRIZE study of ersodetug in patients with congenital hyperinsulinism; the Marketing Authorization Application (MAA) for Day One Biopharmaceuticals and Ipsen’s tovorafenib was accepted for review by the European Marketing Authority (EMA), resulting in a $4 million milestone payment to XOMA Royalty; Zevra Therapeutics submitted an MAA with EMA seeking marketing approval for arimoclomol as a treatment for Niemann-Pick Type C. Cash receipts: In the first half of 2025, XOMA Royalty received $29.6 million in royalties and milestones from its partners, including $11.7 million during the second quarter. EMERYVILLE, Calif., Aug. 13, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its 2025 second quarter and year to date financial results and highlighted recent actions that have the potential to deliver shareholder value. “We continue to add to our diversified portfolio of both early- and late-stage assets through disciplined capital deployment and creative financial structures,” stated Owen Hughes, Chief Executive Officer of XOMA Royalty. “Recently approved drugs are addressing key unmet patient needs, which is driving increased royalty receipts, and we await data from several key Phase 3 assets over the coming quarters.” Royalty and Milestone Acquisitions CompanyAsset and Transaction DetailBioInventXOMA Royalty deployed $20 million to purchase the future mezagitamab royalty and milestone interests held by BioInvent and will pay an additional $10 million as the asset achieves a certain regulatory milestone. With its existing entitlement, plus the newly acquired economics from BioInvent, XOMA Royalty will be entitled to milestones of up to $16.25 million from Takeda and mid-single digit royalties on future mezagitamab commercial sales.LAVA TherapeuticsXOMA Royalty will secure an economic interest in two partnered assets through the Company’s recently announced acquisition of LAVA. The partnered assets are PF-08046052, which is being developed by Pfizer, and JNJ-89853413, which is being developed by Johnson & Johnson. Company Acquisitions CompanyTransaction DetailsTurnstone BiologicsXOMA Royalty and Turnstone Biologics entered into a definitive merger agreement, whereby XOMA Royalty will acquire Turnstone for $0.34 in cash per share of Turnstone common stock plus one non-transferable contingent value right (CVR). The transaction closed on August 11.HilleVaxXOMA Royalty and HilleVax have entered into a definitive merger agreement, whereby XOMA Royalty will acquire HilleVax for $1.95 per share upon closing. HilleVax stockholders also will receive a CVR that entitles them to receive certain potential payments following the closing of a pro rata portion of any remaining HilleVax cash in excess of $102.95 million; certain savings realized related to HilleVax’ office lease obligations, and should XOMA Royalty sell or out license the HilleVax norovirus programs within two years after the acquisition closes, 90% of any net proceeds received within five years by XOMA Royalty. The acquisition is expected to be completed in September.LAVA TherapeuticsXOMA Royalty and LAVA Therapeutics have entered into a definitive merger agreement, whereby XOMA Royalty will acquire LAVA for between $1.16 and $1.24 in cash per share of LAVA common stock plus one non-transferable CVR representing the right to receive 75% of the net proceeds related to LAVA’s two partnered assets and 75% of any proceeds related to the sale or out license of LAVA’s unpartnered programs. The acquisition is expected to close in the fourth quarter of 2025.XenoTherapeutics Acquisition of ESSA PharmaXOMA Royalty acted as structuring agent and is providing short-term financing for XenoTherapeutics’ acquisition of ESSA Pharma.KinnateXOMA Royalty sold the remaining Kinnate pipeline assets for a total of up to $270 million in upfront and milestone payments, plus royalties on commercial sales at rates ranging from low-single digits to mid-teens. In July, the Kinnate CVRs holders received their 85% share of the modest upfront payments. Pipeline Partner Updates through August 8, 2025 PartnerEventRezoluteIn May, the company announced the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) to its investigational therapy, ersodetug, for the treatment of hypoglycemia caused by tumor HI.1 In May, Rezolute announced the completion of enrollment in the Phase 3 sunRIZE study. Topline data are anticipated in December of 2025.2As a result of Rezolute completing enrollment in the study, XOMA Royalty received a $5 million milestone payment.TakedaThe first patient was dosed in Takeda’s Phase 3 clinical trial investigating mezagitamab as a treatment for adults with chronic primary immune thrombocytopenia (ITP). This achievement resulted in XOMA Royalty receiving a $3.0 million milestone payment, net, during the second quarter.Day One BiopharmaceuticalsIpsen, Day One’s partner outside of the U.S., announced its Marketing Authorization Application (MAA) for tovorafenib as a treatment for pediatric low-grade glioma (pLGG) had been accepted for review by the European Medicines Agency (EMA)3.Zevra TherapeuticsOn July 28, Zevra announced it had submitted an MAA to EMA for the evaluation of arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC)4.Gossamer BioOn June 16, Gossamer announced it had completed enrollment in the ongoing Phase 3 PROSERA Study that is evaluating seralutinib in Functional Class II and III pulmonary atrial hypertension (PAH) patients5. Topline results continue to be anticipated in February 20266.Daré BiosciencesAnnounced positive interim safety and efficacy results from its ongoing Phase 3 clinical trial evaluating the contraceptive effectiveness, safety, and acceptability of Ovaprene®, an investigational monthly, hormone-free intravaginal contraceptive.7 Anticipated 2025 Partner Events of Note PartnerEventRezoluteTopline data from sunRIZE Phase 3 clinical trial, which is investigating ersodetug in infants and children with congenital hyperinsulinism (cHI). Topline data are expected in December 20252. First patient dosed in the Phase 3 registrational study for ersodetug for the treatment of hypoglycemia due to tumor hyperinsulinism8.TakedaFirst patient dosed in Takeda’s Phase 3 clinical trial investigating mezagitamab as a treatment for adults with IgA Nephropathy.Gossamer BioActivates first clinical sites for the global, registrational Phase 3 SERANATA Study examining seralutinib in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) in the fourth quarter5.Daré BioscienceMakes Sildenafil Cream, 3.6% available commercially via prescription in the fourth quarter of 2025 as a compounded drug under Section 503B of the Federal Food, Drug, and Cosmetic Act.9 Commencement of one of two registrational Phase 3 clinical trials investigating Sildenafil Cream, 3.6%, for the treatment of female sexual arousal disorder10. Second Quarter and Year to Date 2025 Financial Results Tom Burns, Chief Financial Officer of XOMA Royalty, commented, “In the first six months of 2025, we have received $29.6 million in cash from partners, of which $16.0 million were royalty payments related to commercial sales and $13.6 million in milestone payments and fees. In the second quarter, we received $11.7 million in cash, $2.6 million from our partners’ commercial sales and $9.0 million from milestones and fees. Our partners’ product marketing activities continue to be well executed and as new commercial opportunities within our portfolio emerge, our line of sight to becoming cash flow positive on a consistent basis exclusively from the cash payments received from royalties grows clearer. With this outlook, we deployed $1.8 million to repurchase 81,682 shares of our common stock in the second quarter, bringing the total number of shares repurchased in 2025 to over 107,500 shares.” Income and Revenue: Income and revenue for the three and six months ended June 30, 2025, were $13.1 million and $29.0 million, respectively, as compared with $11.1 million and $12.6 million for the corresponding periods of 2024. The increase in both periods presented was primarily driven by increased income related to VABYSMO and OJEMDA. Research and Development (R&D) Expenses: R&D expenses for the three and six months ended June 30, 2025, were $0.1 million and $1.4 million, respectively, compared with $1.2 million for each of the corresponding periods of 2024. The R&D expenses in the first quarter of 2025 and the three- and six-month periods of 2024 were related to the clinical trial costs incurred subsequent to XOMA Royalty’s acquisition of Kinnate in April 2024 related to KIN-3248 and the associated wind-down activities. General and Administrative (G&A) Expenses: G&A expenses for the three and six months ended June 30, 2025, were $7.8 million and $15.9 million, respectively, as compared with $11.0 million and $19.5 million for the corresponding periods of 2024. The decrease in the second quarter of 2025 compared to the second quarter of 2024 was primarily due to the $3.6 million in exit packages paid to Kinnate senior leadership in the second quarter of 2024. In 2025, XOMA Royalty’s G&A expenses included non-cash stock-based compensation expenses during the three and six months ended June 30, 2025, of $1.6 million and $3.6 million, respectively, as compared to $2.7 million and $5.5 million for the corresponding periods of 2024. The 2024 periods reflect non-cash stock-based compensation related to the appointment of Mr. Hughes to full-time Chief Executive Officer and issuance of performance stock units. Credit Losses on Purchased Receivables: In the second quarter of 2024, XOMA Royalty recorded a one-time, non-cash credit loss on purchased receivables of $9.0 million and a corresponding reduction of royalty receivables of $9.0 million associated with the Aronora assets. To date there have been no credit losses in 2025. Amortization of Intangible Assets: Amortization of intangible assets relates to the IP acquired in the Company’s acquisitions of Pulmokine in November 2024 and the mezagitamab economics from the BioInvent transaction in May 2025. Amortization of non-cash intangible assets were $0.7 million and $1.2 million for the three and six months ended June 30, 2025. Gain on Acquisition of Kinnate: In the second quarter of 2024, XOMA Royalty recorded a $19.3 million gain on the acquisition of Kinnate due to the fair value of net assets that exceeded total purchase consideration. Interest Expense: For the three and six months ended June 30, 2025, interest expense was $3.2 million and $6.7 million, respectively, as compared with $3.4 million and $7.0 million for the corresponding periods of 2024. Interest expense relates to the Blue Owl Loan established in December 2023. Other Income, net: For the three and six months ended June 30, 2025, other income, net was $7.8 million and $7.7 million, respectively, as compared with $2.1 million and $4.0 million for the corresponding periods of 2024. The increases for the periods presented were primarily driven by increases in the fair value of XOMA Royalty’s investments in equity securities. Net Income: XOMA Royalty reported net income of $9.2 million and $11.6 million for the three and six months ended June 30, 2025, as compared to $16.0 and $7.4 million in the corresponding periods of 2025. Cash Position: On June 30, 2025, XOMA Royalty had cash and cash equivalents of $78.5 million (including $3.4 million in restricted cash), compared with cash and cash equivalents of $106.4 million (including $4.8 million in restricted cash) on December 31, 2024. In the second quarter of 2025, XOMA Royalty received $11.7 million in cash receipts including $2.6 million in royalties and commercial payments and $9.0 million in milestones and fees. During the second quarter of 2025, XOMA Royalty deployed $20 million to acquire additional economics in mezagitamab, repurchased approximately 81,700 shares of XOMA Royalty common stock for a cost of $1.8 million, and paid $1.4 million in dividends on the XOMA Royalty Perpetual Preferred stocks. In the first six months of 2025, XOMA Royalty received $29.6 million in cash receipts, including $16.0 million in royalties and commercial payments and $13.6 million in milestone payments and fees. During the first half of 2025, XOMA Royalty deployed $25.0 million to acquire additional assets for its royalty and milestone portfolio, repurchased approximately 107,500 shares of its common stock for a cost of $2.4 million, and paid $2.7 million in dividends on the XOMA Royalty Perpetual Preferred stocks. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com or follow XOMA Royalty Corporation on LinkedIn. Forward-Looking Statements/Explanatory NotesCertain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the timing and amount of potential commercial payments to XOMA Royalty and other developments related to VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DSUVIA® (sufentanil sublingual tablet), and Sildenafil Cream, 3.6%; the potential occurrences of the events listed under “Anticipated 2025 Events of Note”; the anticipated timings of regulatory filings and approvals related to assets in XOMA Royalty’s portfolio; and the potential of XOMA Royalty’s portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will”, “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-Q and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. 1 https://ir.rezolutebio.com/news/detail/354/rezolute-receives-breakthrough-therapy-designation-from-fda-for-ersodetug-in-the-treatment-of-hypoglycemia-due-to-tumor-hyperinsulinism2 https://ir.rezolutebio.com/news/detail/356/rezolute-announces-completion-of-enrollment-in-the-phase-3-sunrize-study-of-ersodetug-in-patients-with-congenital-hyperinsulinism3 https://www.ipsen.com/press-releases/ipsen-delivers-strong-sales-in-the-first-quarter-2025-and-confirms-its-full-year-guidance-3062256/4 https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-submits-marketing-authorization-application5 https://ir.gossamerbio.com/news-releases/news-release-details/gossamer-bio-announces-completion-enrollment-registrational6 https://ir.gossamerbio.com/news-releases/news-release-details/gossamer-bio-announces-second-quarter-2025-financial-results-and7 https://ir.darebioscience.com/news-releases/news-release-details/positive-interim-phase-3-results-highlight-potential-ovaprener8 https://ir.rezolutebio.com/news/detail/337/rezolute-announces-fda-clearance-of-ind-application-for-phase-3-registrational-study-of-rz358-for-treatment-of-hypoglycemia-due-to-tumor-hyperinsulinism9 https://ir.darebioscience.com/news-releases/news-release-details/dare-bioscience-and-rosy-wellness-announce-strategic10 https://ir.darebioscience.com/news-releases/news-release-details/dare-bioscience-announces-phase-3-plans-sildenafil-cream-36 XOMA CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(unaudited)(in thousands, except share and per share amounts) Three Months Ended June, Six Month Ended June 30, 2025 2024 2025 2024 Income and Revenues: Income from purchased receivables under the EIR method$6,007 $4,562 $12,077 $4,562 Income from purchased receivables under the cost recovery method 1,743 870 7,268 870 Revenue from contracts with customers 5,025 5,025 9,025 6,025 Revenue recognized under units-of-revenue method 354 629 671 1,119 Total income and revenues 13,129 11,086 29,041 12,576 Operating expenses: Research and development 69 1,161 1,362 1,194 General and administrative 7,802 11,004 15,948 19,465 Credit losses on purchased receivables - 9,000 - 9,000 Amortization of intangible assets 655 - 1,199 - Total operating expenses 8,526 21,165 18,509 29,659 Income (Loss) from operations 4,603 (10,079) 10,532 (17,083) Other income (expense) Gain on the acquisition of Kinnate - 19,316 - 19,316 Change in fair value of embedded derivative related to RPA - 8,100 - 8,100 Interest expense (3,236) (3,402) (6,703) (6,953)Other income, net 7,824 2,050 7,729 4,010 Net income$9,191 $15,985 $11,558 $7,390 Net income available to common stockholders, basic$5,522 $10,224 $6,225 $3,253 Basic net income per share available to common stockholders$0.46 $0.88 $0.52 $0.28 Weighted average shares used in computing basic net income per share available to common stockholders 12,007 11,643 11,988 11,611 Net income available to common stockholders, diluted$7,823 $14,617 $8,822 $4,654 Diluted net income per share available to common stockholders$0.44 $0.84 $0.50 $0.27 Weighted average shares used in computing diluted net income per share available to common stockholders 17,761 17,321 17,777 17,263 XOMA CORPORATIONCONDENSED CONSOLIDATED BALANCE SHEETS(unaudited)(in thousands, except share and per share amounts) June 30, December 31, 2025 2024 ASSETS Current assets: Cash and cash equivalents$75,060 $101,654 Short-term restricted cash 80 1,330 Investment in equity securities 8,801 3,529 Trade and other receivables, net 1,817 1,839 Short-term royalty and commercial payment receivables under the EIR method 17,960 14,763 Short-term royalty and commercial payment receivables under the cost recovery method 700 413 Prepaid expenses and other current assets 507 2,076 Total current assets 104,925 125,604 Long-term restricted cash 3,345 3,432 Property and equipment, net 26 32 Operating lease right-of-use assets 288 319 Long-term royalty and commercial payment receivables under the EIR method 4,775 4,970 Long-term royalty and commercial payment receivables under the cost recovery method 58,937 55,936 Exarafenib milestone asset (Note 4) 3,402 3,214 Investment in warrants 609 - Intangible assets, net 45,434 25,909 Other assets - long term 1,715 1,861 Total assets$223,456 $221,277 LIABILITIES, CONVERTIBLE PREFERRED STOCK AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable$1,138 $1,053 Accrued and other liabilities 5,411 5,752 Contingent consideration under RPAs, AAAs, and CPPAs - 3,000 Operating lease liabilities 472 446 Unearned revenue recognized under units-of-revenue method 1,434 1,361 Preferred stock dividend accrual 1,368 1,368 Current portion of long-term debt 11,672 11,394 Total current liabilities 21,495 24,374 Unearned revenue recognized under units-of-revenue method – long-term 3,666 4,410 Exarafenib milestone contingent consideration 3,402 3,214 Long-term operating lease liabilities 238 483 Long-term debt 102,201 106,875 Total liabilities 131,002 139,356 Convertible preferred stock, $0.05 par value, 5,003 shares authorized, issued and outstanding as of June 30, 2025 and December 31, 2024 20,019 20,019 Stockholders’ equity: 8.625% Series A cumulative, perpetual preferred stock, $0.05 par value, 984,000 shares authorized, issued and outstanding as of June 30, 2025 and December 31, 2024 49 49 8.375% Series B cumulative, perpetual preferred stock, $0.05 par value, 3,600 shares authorized, 1,600 shares issued and outstanding as of June 30, 2025 and December 31, 2024 — — Common stock, $0.0075 par value, 277,333,332 shares authorized, 12,062,466 and 11,952,377 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively 90 90 Additional paid-in capital 1,300,066 1,298,747 Accumulated other comprehensive income 122 73 Accumulated deficit (1,227,892) (1,237,057)Total stockholders’ equity 72,435 61,902 Total liabilities, convertible preferred stock and stockholders’ equity$223,456 $221,277 XOMA CORPORATIONCONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (unaudited)(in thousands) Six Months Ended June 30, 2025 2024 Cash flows from operating activities: Net income$11,558 $7,390 Adjustments to reconcile net income to net cash provided by (used in) operating activities: Adjustment for income from EIR method purchased receivables (3,935) (4,562)Stock-based compensation expense 3,588 5,546 Credit losses on purchased receivables — 9,000 Gain on the acquisition of Kinnate — (19,316)Common stock contribution to 401(k) 141 118 Amortization of intangible assets 1,199 — Depreciation 6 5 Accretion of long-term debt discount and debt issuance costs 749 508 Non-cash lease expense 31 29 Change in fair value of equity securities (5,173) (535)Change in fair value of available-for-sale debt securities classified as cash equivalents 49 — Change in fair value of derivatives (5) — Changes in assets and liabilities: Trade and other receivables, net 22 478 Prepaid expenses and other assets 1,715 (603)Accounts payable and accrued liabilities (387) 921 Operating lease liabilities (219) (82)Unearned revenue recognized under units-of-revenue method (671) (1,117)Net cash provided by (used in) operating activities 8,668 (2,220) Cash flows from investing activities: Net cash acquired in Kinnate acquisition — 18,926 Payments of consideration under RPAs, AAAs, and CPPAs (8,000) (37,000)Receipts under RPAs, AAAs, and CPPAs 2,039 16,741 Payment for BioInvent contract-based intangible asset (20,614) — Purchase of property and equipment — (17)Purchase of equity securities (99) — Net cash used in investing activities (26,674) (1,350) Cash flows from financing activities: Principal payments – debt (5,065) (3,616)Debt issuance costs and loan fees paid in connection with long-term debt (80) (661)Payment of preferred stock dividends (2,736) (2,736)Repurchases of common stock (2,370) (13)Proceeds from exercise of options and other share-based compensation 896 2,353 Taxes paid related to net share settlement of equity awards (570) (1,387)Net cash used in financing activities (9,925) (6,060) Net decrease in cash, cash equivalents and restricted cash (27,931) (9,630)Cash, cash equivalents and restricted cash at the beginning of the period 106,416 159,550 Cash, cash equivalents and restricted cash at the end of the period$78,485 $149,920 Supplemental Cash Flow Information: Cash paid for interest$6,078 $3,780 Cash paid for taxes$277 $— Non-cash investing and financing activities: Estimated fair value of the Exarafenib milestone asset$— $2,922 Estimated fair value of the Exarafenib milestone contingent consideration$— $2,922 Right-of-use assets obtained in exchange for operating lease liabilities in Kinnate acquisition$— $824 Relative fair value basis reduction of rights-of-use assets in Kinnate acquisition$— $(824)Accrual of contingent consideration under the Affitech CPPA$— $3,000 Preferred stock dividend accrual$1,368 $1,368 Excise tax accrual due to stock repurchases$24 $— Transaction costs in connection with BioInvent IP acquisition included in accounts payable$111 $— Investor contact:Media contact:Juliane SnowdenKathy VincentXOMA Royalty CorporationKV Consulting & Management+1-646-438-9754kathy@kathyvincent.com juliane.snowden@xoma.com

Phase 3Financial StatementBreakthrough TherapyAcquisitionNDA

29 Jul 2025

·www.globenewswire.com

Zevra Therapeutics Submits Marketing Authorization Application to European Medicines Agency to Review Arimoclomol for the Treatment of Niemann-Pick Disease Type C

July 28, 2025 -- 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 an ultra-rare, neurological disease caused by genetic mutations that result in lipid accumulation in cells, leading to visceral, neurological, and psychiatric symptoms. Arimoclomol is the only treatment shown to directly target the underlying pathology of NPC by increasing gene expression for improved lipid clearance. Arimoclomol for the treatment of NPC has been designated as an Orphan Medicinal Product by the EMA. Arimoclomol is marketed in the U.S. under the brand name MIPLYFFA®. “This EMA submission marks a significant milestone for the Company as we continue to expand access to MIPLYFFA® in NPC patients across the globe,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer. “Concurrently, we continue to advance our global Expanded Access Program, with 89 patients enrolled in Europe at the end of Q2, reinforcing MIPLYYFA’s potential to serve as a foundational treatment option across European markets. We extend our deepest gratitude to everyone involved – especially those patients and clinicians who participated in our clinical development programs and look forward to potential European approval of arimoclomol to expand on our approval in the U.S. last year.” The EMA will review the application under the centralized marketing authorization procedure. If a marketing authorization is granted by the European Commission, the authorization is valid in all EU Member States as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway. Adrian Quartel, M.D., Zevra’s Chief Medical Officer added, “The extensive data generated for arimoclomol has shown long-term, meaningful clinical outcomes with 5 to 7 years of patient experience across more than 270 NPC patients through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. We are confident in the strength of our MAA data package and look forward to our interactions with the EMA.” Zevra currently offers an EAP for NPC patients in certain European countries, and more information can be found at: https://zevra.com/patients-and-providers/expanded-access-policy. Arimoclomol is Zevra’s therapy for the treatment of Niemann-Pick disease type C (NPC), which was approved by the U.S. Food and Drug Administration on Sep. 20, 2024. Arimoclomol increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. Arimoclomol has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal Phase 3 trial, arimoclomol halted disease progression compared to placebo over the twelve month duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. Arimoclomol has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC. Niemann-Pick disease type C (NPC) is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various cell types, including neurons. The disease is caused by mutations in the NPC1 or NPC2 genes, which are responsible for making the NPC1 and NPC2 lysosomal proteins. Both children and adults can be affected by NPC with varying clinical presentations. Those living with NPC can lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation, and fine motor skills. Disease diagnosis can often take years, with disease progression being irreversible and often leading to early mortality. Zevra Therapeutics, Inc. is a commercial-stage rare disease company combining science, data, and patient needs to create transformational therapies for diseases with limited or no treatment options. Our mission is to bring life-changing therapeutics to people living with rare diseases. With unique, data-driven development and commercialization strategies, the Company is overcoming complex drug development challenges to make new therapies available to the rare disease community. The content above comes from the network. if any infringement, please contact us to modify.

Orphan DrugDrug ApprovalClinical ResultNDA

25 Jul 2025

·www.fiercepharma.com

Europe's CHMP gives thumbs-up to Lilly's Kisunla and Gilead's long-acting PrEP drug Yeytuo

Europe’s Committee for Medicinal Products for Human Use has reversed its decision on Eli Lilly's Alzheimer's disease treatment Kisunla, giving it a positive opinion in a limited patient population. Four months after giving Eli Lilly’s Alzheimer’s disease treatment a thumbs-down, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended it for approval, albeit in a restricted patient population.The positive opinion covers Kisunla's use in patients with early Alzheimer’s symptoms who have one or no copies of the apolipoprotein E ε4 (ApoE4) gene. The drug is not recommended for individuals carrying two copies of the ApoE4 gene. Those patients are more susceptible to amyloid-related imaging abnormalities (ARIA), which are a primary side effect of currently approved Alzheimer’s drugs.The restriction is the same as the one applied by European regulators when it granted marketing authorization to Eisai and Biogen’s Alzheimer’s drug Leqembi three months ago. Those companies went through the same rejection/appeal process as Lilly did with European regulators.Lilly went through the same go-round with regulators in the U.S. before it secured approval of Kisunla in July of last year. Then, earlier this month, the FDA signed off on a new dosing regimen for the treatment, featuring a more gradual titration, which leads to a significantly lower rate of ARIA-related brain swelling. The new dosing regimen also applies to Thursday’s thumbs-up from the CHMP. Endorsement for Gilead’s PrEP injection The CHMP has also provided a positive opinion on Gilead Sciences' HIV pre-exposure prophylaxis (PrEP) Yeytuo (lenacapavir), a twice-a-year subcutaneous injection which has been hailed as one of the most important breakthroughs in the effort to end the epidemic.The FDA blessed the shot last month under its commercial name Yeztugo.The CHMP recommendation applies to adults and adolescents and came under an accelerated pathway, “because it is considered to be of major public health interest in the EU and the rest of the world,” the regulator said.It was also evaluated under the EU-Medicines for all program, which included participation from the World Health Organization and regulators from several African and Asian nations. The program helps speed approvals where “regulatory capacity may be limited,” the CHMP said.“Lenacapavir for PrEP has the potential to become a critical tool for public health, helping to expand prevention options for people who face the highest barriers to care,” Dietmar Berger, M.D., Ph.D., Gilead’s chief medical officer, said in a release. Other CHMP decisions A month after Supernus Pharmaceuticals made a $561 million bet on Zurzuvae, buying out its developer Sage Therapeutics, the CHMP has registered a positive opinion on the treatment for postpartum depression. The Biogen-partnered treatment was endorsed by the FDA in August 2023, along with a rejection from the regulator in a much larger patient population, major depressive disorder.The European regulator has also signed off on rare disease specialist IntraBio’s Aqneursa for the treatment of the lysosomal storage disorder Niemann-Pick disease type C (NPC). The endorsement comes 10 months after the FDA approved two treatments for NPC in consecutive weeks, Aqneursa and Zevra Therapeutics’ Miplyffa, which has yet to win a nod in Europe.Less than three weeks after the FDA approved KalVista Pharmaceuticals' Ekterly for acute attacks of hereditary angioedema (HAE), the CHMP has followed suit. This is the first oral treatment for HAE recommended for approval in Europe, reducing the burden of injection on patients and speeding up the time between onset of attack and administration of the medicine, the CHMP said. Ono Pharmaceuticals has won a CHMP nod for Romvimza, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT), a condition where the tissue surrounding the joints and tendons expands abnormally, forming outgrowths of the joint. The drug, which was acquired last year in Ono’s $2.4 billion buyout of Deciphera Pharmaceuticals, was endorsed by the FDA in February of this year, joining Daiichi Sankyo’s Turalio as the only other marketed treatment for TGCT.Ionis Pharmaceuticals' Tryngolza, for the treatment of adults with familial chylomicronemia syndrome, has gotten a thumbs-up from CHMP. The rare, autosomal recessive inherited disorder is characterized by elevated levels of triglycerides in the blood. The FDA approved the treatment in December of last year.Also receiving a CHMP nod is Servier’s Voranigo for low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumors. The FDA blessed the treatment in August of last year.

Drug ApprovalAccelerated Approval

100 Deals associated with Arimoclomol

External Link

KEGG	Wiki	ATC	Drug Bank
-	Arimoclomol	-	DB05025

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Niemann-Pick Disease, Type C	United States	Zevra Denmark as	20 Sep 2024
Niemann-Pick Disease, Type C	United States	Zevra Therapeutics, Inc.	20 Sep 2024

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myositis, Inclusion Body	Phase 3	United States	University College London	20 May 2019
Myositis, Inclusion Body	Phase 3	United Kingdom	University College London	20 May 2019
Amyotrophic Lateral Sclerosis	Phase 3	United States	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Belgium	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Canada	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	France	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Germany	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Italy	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Netherlands	Zevra Denmark as	31 Jul 2018
Amyotrophic Lateral Sclerosis	Phase 3	Poland	Zevra Denmark as	31 Jul 2018

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02612129 (NPC-002, Pubmed) Manual	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (DB phase)	-	Positive	01 Aug 2025
				arimoclomol (OLE phase)	akxtxkhpkc(vpznfbqado) = ikejcsbiuz cuescbigdt (zgkmcdmrbr, 4.8) View more
NCT02612129 (FDA_CDER) Manual	Phase 3	Niemann-Pick Disease, Type C	50	MIPLYFFA with miglustat	sxwogtcklf(ytjsklhtvw) = zqobwmosna kfzfzexmia (ljcczfiaol, 1)	Positive	20 Sep 2024
				Placebo with miglustat	sxwogtcklf(ytjsklhtvw) = nmyzqpakfs kfzfzexmia (ljcczfiaol, 3.4)
NCT04049097 (CTgov)	Phase 3	Myositis, Inclusion Body	121	Arimoclomol	ifhsufpwzv(zkhqgjmzsq) = vetktodksh ovxcqxeuvk (bywgirywhp, 2.60) View more	-	15 Sep 2023
NCT03836716 (CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	120	Arimoclomol	zotnlovqty = vlpvjalyfy svkeudphvn (dwbnvldudl, emhtfrdhze - mjbcovbfit) View more	-	24 Aug 2023
NCT03491462 (ORARIALS-01, CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	245	Arimoclomol (Arimoclomol (up to 76 Weeks))	gbrqcvuenw(qnqkxqrdop) = cpqpcdjvcs tvivhogakt (ohgeyjpfof, 0.291) View more	-	24 Aug 2023
				Placebo (Placebo (up to 76 Weeks))	gbrqcvuenw(qnqkxqrdop) = jnacmmftxj tvivhogakt (ohgeyjpfof, 0.283) View more
NCT02612129 (NPC-002, CTgov)	Phase 2/3	Niemann-Pick Disease, Type C	50	arimoclomol (Arimoclomol (12-month Double-blind Phase))	uuwwtalatj(roldrcmdxt) = psvpjrvxzj bztzfikzdz (obgozllurc, ntwbhnthhc - mohsgmolia) View more	-	09 Jun 2023
				Placebo (Placebo (12-month Double-blind Phase))	uuwwtalatj(roldrcmdxt) = eybfyuxlyx bztzfikzdz (obgozllurc, inecszppwp - wjevyevskv) View more
NCT02753530 (CTgov)	Phase 2	Myositis, Inclusion Body	152	Arimoclomol (Arimoclomol (20 Months))	vkotiedief(fedhtjlxrq) = ovbuutqqvy wfrmdezowj (qrnyguihzc, yrwdllubjz - dpbapfztlg) View more	-	10 May 2023
				Placebo (Placebo (20 Months))	vkotiedief(fedhtjlxrq) = ppgwjcjftx wfrmdezowj (qrnyguihzc, xfnkzzveec - lgjiaoavfr) View more
NCT03491462 (ORARIALS-01, GlobeNewswire) Manual	Phase 3	Amyotrophic Lateral Sclerosis	245	arimoclomol 248 mg	pvngseoofb(onboxioiag) = did not meet its primary endpoints sxdynbempo (metrmtxtqa ) View more	Negative	07 May 2021
				Placebo
NCT00706147 (Pubmed \| Ann Neurol \| Neurology) Manual	Phase 2/3	Amyotrophic Lateral Sclerosis	36	arimoclomol	lhpfjoigto(dbxrwdkejl) = czcfsmtppx arwrxcbvxb (krdrijjehx, -0.63 to 1.63) View more	Positive	13 Feb 2018
				Placebo	-
NCT00769860 (CTgov)	Phase 2/3	Myositis, Inclusion Body	24	Arimoclomol (Arimoclomol)	vkuuckpzla = pfdrghsjky wzdleikkiz (xlxbkffaeq, doingziouf - dlzjaykexj) View more	-	19 Jan 2017
				placebo (Placebo)	vkuuckpzla = tjnwkitquy wzdleikkiz (xlxbkffaeq, vhjpiignhj - kaydjyucbo) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Arimoclomol

Overview

Basic Info

Structure/Sequence

Related

External Link

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation