Nilopki (nilotinib amorphous) / Xspray Pharma - LARVOL DELTA

XS003 ELIMINATES NILOTINIB FOOD INTERACTIONS ENABLING OPTIMIZED DOSE REDUCTIONS, RESULTING IN MITIGATED QT-PROLONGATION RISK (EHA 2026) - "2012. 203585Orig1s000CrossR.pdf"

Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia

Xspray Pharma AB…has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for Nilopki, an optimized formulation of nilotinib (Xspray Pharma Press Release) - "The FDA has determined that they are currently not able to approve the NDA based on three reasons. Firstly, they require that each Tasigna dose level must have a single corresponding Nilopki dose level starting from a dose with an exposure similar to the Tasigna 200 mg. Secondly, the FDA has requested more data to adequately demonstrate commercial manufacturing capability of nilotinib ASD batches. Thirdly, as previously communicated, Xspray’s third-party manufacturer needs to provide satisfactory responses to FDA’s inspection findings and a pre-approval inspection of the facility related to Xspray’s manufacturing may need to be conducted. Xspray will now carefully analyze the CRL in detail and seek clarification where needed in dialogue with the FDA and industry experts."

CRL • Chronic Myeloid Leukemia

FDA accepts trade name Nilopki for Xspray's drug candidate XS003 (nilotinib) (Nasdaq) - "Upon market approval, Xspray plans to launch Nilopki in the US during the second half of 2026, coordinated with the planned launch of Dasynoc...Xspray Pharma's application for market approval (NDA) for Nilopki is under review by the FDA with a PDUFA date of June 18, 2026...Xspray’s lead drug candidate Dasynoc (dasatinib) is also being reviewed by the FDA, with a PDUFA date of August 25, 2026, for the treatment of CML and acute lymphoblastic leukemia (ALL). Upon market approval, Xspray plans to launch both products in the US in the second half of 2026."

FDA event • Launch non-US • PDUFA • Chronic Myeloid Leukemia

Xspray resolves on a rights issue of approximately SEK 83 million with an over-allotment issue (TradingView) - "The Rights Issue is carried out to support the continued preparations and launch of the Company’s two lead product candidates, Dasynoc and XS003-nilotinib, to advance the Company’s broader development portfolio as well as to improve the manufacturing capabilities. Based on the Company’s current plans, assuming approval and launch of Dasynoc and XS003-nilotinib, the proceeds are expected to cover working capital requirements through 2026."

Financing • Acute Lymphocytic Leukemia • Chronic Myeloid Leukemia

Xspray Pharma re-submits its FDA application for Dasynoc (TradingView) - "The resubmission includes the additional information requested by the FDA. A new PDUFA-date is expected to be set for August 2026 at the latest...The current resubmission includes additional data specifically designed to address FDA’s concerns regarding risk for medication error. A qualitative human factors study has been completed to address this, confirming high prescriber comprehension using the new tablet strengths....'We maintain our plan to launch two products, Dasynoc and XS003 nilotinib during the second half of 2026.'"

FDA approval • FDA filing • Launch US • Acute Lymphocytic Leukemia • Chronic Myeloid Leukemia

Xspray Pharma AB…announces that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for XS003 (nilotinib) for review under the 505(b)(2) regulatory pathway. (Xspray Pharma Press Release) - "The FDA has set a PDUFA date to June 18, 2026, which is the date by when the agency is expected to announce a decision on the application."

FDA filing • PDUFA • Chronic Myeloid Leukemia

XSpray Pharma achieves significant milestone - demonstrating bioequivalence with absorption advantages compared to Tasigna (Nasdaq) - P1 | N=27 | NCT02068898 | Sponsor: XSpray Microparticles | "Xspray Pharma has now completed the population pharmacokinetic (PopPK) modeling that constitutes key regulatory documentation ahead of submitting a New Drug Application (NDA) for the product candidate XS003. The analysis confirms bioequivalence with the reference drug Tasigna, at less than half the dose – a strong indication marking a potential paradigm shift in the treatment of CML with nilotinib...The results confirm formal bioequivalence between XS003 and Tasigna in terms of systemic exposure (AUC and Cmax) – despite the XS003 dose being less than half that of the reference drug. In addition, a clinical study has shown that XS003 has significantly improved food interaction and a more predictable dose response, which can facilitate precise dose adjustments in clinical practice....With these results, Xspray Pharma plans to submit a NDA application in the near future."

P1 data • Chronic Myeloid Leukemia

Xspray Pharma…has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its product candidate XS003 (nilotinib) for the treatment of chronic myeloid leukemia (CML) (Businesswire) - "The application is based on successful studies demonstrating bioequivalence with the reference product Tasigna....Data from registration studies demonstrate bioequivalence with the reference product, despite XS003 being administered at less than half the dose of the reference product....Xspray expects the FDA to initiate its review within 60 days, with a regulatory decision anticipated approximately eight months thereafter."

FDA approval • FDA filing • Chronic Myeloid Leukemia

Xspray Pharma’s XS003 Study Shows Matching Bioavailability to Tasigna at More than a 50% Lower Dose (Businesswire) - P=NA | N=NA | "Xspray Pharma AB...announced today new clinical data from its XS003 registration study program....In the announced study, XS003 demonstrates matching bioavailability to Tasigna, with a 50% reduced dose.... The results show that the XS003 amorphous formulation of nilotinib uses less than 50% of the active ingredient compared to crystalline forms. Thus, there is significantly less excess material available for overdosing, potentially making it a safer drug....XS003 is being developed under the 505(b)(2) NDA process, which streamlines the approval process, and is expected to be submitted to the FDA in the first half of 2025."

Clinical data • FDA filing • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology