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Xspray Pharma AB (publ) (Nasdaq Stockholm: XSPRAY) 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. 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.

On October 21, 2025 Xspray pharma reported 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. 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.

XS003 is an improved formulation of nilotinib (Tasigna) for the treatment of chronic myeloid leukemia (CML), developed using Xspray’s proprietary HyNap technology. Data demonstrate bioequivalence to the reference product at less than half the dose, as well as a significantly reduced food effect (29% compared to 82% for Tasigna). The food effect associated with the reference product complicates patient adherence and is related to the requirement that the patient must be in a fasting state, which is included in its so-called "boxed warning" regarding QTc prolongation and the risk of sudden death. The significantly reduced food effect with XS003 means it is expected that it may be taken with or without food and thereby avoid warning text regarding food interaction. Any final labeling and related warnings will be determined by the FDA during the review process.

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"The FDA’s decision to accept our NDA for review marks an important milestone," says Per Andersson, CEO of Xspray Pharma. "With two product candidates under FDA review, we are demonstrating that the HyNap platform has broad applicability and the potential to deliver more improved TKIs to the market."

Together with the company’s first product candidate Dasynoc, XS003 addresses a U.S. market of approximately USD 2.7 billion. The FDA’s notification confirms that the application is complete and that a full review is now underway. XS003 is manufactured at the same external facility as Dasynoc.

"Since the FDA now has a solid understanding of our manufacturing process, I am confident that the upcoming Pre-Approval Inspection will be completed without observations. It is essentially the same process that has previously been inspected. Furthermore, we are working closely with the manufacturer to ensure that all planned improvements to the other parts of the facility that previously received observations are completed well ahead of the PDUFA date," comments CEO Per Andersson.

(Press release, Xspray, OCT 21, 2025, View Source [SID1234656877])

Arrowhead Pharmaceuticals Announces Closing of Global License and Collaboration Agreement with Novartis

On October 21, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported that a global licensing and collaboration agreement with Novartis, previously announced on September 2, 2025, has now closed. Closing of the transaction was subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.

Summary of License and Collaboration Agreement

Novartis received an exclusive worldwide license to research, develop, manufacture, and commercialize ARO-SNCA, a preclinical stage program that utilizes Arrowhead’s Targeted RNAi Molecule (TRiMTM) platform for subcutaneous administration and delivery to the CNS designed to target the gene that encodes the alpha-synuclein protein as a potential treatment for patients with Parkinson’s Disease, and other synucleinopathies. Novartis may also select additional collaboration targets outside of Arrowhead’s current pipeline to be developed using the TRiMTM platform.

For all licensed programs under the agreement, Arrowhead will conduct and complete preclinical research activities necessary to enable a clinical trial application (CTA) filing. Novartis will then assume sole control over development, manufacturing, medical affairs, and commercialization activities.

Summary Financial Terms

Arrowhead will receive a $200 million upfront payment from Novartis and is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to the low double digits.

(Press release, Arrowhead Pharmaceuticals, OCT 21, 2025, View Source [SID1234656875])

Xencor to Host Webcast and Conference Call to Discuss Initial Results from the Ongoing Phase 1 Dose-Escalation Study of XmAb819 in Advanced Clear Cell Renal Cell Carcinoma

On October 21, 2025 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies for the treatment of cancer and autoimmune diseases, reported it will host a conference call and webcast on Friday, October 24 at 1:30 p.m. ET (10:30 a.m. PT) to discuss initial results from the ongoing Phase 1 dose-escalation study of XmAb819, an ENPP3 x CD3 T-cell engaging bispecific antibody, in development for patients with advanced clear cell renal cell carcinoma.

The live webcast of the conference call may be accessed through this link and through "Events & Presentations" in the Investors section of the Company’s website, located at investors.xencor.com. A recording will be available for at least 30 days.

The results will be presented at the AACR (Free AACR Whitepaper)-NCI-EORTC Conference on Molecular Targets and Cancer Therapeutics in Boston, Massachusetts, during Poster Session B on Friday, October 24 from 12:30 to 4:00 p.m. ET in a poster titled "Preliminary Phase 1 safety and antitumor activity of XmAb819, a first-in-class ENPP3 x CD3 bispecific antibody, in patients with advanced clear cell renal cell carcinoma (ccRCC)."

About XmAb819

XmAb819 is a first-in-class, tumor-targeted, T-cell engaging XmAb 2+1 bispecific antibody in development for patients with clear cell renal cell carcinoma (ccRCC). XmAb819 engages the immune system and activates T cells for highly potent and targeted lysis of tumor cells expressing ENPP3, an antigen highly expressed on kidney cancers. ENPP3 is a differentially expressed target, with high level expression in renal cell carcinoma (RCC) and low-level expression on normal tissues. With two tumor-antigen binding domains and one T-cell binding domain, Xencor’s XmAb 2+1 format enables antibodies to bind more avidly and selectively kill tumor cells with higher antigen density, potentially sparing normal cells. Xencor is conducting a Phase 1 study to evaluate XmAb819 in patients with advanced ccRCC.

(Press release, Xencor, OCT 21, 2025, View Source [SID1234656874])

Step Pharma to spotlight work in CTPS2-null solid tumours at 2025 EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics

On October 21, 2025 Step Pharma ("the Company"), the global leader in CTPS1 inhibition for targeted cancer treatment, reported that its Chief Scientific Officer, Philip Beer, will be presenting data on the identification of a CTPS1-dependent synthetic lethality in solid tumours at the 2025 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), being held from 22-26 October in Boston, MA.

The presentation will focus on the use of the AACR (Free AACR Whitepaper) Project GENIE data, a publicly accessible cancer registry of real-world clinico-genomic data, to identify loss of CTPS2 as a novel biomarker to select solid tumours with a synthetic lethal dependency on CTPS1. Deletion of CTPS2 was subsequently identified as a frequent event in many cancer types, with the highest prevalence found in ovarian cancer. As tumours derived from epithelial tissues can utilise both CTPS1 and CTPS2 in the final step of the CTP synthesis cascade, loss of CTPS2 in solid tumours exposes a dependence which could be targeted with CTPS1 inhibition to prevent tumour cell proliferation.

As a result of these findings, dencatistat, an orally available and highly potent inhibitor of CTPS1, which is currently being evaluated in a phase 1a dose escalation study in patients with solid tumours, will be evaluated in safety expansion cohorts in patients with CTPS2 null cancers, with the first being ovarian cancer (NCT06297525). Selecting patients whose tumours have deleted CTPS2 represents a precision oncology approach that is expected to maximise the therapeutic potential of dencatistat.

Philip Beer, Chief Scientific Officer of Step Pharma, said:

"Our work analysing clinico-genomic data led us to identify patients with CTPS2 null solid tumours as a promising therapeutic application for dencatistat. About 15-20% of ovarian cancers harbour this deletion, and so through inhibition of CTPS1 with dencatistat, we can selectively target the de novo pyrimidine synthesis pathway in ovarian cancer and other solid tumours. I look forward to presenting at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) international conference and to initiating the first expansion cohort in the near future."

Session: AACR (Free AACR Whitepaper) Project GENIE: Powering Drug Discovery Through an International Consortium’

Presentation title: Using GENIE data to drive oncology target identification and drug development.

Date: Thursday 23 October 2025

Time: 17:55 EDT

(Press release, Step Pharma, OCT 21, 2025, View Source [SID1234656872])

RAPT Therapeutics Announces Proposed Public Offering of Common Stock

On October 21, 2025 RAPT Therapeutics, Inc. (Nasdaq: RAPT) ("RAPT"), a clinical-stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing novel therapies for patients living with inflammatory and immunological diseases, reported that it has commenced an underwritten public offering of shares of its common stock. In addition, RAPT expects to grant the underwriters a 30-day option to purchase up to an additional 15% of the total number of shares of common stock RAPT is offering, at the public offering price, less underwriting discounts and commissions. All of the shares of common stock are being offered by RAPT. The proposed offering is subject to market conditions, and there can be no assurance as to whether or when the proposed offering may be completed or as to the actual size or terms of the proposed offering.

Leerink Partners, TD Cowen, Guggenheim Securities, Wells Fargo Securities and LifeSci Capital are acting as joint bookrunning managers for the proposed offering. H.C. Wainwright & Co. and Clear Street are acting as lead managers for the proposed offering.

The offering is being made pursuant to a shelf registration statement, including a base prospectus, filed by RAPT with the Securities and Exchange Commission (the "SEC"), which was declared effective by the SEC on August 17, 2023. The offering may be made only by means of a prospectus supplement and accompanying prospectus. A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website located at www.sec.gov. When available, electronic copies of the preliminary prospectus supplement and the accompanying prospectus may also be obtained from: Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at 1-800-808-7525 ex. 6132 or by email at [email protected]; TD Securities (USA) LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by email at [email protected]; Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected]; Wells Fargo Securities, LLC, Attention: Wells Fargo Securities, 90 South 7th Street, 5th Floor, Minneapolis, MN 55402, at 800-645-3751 (option #5) or email a request to [email protected]; or LifeSci Capital LLC at 1700 Broadway, 40th Floor, New York, New York 10019, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, RAPT Therapeutics, OCT 21, 2025, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-announces-proposed-public-offering-common-1 [SID1234656871])