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PharmaMar receives US$50 million milestone payment from Jazz Pharmaceuticals for the FDA’s approval of Zepzelca® (lurbinectedin)

On October 15, 2025 PharmaMar Group (MSE: PHM) reported it has received a milestone payment of $50 million from Jazz Pharmaceuticals plc (Nasdaq:JAZZ) related to the full approval granted on October 2nd of this year from the U.S Food and Drug Administration (FDA) for Zepzelca (lurbinectedin) in combination with atezolizumab (Tecentriq) as a first-line maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.

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In December 2019, PharmaMar entered into an exclusive license agreement with Jazz Pharmaceuticals for lurbinectedin in the United States.

(Press release, PharmaMar, OCT 15, 2025, View Source [SID1234656675])

Keros Therapeutics Announces Plan for Return of $375 Million in Excess Capital

On October 15, 2025 Keros Therapeutics, Inc. ("Keros" or the "Company") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta ("TGF-ß") family of proteins, reported that, as part of its previously announced $375 million capital return program, it has entered into share purchase agreements (the "Purchase Agreements") to repurchase all of the shares of the Company’s common stock beneficially owned by each of ADAR1 Capital Management ("ADAR1") and Pontifax Venture Capital ("Pontifax") at a purchase price of $17.75 per share in cash. The aggregate purchase price for the repurchase is approximately $181 million, which is expected to be funded from the Company’s existing cash and cash equivalents. The transactions are expected to close on October 15, 2025. In connection with the Purchase Agreements, Mr. Ran Nussbaum and Mr. Tomer Kariv have tendered their resignations to the Board of Directors, effective immediately.

In connection with its entry into the Purchase Agreements, Keros also reported that it plans to distribute 25% of any net cash proceeds it receives on or before December 31, 2028 from its global license agreement with Takeda Pharmaceuticals U.S.A., Inc. ("Takeda") to Keros stockholders.

Following the closing of the transactions with ADAR1 and Pontifax, the Company intends to commence a tender offer for up to $194 million in value of shares of the Company’s common stock, subject to market conditions, at a purchase price of $17.75 per share in cash. The Company presently intends to commence the tender offer by the end of October 2025. The Company expects to fund the tender offer from its existing cash and cash equivalents.

"We are pleased to have reached these agreements with ADAR1 and Pontifax, and look forward to completing our capital return program in the near term. The capital return program – which includes an additional commitment to distribute future near-term Takeda proceeds – reflects our confidence in the outlook for Keros and the prospects for our key clinical program, KER-065," said Jean-Jacques Bienaimé, Chair of the Board of Directors. "With a more focused and streamlined organization, supported by a strong financial position, we are moving forward fully focused on the execution of our clinical strategy. We continue to target a first quarter 2026 start of the Phase 2 clinical trial of KER-065 in patients with Duchenne muscular dystrophy ("DMD"), subject to positive regulatory interaction, and believe Keros is well-positioned to deliver meaningful value to both patients and stockholders."

"Our engagement with the management team and Board has delivered results for all stockholders, including a commitment by Keros to return a portion of the Takeda licensing revenue directly to investors," said Daniel Schneeberger, Founder and Chief Investment Officer at ADAR1 Capital Management. "We appreciate the collaborative dialogue we have had with the Company and believe today’s announcement is a thoughtful and positive step toward enhancing long-term stockholder value."

The negotiation and approval of the repurchase transactions with ADAR1 and Pontifax were overseen by a Capital Return Committee of Keros’ Board of Directors, composed entirely of independent and disinterested directors. The Committee recommended that the full Board approve the repurchase transactions and the subsequent tender offer, which the Board approved.

The Purchase Agreements include certain customary standstill, voting and other provisions. Details of the Purchase Agreements will be filed on a Form 8-K with the U.S. Securities and Exchange Commission (the "SEC").

Goldman Sachs & Co. LLC is serving as Keros’ financial advisor, and Cooley LLP is serving as legal counsel.

(Press release, Keros Therapeutics, OCT 15, 2025, sec.gov/Archives/edgar/data/1664710/000110465925099512/tm2528773d1_ex99-1.htm [SID1234656674])

Aprea Therapeutics Establishes Recommended Phase 2 Dose (RP2D) for ATRN-119, Considering Combination Therapies

On October 15, 2025 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company developing innovative treatments that exploit specific cancer cell vulnerabilities while minimizing damage to healthy cells, reported that it has determined the recommended Phase 2 dose (RP2D) of 1,100 mg once daily for ATRN-119, its oral ATR inhibitor in the monotherapy arm of the ongoing ABOYA-119 Phase 1/2a dose-escalation study, in patients with advanced solid tumors.

ATR program

Building on the completion of dose escalation and supported by new preclinical data suggesting potential synergistic anti-tumor effects, Aprea is considering further ATRN-119 development in combination approaches that could expand its therapeutic potential. The Company believes ATRN-119’s mechanism of action, favorable safety profile, and pharmacologic characteristics make it an ideal candidate for combination with DNA-damaging agents, including radiation therapy, antibody-drug conjugates and immune checkpoint inhibitors.

As part of this strategic focus, Aprea is pausing further enrollment in both once daily and twice daily monotherapy dosing arms of ABOYA-119. Importantly, patients currently being dosed with ATRN-119 as part of this ongoing clinical trial will continue to have access to therapy without interruption.

The Company is currently in discussions with leading academic centers to explore combining ATRN-119 with radiation in patients with HPV+ head and neck cancer, an indication where synergistic anti-tumor effects have been observed in preclinical data. Additional investigator-led studies evaluating ATRN-119 in combination with an I/O agent and antibody-drug conjugates, are also being explored, based on preclinical evidence that ATR inhibition may enhance anti-tumor immune responses.

Phase 1 monotherapy data in the ABOYA-119 dose-escalation study, ATRN-119 demonstrated:

Favorable tolerability profile with manageable adverse events at the RP2D of 1100 mg once daily
Durable disease stabilization in heavily pretreated patients across multiple tumor types
Dose-proportional pharmacokinetics supporting once-daily dosing
Preliminary signs of clinical activity in biomarker-selected populations
"We are very pleased to have identified the recommended monotherapy Phase 2 dose for ATRN-119, which is an important step in our transition to the next stage of development," said Oren Gilad, Ph.D., President and Chief Executive Officer of Aprea. "Based on the growing body of evidence supporting ATR inhibition as a potent sensitizer to DNA-damaging therapies and immunotherapy, we are now considering ATRN-119 in combination approaches that we believe could expand its clinical impact. We believe this candidate’s mechanism, safety profile, and pharmacologic characteristics make it a compelling candidate for pairing with other anti-cancer therapies, including radiation or checkpoint inhibitors, where synergistic anti-tumor effects have been demonstrated preclinically."

A poster titled Updated data from ABOYA-119: A phase 1/2a trial of ATRN-119, a novel macrocyclic ATR inhibitor, in patients with advanced solid tumors harboring DNA damage will be presented at the forthcoming AACR (Free AACR Whitepaper)-NCI-EORTC 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) on Friday, October 24, 2025.

WEE1 Program

Aprea continues to advance its lead program, WEE1 kinase inhibitor APR-1051 at full speed. The ongoing Phase 1, first-in-human study (NCT06260514) is actively enrolling patients at three leading clinical sites in the United States. To date, patients with advanced solid tumors harboring specific cancer-associated gene alterations have been treated with APR-1051 at doses up to 150 mg once daily. Early signals of clinical benefit, including disease stabilization in multiple patients, have been observed, supporting continued dose escalation and further clinical evaluation of APR-1051. The Company expects to report clinical data from this study later this month at the AACR (Free AACR Whitepaper)-NCI-EORTC 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) and is planning to further explore safety, pharmacokinetics, and signals of antitumor activity.

(Press release, Aprea, OCT 15, 2025, View Source [SID1234656672])

Amphista Therapeutics nominates AMX-883, an orally available Targeted Glue™ degrader of BRD9, as its first clinical development candidate for the treatment of acute myeloid leukaemia

On October 15, 2025 Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery of next generation Targeted Protein Degradation (TPD) medicines, reported the nomination of AMX-883; a potent, selective and orally bioavailable degrader of BRD9, as its first clinical development candidate. Amphista is advancing AMX-883 for the treatment of acute myeloid leukaemia (AML).

Leveraging Amphista’s Eclipsys platform and proprietary Targeted Glue technology, AMX-883 induces the degradation of BRD9 via induction of the E3 ligase DCAF16, a completely novel mechanism, distinct from CRBN and VHL-based technologies. AMX-883 is a highly potent and rapid degrader, inducing almost complete degradation of BRD9 within just two hours of treatment, whilst retaining exquisite selectivity over all other bromodomain containing proteins, and beyond, as illustrated by global proteomics. This profile translates into robust in vivo efficacy, including in disseminated patient derived xenograft models. Amphista plans to initiate its first clinical trial with AMX-833 in the second half of 2026.

"Nominating AMX-883 as our first clinical candidate, marking the first time a BRD9 degrader has been developed in AML, is a key milestone for Amphista and the TPD field. Our preclinical data package underlines the fundamental role BRD9 plays in the pathogenesis of AML. AMX-883 has the potential to transform the treatment paradigm for this terrible disease, where 5-year survival rates remain at just 33% and is the cause of death for an estimated 130,000 patients globally each year. As a broad-acting, pro-differentiation agent, AMX-883 has the potential to treat AML in a karyotype-independent way, bringing benefit to a larger patient population than current treatments." commented Martin Pass, Chief Development Officer at Amphista.

The nomination of AMX-883 triggers the drawdown of the third tranche of Amphista’s Series B financing, enabling the continued progression of the Company’s portfolio of next generation Targeted Glue therapeutics towards clinical stage development.

Antony Mattessich, Chief Executive Officer at Amphista, said: "We have built a truly unique discovery platform in Eclipsys, which offers the opportunity to deliver advanced protein degraders with performance characteristics beyond what has been achievable with earlier generation approaches. The nomination of AMX-883 as our first development candidate is a testament to our capabilities and we now look forward to filing an IND application in early 2026."

This news follows an announcement in May 2024 when Amphista unveiled its discovery of a new differentiated mechanism of action for BRD9 degradation. This year, the Company also unveiled a new mechanism of action for TEAD degradation via FBXO22, and for SMARCA2 degradation via induction E3 ligase DCAF16.

About BRD9 and Acute Myeloid Leukaemia

Acute myeloid leukaemia (AML) is one of the most aggressive blood cancers and despite the availability of anti-proliferative treatments, patient survival rates remain alarmingly low. The disease is characterized by a differentiation block which prevents myeloid cell maturation and results in an accumulation of immature cells/AML blasts. Therapies which remove the differentiation block and allow maturation of these AML blasts including ATRA, FLT-3 inhibitors, and most recently Menin inhibitors have demonstrated clinical benefit in several sub-sets of AML. However, there is a pressing need for broader-acting treatments that can benefit patients regardless of their genetic profile.

BRD9 is a subunit of the non-canonical BAF complex where it plays a key structural and functional role, being linked to regulation of chromatin structure and maintaining genomic stability in AML. Degradation of BRD9 releases the differentiation block and leads to the differentiation and death of AML blasts

(Press release, Amphista Therapeutics, OCT 15, 2025, View Source [SID1234656671])

Akari Therapeutics Announces $2.5 Million Registered Direct Offering

On October 15, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX) (the "Company"), an oncology biotechnology company developing novel payload antibody drug conjugates (ADCs), reported that it has entered into a definitive agreement for the issuance and sale of an aggregate of 3,125,000 of the Company’s American Depositary Shares ("ADSs"), each representing 2,000 ordinary shares at a purchase price of $0.80 per ADS in a registered direct offering.

Ladenburg Thalmann & Co. Inc. is acting as the exclusive placement agent for the offering.

In addition, in a concurrent private placement, the Company will issue unregistered Series E Warrants to purchase up to 3,125,000 of the Company’s ADSs and unregistered Series F Warrants to purchase up to 3,125,000 of the Company’s ADSs, each at an exercise price of $0.98 per share. The Series E Warrants will be exercisable on the date of shareholder approval (the "Shareholder Approval Date") for the exercisability of the warrants and have a term of five years from the initial exercise date. The Series F Warrants will be exercisable on the Shareholder Approval Date and have a term of thirty months from the initial exercise date. The offering is expected to close on or about October 16, 2025, subject to the satisfaction of customary closing conditions.

The gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses payable by the Company, are expected to be approximately $2.5 million. The Company intends to use the net proceeds for working capital, general corporate purposes, and continued research and development ("R&D"). Specifically with regard to its focused R&D work, the Company intends to use these funds to generate differentiated data on the novel ADC payload that highlights its unique action against cancer and builds on new data being presented at the upcoming Society for Immunotherapy Cancer Society Annual Meeting in early November.

The ADSs (but not the warrants issued in the private placement or the ADSs underlying such warrants) are being offered by the Company pursuant to a "shelf" registration statement on Form S-3 (File No. 333-289056) originally filed with the U.S. Securities and Exchange Commission (the "SEC") on July 29, 2025 and declared effective by the SEC on July 31, 2025. The ADSs to be issued in the registered direct offering are being offered only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and the accompanying base prospectus relating to, and describing the terms of, the registered direct offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the final prospectus supplement and the accompanying base prospectus relating to the registered direct offering, when available, may also be obtained by contacting Ladenburg Thalmann & Co. Inc., 640 Fifth Avenue, 4th Floor, New York, NY 10019, by phone at (212) 409-2000, or by email at [email protected].

The warrants described above are being issued in a concurrent private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), and Regulation D promulgated thereunder and, along with the ADSs underlying the warrants, have not been registered under the Securities Act, or applicable state securities laws. Accordingly, the warrants and underlying ADSs may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

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

(Press release, Akari Therapeutics, OCT 15, 2025, View Source [SID1234656670])