On July 8, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that the European Commission, based on a positive opinion issued by the European Medicines Agency (EMA), has granted Orphan Drug Designation (ODD) for SLS009, a novel, and highly selective CDK9 inhibitor, for the treatment of acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, JUL 8, 2024, View Source [SID1234644717]).

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"We are thrilled to receive ODD from the EMA for the treatment of AML. This designation along with the recently announced strong preliminary Phase 2 data and previous FDA ODD designation reinforces our continued progress and commitment to developing SLS009 as a potential treatment for AML," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "We look forward to working closely with the EMA and the FDA to advance SLS009 clinical development and ultimately deliver it to the patients who need it most. To that end, we remain on track to share further data around SLS009 in the third quarter of this year."

Orphan drug designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medical Association (EMA) Committee for Orphan Medicinal Products. The EMA’s orphan designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the EU. Medicines that meet the EMA’s orphan designation criteria qualify for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase, and access to centralized marketing authorization. The treatment must also provide significant benefit to those affected by the condition.

"We are excited to receive this designation from the EMA to complement the earlier FDA’s ODD. The ongoing clinical trial data continues to support significant benefit in patients with AML relapsed after or refractory to venetoclax regimens and the EMA recognizes the significant benefit of SLS009 for patients impacted by AML. Together with EMA’s Protocol Assistance will define a path to an eventual regulatory approval in the European Union and working with the FDA towards a potential approval in the US," said Andrew Elnatan, Vice President of Regulatory Affairs at SELLAS.

The Phase 2a clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with aza/ven at two dose levels, 45 and 60 mg. In the 60 mg dose cohort patients were randomized into either a 60 mg dose once per week or a 30 mg dose two times per week. The target response rate at the optimal dose level is 20% with a target median survival over 3 months. ASXL1 mutation has been identified as the most promising target mutation based on biology of the mutation and the SLS009 mechanism of action that has been confirmed by the clinical results to date. The trial continues enrollment in two cohorts, both enrolling patients with myelodysplasia-related mutations, one with ASXL1 mutations and the other with myelodysplasia-related mutations other than ASXL1. For more information on the study, visit clinicaltrial.gov identifier NCT04588922.

On July 8, 2024 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing targeted therapies for patients with RAS-addicted cancers, reported that it will host an investor webcast to provide an update on its RMC-6236 pancreatic ductal adenocarcinoma (PDAC) clinical development program (Press release, Revolution Medicines, JUL 8, 2024, View Source [SID1234644716]). Speakers will include members of Revolution Medicines’ management team, along with Brian M. Wolpin, M.D., M.P.H., professor of medicine at Harvard Medical School, and director of the Gastrointestinal Cancer Center and Robert T. & Judith B. Hale Chair in Pancreatic Cancer at Dana-Farber Cancer Institute.

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The webcast will take place at 8:00 a.m. Eastern Time on Monday, July 15, 2024. To access the live webcast, please visit the "Events & Presentations" page of Revolution Medicines’ website at View Source Additionally, a replay of the webcast will be available on the "Events & Presentations" page of the Revolution Medicines website for at least 14 days following the event.

On July 8, 2024 PharmaMar (MSE:PHM) reported to have received a notification from the European Commission (EC) informing the Company of its decision to revoke the refusal to grant Marketing Authorization for Aplidin in Multiple Myeloma (Press release, PharmaMar, JUL 8, 2024, View Source [SID1234644715]).

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According to the communication received, the EC has re-evaluated the criteria applied for the participation of experts in the administrative procedure for the Marketing Authorization of Aplidin, as well as the relevant EMA rules governing conflicts of interest, so that they can ensure the objective impartiality of these experts.

Therefore, the EC notes that one of the experts of the Scientific Advisory Group (SAG) involved in the development of a rival product, was allowed to participate in the Marketing Authorization procedure for Aplidin, in accordance with the EMA rules applicable at the time.

Consequently, in order to avoid any doubt as to the objective impartiality of the assessment of the application, the Commission has decided it is appropriate to revoke the decision to refuse Marketing Authorization for Aplidin.

It is also reported that the Commission has forwarded to the EMA the opinions of the Committee for Medicinal Products for Human Use (CHMP), to request the re-evaluation of the application from the time of the onset of the detected procedural irregularity.

The European Commission’s reversal of the decision, which is totally exceptional, is a de facto acknowledgement that PharmaMar did not have all the necessary guarantees in the evaluation process for Aplidin. Now that the registration dossier has been returned to the EMA, the Company will ensure that the procedure is conducted with absolute impartiality and on a level playing field.

History of the lawsuit, 7 years of litigation

PharmaMar filed a lawsuit in October 2018 before the General Court of the European Union against the EC, seeking the annulment of the Commission’s Implementing Decision, by which it denied Marketing Authorization for Aplidin as a treatment for patients with Multiple Myeloma.

The reason for the lawsuit related to the strict conflict-of-interest checks carried out by the experts appointed by the EMA and the correct analysis of the scientific evidence presented by PharmaMar.

In October 2020, the General Court of the European Union upheld PharmaMar’s claim in full, at the extreme end of the conflict of interest, annulling the European Commission’s decision to refuse Marketing Authorization for Aplidin for the treatment of patients with Multiple Myeloma, and ordered the Commission to pay the costs.

In 2021, Estonia and Germany appealed the decision to the EU Court of Justice, although the EC decided not to do it, which could be understood as implicitly accepting the ruling.

In 2023, the Court of Justice of the European Union annulled the judgment of the General Court and referred the case back to the General Court, to rule again on the first ground for annulment urged by PharmaMar in its initial application, and to rule, if it considered it necessary, on the other claims for annulment in its action. That is, to rule not only on the conflict of interest and the breach of the Principle of Objective Impartiality by the EMA, but also on the breach of the Principle of Good Administration, the breach of the Principle of Equal Treatment and incorrect analysis of the scientific evidence presented by PharmaMar, the breach of the obligation to state reasons and the breach of the rights of defense.

The Company has always maintained that, during the evaluation process of its drug, Aplidin for the treatment of Multiple Myeloma, there was a conflict of interest of several members based on numerous objective elements, including the cooperation of one of its members with a Swedish company, XNK Therapeutics AB, developing a rival drug, as well as its participation in the development of other competing drugs.

Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) reported that it has entered into a definitive agreement to acquire APEIRON Biologics AG, which holds royalty rights to QARZIBA (dinutuximab beta) for the treatment of high-risk neuroblastoma, for $100 million in cash (Press release, Ligand, JUL 8, 2024, View Source [SID1234644714]). In addition, Ligand will pay APEIRON shareholders additional consideration based on future commercial and regulatory events, including up to $28 million if QARZIBA royalties exceed certain predetermined thresholds by either 2030 or 2034, respectively.

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APEIRON is a private biopharmaceutical company based in Vienna, Austria. The company co-developed QARZIBA for the treatment of high-risk neuroblastoma in patients aged 12 months and above. QARZIBA was approved by the European Medicines Agency in 2017 and is commercially available today in more than 35 countries. APEIRON receives an undisclosed royalty on net sales of QARZIBA outside of mainland China from Recordati S.p.A, a leading global pharmaceutical company with a presence in over 150 countries and sales of more than $2.2 billion,2 and on net sales of QARZIBA within mainland China from BeiGene, Ltd.

"The addition of QARZIBA to our commercial royalty portfolio further supports our growth strategy to invest in high-value medicines that deliver significant clinical value and generate predictable and long-term revenue streams for our investors," said Todd Davis, CEO of Ligand. "QARZIBA is the only immunotherapy for high-risk neuroblastoma marketed across Europe and in other parts of the world. We believe this drug will be a meaningful contributor to our royalty revenue which is now driven by a diversified portfolio of 12 key commercial-stage products."

Peter Llewellyn-Davies, CEO of APEIRON commented, "This transaction is an important milestone for our company and shareholders. We have spent more than 20 years translating academic research into therapeutic products for diseases with high unmet needs. Our team was honored to help bring QARZIBA to the young patients who need it. We appreciate that Ligand recognizes the long-term potential of this critical drug for a rare pediatric cancer."

Transaction Terms
Under the terms of the agreement, which has been unanimously approved by both the Board of Directors at Ligand and APEIRON’s Supervisory Board, Ligand will acquire all the outstanding shares of APEIRON for $100 million in cash at closing. Ligand will also pay APEIRON shareholders additional consideration based on future commercial and regulatory events, including up to $28 million if QARZIBA royalties exceed certain predetermined thresholds by either 2030 or 2034, respectively. The transaction is subject to a 30-day shareholder objection period and other customary closing conditions and is expected to close in July 2024.

Concurrently, Ligand is also entering into a stock purchase agreement whereby it has committed to investing up to $4 million in invIOs Holding AG, a privately held spin-off of APEIRON. The proceeds will help finance the research and development of three innovative early-stage immuno-oncology assets. APEIRON is entitled to royalties and milestone payments on these assets which will further expand Ligand’s development stage portfolio. This transaction is expected to close in July 2024.

Financial Guidance Update
The APEIRON acquisition will be immediately accretive to Ligand’s earnings per share (EPS) by approximately $1.00 on an annualized basis. Ligand is increasing its 2024 revenue guidance to be in the range of $140 million to $157 million (previously $130 million to $142 million) and is raising core adjusted EPS guidance to $5.00 to $5.50 (previously $4.25 to $4.75). Royalties are now expected to range from $100 million to $105 million (previously $90 million to $95 million). Guidance for sales of Captisol is unchanged at $25 million to $27 million and contract revenue is now expected to range from $15 million to $25 million (previously $15 million to $20 million).

McDermott Will & Emery and E+H Rechtsanwälte GmbH served as Ligand’s legal counsel. Baker McKenzie and DORDA served as APEIRON’s legal counsel.

About QARZIBA
QARZIBA is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Medicines Agency in 2017 for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease.

Dinutuximab beta was originally discovered by EMD Lexigen Research Center and ultimately developed by the Children’s Cancer Research Center (CCRI) and European Neuroblastoma Research Network (SIOPEN) for the treatment of high-risk neuroblastoma. APEIRON in-licensed dinutuximab beta from CCRI and SIOPEN in 2011, and upon completing the clinical development, out-licensed the exclusive global commercialization rights to EUSA Pharma (UK) Limited in 2016. QARZIBA is marketed outside of mainland China by the global pharmaceutical company Recordati S.p.A., which acquired EUSA Pharma (UK) Limited in 2022.

On July 8, 2024 Kineta, Inc. (Nasdaq: KA), a clinical-stage biotechnology company focused on the development of novel immunotherapies in oncology that address cancer immune resistance, reported that it has entered into an exclusivity and right of first offer agreement (the "Agreement") with TuHURA Biosciences, Inc. ("TuHURA"), a Phase 3 registration-stage immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy (Press release, Kineta, JUL 8, 2024, View Source;utm_medium=rss&utm_campaign=kineta-announces-exclusivity-and-right-of-first-offer-agreement-for-its-vista-blocking-antibody-with-tuhura-biosciences [SID1234644713]). Pursuant to the Agreement, among other things, Kineta has granted TuHURA an exclusive right to acquire Kineta’s worldwide patents, patent rights, patent applications, product and development program assets, technical and business information, and other rights and assets associated with and derived from its development program related to KVA12123, the Company’s VISTA blocking immunotherapy. This exclusive right shall continue through the first to occur of (a) the execution of any definitive agreement with respect to a potential transaction by TuHURA or one or more of its affiliates and (b) 11:59 PM Eastern Time on October 1, 2024, subject to extension. In consideration for Kineta’s compliance with its obligations set forth in the Agreement, TuHURA will pay Kineta a $5 million nonrefundable payment.

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KVA12123 is a novel VISTA blocking monoclonal antibody being evaluated in a Phase 1/Phase 2 clinical trial for patients with advanced solid tumors. The study includes a monotherapy arm with KVA12123 alone, and a combination arm utilizing KVA12123 together with Merck’s anti-PD1 therapy, KEYTRUDA (pembrolizumab). Initial results from this study were reported in April this year at the American Association of Cancer Research. To date, the drug has been well tolerated with no dose limiting toxicities and no cytokine release syndrome. Additional data is expected to be released in the fourth quarter of 2024.

"TuHURA Biosciences is well positioned to advance KVA12123," said Craig W. Philips, President of Kineta. "TuHURA is a Phase 3 registration stage immuno-oncology company with expertise and deep experience in the field. We believe they will make an excellent partner for this program and in advancing this novel drug program which could provide an important new treatment option for cancer patients."