On August 18, 2022 Oncopeptides, a biotech company focused on research and development of therapies for difficult-to-treat hematological diseases, reported that the European Commission has granted Pepaxti[] (melphalan flufenamide, also called melflufen) marketing authorization in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy (Press release, Oncopeptides, AUG 18, 2022, View Source [SID1234646791]). For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation. The marketing authorization is valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Lichtenstein, and Norway.
The marketing authorization is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study as confirmatory study. Oncopeptides intends to submit a type II variation in Q4 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).

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"The approval of Pepaxti in Europe is foundational for Oncopeptides, and brings excellent news for patients and shareholders," says Jakob Lindberg, CEO Oncopeptides AB. "Despite the introduction of novel therapies, patients with triple class refractory disease have a high unmet medical need, since their treatment options ultimately become exhausted."

Oncopeptides will now advance market access activities to pave the way for a successful launch of Pepaxti in Germany in Q4, 2022. The Company is dedicated to making the drug available for patients across Europe and is actively considering various options to commercialize the product.

For further information, please contact:

Rolf Gulliksen, Global Head of Corporate Communications, Oncopeptides AB (publ)
E-post: [email protected]
Mobil: + 46 70 262 96 28

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person above, on August 18, 2022, at 13:00 (CET).

About HORIZON study

The HORIZON study is a pivotal phase 2 study, that evaluated melflufen in combination with dexamethasone, in heavily pre-treated patients with poor prognosis. This multi-center single arm study evaluated 157 patients with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The efficacy results for triple-class refractory patients who have received at least 3 prior lines of therapies and who had no ASCT or progressed more than 36 months after an ASCT in the HORIZON study is outlined below:

Response (n=52) HORIZON study (assessed by investigator)
Overall response rate (ORR), 95% CI (%) 28.8% (17.1%, 43.1%)
Duration of response (DOR) 95% CI (months) 7.6 (3.0-12.3)
Time to response (TTR) (months) 2.3 (1.0-10.5)
About Pepaxti

Pepaxti (melphalan flufenamide, also called melflufen) is a lipophilic peptide conjugated alkylating drug that rapidly and selectively is delivering cytotoxic agents into tumor cells. The drug is composed of a di-peptide and an alkylating moiety. The lipophilicity allows a faster cellular uptake whereas the peptide hydrolysis mediated by aminopeptidases, results in accumulation of alkylating moieties in cancer cells. This results in an improved efficacy without an increased toxicity compared to melphalan. Pepaxti inhibits proliferation and induces apoptosis of hematopoietic and solid tumor cells. It shows synergistic cytotoxicity in combination with dexamethasone in melphalan resistant and non-resistant multiple myeloma cell lines.

Pepaxti is indicated in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapies. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

About Multiple Myeloma

Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cells which produce antibodies to help fight infection, and cause cancer cells to accumulate in the bone marrow. Multiple Myeloma is the second most common hematologic malignancy, and accounts for approximately 1-2% of all new cancer cases, with a global incidence rate of 1.7 per 100,000 and an age-standardized incidence rate of 2.1-3.4 per 100,000 in France, Germany, Italy, Spain, and the UK. An estimated 35,842 patients were diagnosed in the EU27 during 2020, with an estimated 23,275 deaths due to the disease (ECIS 2020).

Patients with multiple myeloma may have symptom-free periods, but the disease always relapses, and patients may become refractory to all available treatment options due to mutations and/or clonal evolution of the tumor cells. A growing subset of patients are triple-class refractory, and develop disease refractory to immunomodulatory drugs, proteasome inhibitors, and CD38- targeting monoclonal antibodies. These patients have a very short expected overall survival.

On August 16, 2022 Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA therapies (oRNA), reported the initial closing of its $221 million Series B financing (Press release, OrnaTherapeutics, AUG 18, 2022, View Source [SID1234626173]). At signing, the company received approximately $121 million and expects to receive the remaining $100 million subject to customary closing conditions (including regulatory approval under the Hart-Scott-Rodino (HSR) Act). Merck participated as a new investor in the financing alongside commitments from founding investors MPM Capital and BioImpact Capital, an affiliate of MPM, among others. Orna was created in 2019 by MPM Capital and BioImpact Capital, with funding from the UBS Oncology Impact Fund.

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The financing will enable Orna to continue development of its circular RNA + LNP (lipid nanoparticle) delivery platform, as augmented by its joint venture with ReNAgade Therapeutics, an RNA delivery company created by MPM Capital and BioImpact Capital, to advance its lead in situ CAR (isCAR) program toward the clinic and build manufacturing capabilities to support development of an expanding preclinical and clinical pipeline.

"In under three years, Orna has taken proprietary circular RNA from academic literature to the first proof of concept data in preclinical models," said Tom Barnes, Ph.D., Chief Executive Officer of Orna and Entrepreneur at BioImpact Capital. "Investors continue to recognize our premier technology and the hard work of our team, and we are pleased to see such unwavering belief in the potential of circular RNA to revolutionize how we treat disease."

Orna is expanding the reach of current RNA therapeutics with recently presented preclinical data demonstrating the potential of oRNA to treat cancer, genetic disorders, and infectious diseases. Orna’s lead isCAR program demonstrated tumor suppression and eradication in animal models, indicating significant potential that oRNA-based cancer therapies could be superior to traditional cell therapies. With the proceeds of the Series B financing, Orna anticipates advancing this program into clinical trials in 2024.

"In just a few years since we created and built the company, Orna has made remarkable progress in demonstrating the extensive power of its circular RNA + LNP delivery technologies," said Ansbert Gadicke, M.D., Chairman of Orna, Managing Partner of BioImpact Capital and MPM Managing Director. "oRNA, with its numerous advantages over linear mRNA, is the next generation of RNA therapies, and we are excited to see the continued development and applications of this technology."

"It has been validating to see the promise of RNA realized through linear mRNA-based vaccines," said Daniel G. Anderson, Ph.D., Professor of Chemical Engineering and core member of the Koch Institute for Integrative Cancer Research and the Institute for Medical Engineering and Science at MIT, and Co-founder of Orna. "Orna’s next generation of oRNA therapies not only demonstrate promise as vaccines, but also as treatments for a range of diseases where other modalities have fallen short."

SVB Securities acted as financial advisor to Orna on this transaction.

On August 18 , 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported that received a transparency notification from FMR LLC (Press release, Galapagos, AUG 18 , 2022, View Source [SID1234618525]).

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Pursuant to Belgian transparency legislation1, Galapagos received a transparency notification on 15 August 2022 from FMR LLC, who notified that it holds 3,740,842 of Galapagos’ voting rights, consisting of 3,740,842 ordinary shares. FMR LLC controls investment funds FIAM Holdings LLC, Fidelity Management & Research Company LLC, Fidelity Management Trust Company and Strategic Advisers LLC, of which Fidelity Management & Research Company LLC increased its position to 3,303,128 voting rights, and no longer holds any equivalent financial instruments, and Strategic Advisers LLC decreased its position to 10,082 voting rights. FMR LLC’s holding of 3,740,842 Galapagos’ voting rights represents 5.69% of Galapagos’ currently outstanding 65,728,511 shares. FMR LLC thus remains above the 5% threshold of Galapagos’ voting rights considering the change in the nature of holdings. The full transparency notice is available on the Galapagos website.

On August 18, 2022 POINT Biopharma Global Inc. (NASDAQ: PNT) (the "Company" or "POINT"), a company accelerating the discovery, development, and global access to life-changing radiopharmaceuticals, reported the company will be publishing an abstract at ESMO (Free ESMO Whitepaper) Congress 2022, which takes place in Paris, France on September 9-13, 2022 (Press release, Point Biopharma, AUG 18, 2022, View Source [SID1234618513]).

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The abstract is titled "Efficacy and Safety of 177Lu-PNT2002 Prostate-Specific Membrane Antigen (PSMA) Therapy in Metastatic Castration Resistant Prostate Cancer (mCRPC): Initial Results from SPLASH" (Abstract #1400P) and focuses on efficacy and safety data from the 27-patient safety and dosimetry lead-in cohort for the Company’s phase 3 SPLASH trial (NCT04647526) evaluating PNT2002 for the treatment of metastatic castration-resistant prostate cancer (mCRPC).

In advance of the publication of the abstract, the Company also hosted a 45-minute educational webinar entitled "Understanding the PNT2002 SPLASH Trial Control Arm", which provided information regarding:

The SPLASH trial design (including lead-in phase) and rationale for hormone switches
Control arm benchmarks, and why PROfound1 & IMbassador2502 were selected as the SPLASH trial benchmarks
Current treatment patterns, including sequencing considerations, in real-world clinical settings for mCRPC patients
The webinar featured presentations from Dr. Oliver Sartor, MD, Professor of Medicine, Medical Director, Tulane Cancer Center, University of Tulane School of Medicine, and Dr. Kim Chi, MD, Vice President and Chief Medical Officer, British Columbia Cancer Agency.

Drs. Sartor and Chi were joined by the Company’s executive leadership team including Dr. Sherin Al-Safadi, VP Medical Affairs.

A replay of the webinar is now available online at View Source

The phase 3 SPLASH trial is a multi-center, randomized, open label assessment of PNT2002 in participants with PSMA-expressing mCRPC who have progressed on androgen receptor pathway inhibitor (ARPI) therapy and refuse, or are not eligible for, chemotherapy. The randomization phase of the study is expected to enroll approximately 400 participants across North America, Europe, and the United Kingdom. Participants will be randomized 2:1 with participants in arm A receiving PNT2002 and participants in arm B receiving either abiraterone or enzalutamide. Participants in arm B who experience centrally assessed radiographic progression and meet protocol eligibility will have the option to crossover and receive PNT2002. Patients will be subject to follow-up for up to 5 years from their first PNT2002 dose. The primary endpoint of the study is radiographic progression-free survival. Key secondary endpoints include overall response rate, overall survival, and pharmacokinetics.

On August 18, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that a case report, titled "Long Term Complete Response of Advanced Hepatocellular Carcinoma to Glypican-3 Specific Chimeric Antigen Receptor T-Cells plus Sorafenib, A case report", has been published in Frontiers in Immunology (https://www.frontiersin.org/articles/10.3389/fimmu.2022.963031/full) (Press release, Carsgen Therapeutics, AUG 18, 2022, View Source [SID1234618497]).

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Hepatocellular carcinoma (HCC) is the most common histologic subtype of primary liver cancer, which is the sixth most common cancer type worldwide. Clinical efficacies of existing therapies for unresectable HCC are still unsatisfactory. CAR T-cell therapy has been approved for a variety of hematological tumors, but there are still great challenges for CAR T-cell therapies to treat solid tumors. We firstly reported GPC3 as a reasonable target for CAR T-cell therapy and thereafter advanced it into clinic.[1,2] In order to further enhance the efficacy of GPC3 CAR T cells, we proposed a new strategy by combining the GPC3 CAR T cells with sorafenib for the treatment of hepatocellular carcinoma[3]. To further validate this strategy in clinical setting, we conducted an investigator-initiated clinical trial at the First Affiliated Hospital of Wenzhou Medical University. The published case reported a patient with advanced HCC who achieved a complete response (CR) and a long survival period after the combination therapy of CAR-GPC3 T-cell plus sorafenib.

The case showed a 60-year-old Asian male patient with hepatitis B virus (HBV)-related HCC who underwent surgery in May 2018. In August 2018, the recurrence of liver cancer and pulmonary metastasis occurred after the operation, and then he received transarterial chemoembolization (TACE) to treat liver lesions and interventional ablation to treat pulmonary metastases. Two months later, he progressed and was enrolled into the clinical trial. After the enrollment, the patient underwent leukapheresis for CAR-GPC3 T-cell manufacturing. Seven days after leukapheresis, the patient started to receive 400 mg of sorafenib twice daily. The patient received 4 cycles of CAR-GPC3 T cells (CT011) treatment and each cycle was divided into two infusions. Prior to each cycle of CT011 treatment, lymphodepletion was performed. A total of 4×109 CAR-GPC3 T cells were infused.

The CT011 plus sorafenib combination therapy was well tolerated. This patient obtained partial responses (PR) from the 3rd month and achieved CR in the 12th month after the first cycle of CT011 infusion. The tumor had no progression for more than 36 months and maintained the CR status for more than 24 months after the first infusion.

To the best of our knowledge, this is the first reported case with a CR after the combination therapy of CAR T cells with tyrosine kinase inhibitors. The clinical outcome demonstrated that the combination therapy of GPC3 CAR T-cell and Sorafenib may be a new promising approach for GPC3+ advanced HCC patients.

Dr. Zonghai Li, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, commented that, "There is great expectation for CAR T cells to provide curative potential in treating solid tumors. When enrolled into the clinical trial, the patient in this reported case had undergone local therapies such as TACE and interventional ablation but had not received systemic therapies such as anti-angiogenesis inhibitors. Based on the finding of our earlier preclinical research, we adopted the combination therapy of sorafenib and CT011 as treatment regimens. It was very encouraging to see that the patient achieved a complete response and a long survival period without recurrence for more than two years. While directly indicating that GPC3 CAR T may be used for early-line treatment of HCC, this case report also provides new evidence supporting the adoption of CAR T cells in the early-line treatment of other solid tumors."

About CT011

CT011 is an autologous CAR T-cell product candidate with proof-of-concept clinical data for the treatment of hepatocellular carcinoma (HCC) and has the potential to be the first-in-class globally. Dr. Zonghai Li — Founder, Chairman of the Board, Chief Executive Officer and Chief Scientific Officer of CARsgen Therapeutics — led the world’s first successful effort in identifying, validating, and reporting GPC3 as a tumor-associated target for the development of CAR T-cell therapies to treat HCC. CARsgen has completed enrollment of a Phase I trial in China.