On August 19, 2022 AskGene Pharma Inc. (AskGene) reported the appointment of Barbara Hickingbottom, J.D., M.D. as Chief Medical Officer and Matt Hsu, Ph.D. as VP of Preclinical and Clinical Pharmacology (Press release, AskGene Pharmaceuticals, AUG 19, 2022, View Source [SID1234618501]). Dr. Hickingbottom will be responsible for leading the global clinical development of AskGene, including pipeline strategy, clinical development plans and operations, product registration, and guiding the global clinical collaborations with existing and potential partners. Dr. Hsu will be responsible for leading global preclinical and clinical pharmacology activities.

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Jian-Feng (Jeff) Lu, Ph.D., CEO of AskGene commented: "We are pleased to welcome Dr. Hickingbottom and Dr. Hsu to our management team earlier this year. They will each bring us over two decades of global industry experience, which will help AskGene to continue to optimize our clinical development and global registration strategies for our lead biological products and to progress our promising pipeline of novel drugs positioned to address major areas of unmet clinical needs. We believe the appointment will bring strong energy and leadership to AskGene for our global clinical development and facilitate regulatory approval of lead innovative cytokine projects and other innovative molecules."

Barbara Hickingbottom J.D., M.D., CMO of AskGene commented: "I am profoundly excited to bring my experience to AskGene pipeline and join AskGene at this important time as ASKG315 is entering into the clinical stage in Australia. The early preclinical data for ASKG315 and ASKG915 are very promising, and I look forward to working alongside AskGene’s highly skilled scientific teams to bring the whole AskGene portfolio to patients globally."

Dr. Matt Hsu, VP of Preclinical and Clinical Pharmacology commented: "AskGene’s innovative SmartKine platform with a prodrug approach for cytokine therapies has demonstrated promising anti-tumor activities in preclinical species. With strong efficacy and reduced toxicity, this technology has a potential to overcome the limitation of cytokine use clinically and revolutionize immunotherapies. Therefore, I am looking forward to working with the company’s leadership team to advance novel and highly innovative clinical projects and candidates in the areas of oncology and immunological diseases, and to build a robust pipeline that has the opportunity to transform care for many more patients in need around the world."

Before joining AskGene, Dr. Hickingbottom served as the Head of Clinical Development at Xencor, Inc. (Xencor). During her time at Xencor, she was deeply involved in the clinical development of 12 anti-tumor biological innovative drugs in Xencor’s pipeline and responsible for all of the company’s clinical-stage products. Prior to Xencor, Dr. Hickingbottom worked as both a consultant and a scientist in many biopharmaceutical companies, including BioMarin, Mannkind, Novartis, Xoma, Novacea, and CTL Immunotherapies. Dr. Hickingbottom is an accomplished clinical pathologist with over 25 years of life sciences experience in clinical development, medical affairs, and registration strategy. She received her Medicine Doctor degree from the Tufts University School of Medicine and completed her internship and residency in pathology at Stanford. She also received a Juris Doctor degree from the Harvard Law School.

Dr. Hsu has more than two decades of experiences in drug development, ranging from proof of concept to approval. Previously, Dr. Hsu was Senior Principal Scientist in the Clinical Pharmacology Modeling & Simulation group at Amgen, where he led the Technical and Professional Unit for clinical study data and report preparation, conducted clinical pharmacology studies in translational sciences, applied PK/PD knowledge for dose selection at IND and End-of-Phase 2 stages, and prepared NDA/BLA submission for drug approvals. Prior to Amgen, Dr. Hsu worked in Preclinical Development at Johnson & Johnson in New Jersey and received his Ph.D. in Pharmaceutical Sciences from the University of Michigan. He has published more than 35 peer-reviewed papers.

On August 19, 2022, Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported that it has now received approvals from authorities and ethical committees in Denmark and Germany for the next parts of the ongoing CORIST phase II-trial (Press release, Scandion Oncology, AUG 19, 2022, View Source,c3615707 [SID1234618499]). The trial studies Scandion’s lead compound SCO-101 as a combination treatment in patients with metastatic colorectal cancer (mCRC). Thus, the development of SCO-101 can continue as planned and communicated in connection with Scandion’s recently completed rights issue.

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Part 1 of the CORIST trial has been completed and part 2 is currently ongoing with topline data expected to be communicated during the third quarter of 2022. To ensure a seamless continuation of the trial, parts 3 and 4 have been planned, and part 3 is ready to start independently of part 2. Patient recruitment in part 3 is expected to commence during the third quarter of 2022, and topline results from CORIST part 3 are expected most likely within the third quarter of 2023.

CORIST part 3 represents an expansion of the development program, in order to best exploit SCO-101’s potential in mCRC. As described in connection with the rights issue the expansion is expected to approximately double the commercial potential of SCO-101 in this indication.

The trial is expanded by adding a new schedule for combining SCO-101 and chemotherapy (FOLFIRI), which will be evaluated in patients with both RAS wild-type and RAS mutated tumors. The expansion was done through an amendment of the ongoing trial, making it possible to initiate the expansion while finalizing part 2, thereby keeping the momentum in the trial and utilizing already activated trial sites.

"We are pleased to have received formal approvals so swiftly and thereby be able to advance the CORIST trial as soon as practically possible. The expansion of the trial will greatly enhance both the medical and commercial potential of SCO-101 and we are delighted to be able to invest in maximizing the value of this compound to the benefit of patients, physicians and Scandion", says Bo Rode Hansen, President & CEO of Scandion.

The CORIST trial is also carried out in Spain. Parts 3 and 4 of the trial are expected to recruit patients here as well as in Denmark and Germany pending one outstanding approval in Spain.

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.