On December 19, 2018 Medigene AG (FWB: MDG1, Prime Standard, SDAX) reported interim clinical results from its ongoing company-sponsored Phase I/II clinical trial of a dendritic cell (DC) vaccine in 20 patients with acute myeloid leukemia (AML) (Press release, MediGene, DEC 19, 2018, View Source [SID1234532188]). The primary objectives of the study are the safety and feasibility of this active immunotherapy with the patient-derived DCs produced according to Medigene’s proprietary technology.

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The topline data released today is from a period of one year of vaccination of all patients and represents an interim dataset after half of the treatment period.

A total of 20 subjects (median age 59, range 24 to 73) with AML (risk groups good, intermediate, poor: 13, 5, 2), in morphologic complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) after induction or consolidation therapy, not eligible for allogeneic hematopoietic stem cell transplantation, were enrolled into this safety and feasibility Phase I/II trial, vaccinated and followed up for 12 months. Subjects in this trial had AML that was positive for Wilms Tumor-1 (WT-1) antigen with or without positivity for Preferentially Expressed Antigen in Melanoma (PRAME). Vaccination with dendritic cells, presenting the antigens WT-1 and PRAME, was carried out monthly, with a higher frequency within the first 6 weeks. AML diagnoses had been established with a median of 9.8 months before the first vaccination (range 4.5 to 17.5 months), and the last chemotherapy infusion had been performed at a median of 6.9 months (range 2 to 14.8 months).

The vaccinations were well tolerated with no serious adverse events (SAEs) related to the treatment. The most common adverse events (AEs) were injection site related, accounting for 35% of all AEs and mild in nature (Grade 1). The feasibility of the manufacture of the dendritic cell vaccine in these chemotherapy-pretreated subjects was already presented earlier this year (abstract AACR (Free AACR Whitepaper)). Link to poster: View Source

After a 12-months treatment period, the overall survival was 89% (18 of 20 patients, 95% confidence interval: 61 to 97%) and the progression free survival was 60% (12 of 20 patients, 95% confidence interval: 36 to 78%). Most relapses, 5 out of 8, occurred within the first 80 days after the start of the vaccination, out of which the 2 deaths were in patients with relapses on days 45 and 64, which could point to a starting relapse upon entering the study.

The completion of the ongoing trial is scheduled for the end of 2019 following a two-year treatment period.

Dr. Yngvar Floisand, Head Physician of the Department of Hematology at the Oslo University Hospital and Principal Investigator of the trial, comments: "The interim results after one year of treatment demonstrate an excellent safety profile and a very good manufacturability of Medigene’s personalized DC vaccines. The initial data on the efficacy of the therapy point in the right direction, but we have to complete the two-year trial period for a conclusive evaluation."

Dr. Kai Pinkernell, CMO and CDO of Medigene AG, comments: "We are pleased about these first encouraging interim results from our ongoing safety and feasibility trial with our DC vaccine. AML is a serious disease with poor long-term prognosis and we are committed to developing new treatment options for those patients in urgent need."

Medigene intends to present the detailed data from the interim analysis at a scientific conference in the near future.

About acute myeloid leukaemia (AML): Acute myeloid leukaemia is a malignant disease of the hematopoietic system, affecting mainly adults above 60 years of age. In Germany, about 3,600 cases are registered annually.

AML is caused by uncontrolled growth of dysfunctional hematopoietic precursor cells in the bone marrow. These cells prevent the generation of normal blood cells, causing a decrease in erythrocytes and platelets, for example. Typical symptoms of AML include anemia, fever, increased risk of infection, and bleeding. AML progresses rapidly and may be fatal within a few weeks or months, if untreated.

AML treatment is often started with intensive chemotherapy, followed by consolidation with or without allogeneic hematopoietic stem cell transplantation. Unfortunately, a significant proportion of patients suffer a relapse of the original disease. Depending on the biologic risk profile of the disease, age and co-morbidity the long-term survival is highly variable.

About Medigene’s DC vaccines: In addition to Medigene’s development focus on T cell-receptor modified T cells (TCR-Ts), the Company has developed a new generation of antigen-tailored dendritic cell (DC) vaccines.

Dendritic cells (DC) can take up antigens, process them and present peptides on their surface in a form that can induce antigen-specific T cells to mature and proliferate. In this way, T cells recognize and eliminate tumor cells which bear the same antigen peptide on their surface. Dendritic cells can also induce natural killer cells (NK cells) to attack tumor cells. The scientific team of Medigene has developed new, fast and efficient methods for generating autologous (patient-specific) mature dendritic cells which have the relevant characteristics to generate very strong T cell and NK cell immune responses. The dendritic cells can be loaded with various tumor antigens to treat different forms of cancer. Since an immune response builds up over the total time of administration of the DC vaccine, this form of therapy is particularly designed for patients who suffer from a tumor disease which has been reduced to such an extent by chemotherapy that the prevention of the recurrence of the tumor disease is the main goal

On December 18, 2018 CTI BioPharma Corp. (NASDAQ:CTIC) reported that it has received input from the U.S. Food and Drug Administration (FDA) at a recent Type C meeting on key elements of the design of a new randomized Phase 3 study of pacritinib in adult patients with myelofibrosis (primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis) and who have severe thrombocytopenia (as defined by patients with platelet counts of less than 50,000 per microliter), an indication that has been recognized by the medical community as an important unmet medical need (Press release, CTI BioPharma, DEC 18, 2018, View Source [SID1234532119]).

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The planned Phase 3 study is designed to evaluate the effects of pacritinib as compared to physician’s choice of treatment. The primary efficacy endpoint will be the proportion of patients achieving a greater than or equal to 35% spleen volume reduction (SVR) between baseline and Week 24. Secondary efficacy endpoints of the study include total symptom score reduction and overall survival.

Before commencing the Phase 3 study, CTI plans to meet with the FDA to discuss the final optimal dose analysis from the PAC203 Phase 2 study. To expedite the transition to Phase 3, CTI intends to amend the PAC203 protocol to include a Phase 3 component. The PAC203 Phase 3 component is designed to enroll approximately 200 patients with enrollment expected to commence in the third quarter of 2019. The anticipated cost of the Phase 3 study is approximately $25 million. Taking into account the impact of recently-announced cost saving efforts and the anticipated cost of the new Phase 3 trial, the current CTI financial analysis projects a cash runway that extends into 2020.

On December 18, 2018 Erasca, a company dedicated to advancing exceptional scientific approaches to erase cancer, reported that it has completed a $42 million Series A financing round co-led by founding investors City Hill Ventures and Cormorant Asset Management, joined by additional institutional and individual investors (Press release, Erasca, DEC 18, 2018, View Source [SID1234534176]). Proceeds of the financing will support development of a new generation of oncology drugs intended to not just treat, but actually cure cancer.

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"Cancer is a pervasive disease that has impacted nearly everyone I know in some way. Individuals continue to be seriously afflicted and in need of healing beyond what can be provided by available treatments," said Jonathan E. Lim, M.D., Erasca’s executive chairman and co-founder. "Erasca was borne out of a mission to address this significant patient need and, one day, erase cancer. We recognize that this is an ambitious goal, as cancer is a formidable foe. But no mission worth taking is ever easy."

Erasca has multiple discovery programs underway for undisclosed targets that are biological drivers of cancer. The company anticipates it will disclose more about these programs as it moves into human clinical studies. Erasca is also pursuing additional opportunities for pipeline expansion through academic and biopharmaceutical collaborations.

"Founded on exciting new discoveries around key molecular drivers of cancer, and having assembled a team of executives with a legacy of multiple prior successes, Erasca is the company that stands an excellent chance of deftly overcoming multiple obstacles along this exciting, yet long, journey of bringing a breakthrough medicine to patients," said Bihua Chen, Portfolio Manager, Cormorant Asset Management. "Tackling a mission this large requires a combination of passion for the patient and strong business acumen. Jonathan’s background as a physician and biotech company leader who has led multiple successful companies, along with the outstanding team he has assembled, position Erasca well in executing on its mission."

Since 2003, the serial venturepreneur has been a leader and founding investor of four other biotechnology startups that delivered novel therapies for the benefit of patients globally, raised a combined $1 billion in capital and generated more than $4 billion of shareholder value. Dr. Lim’s most recent operational role was as chairman, president, CEO and co-founder of Ignyta, acquired by Roche in February 2018 for $1.7 billion. He has co-founded, funded and led several other successful ventures, including Bonti (acquired by Allergan), Eclipse Therapeutics (acquired by Bionomics) and Halozyme Therapeutics. He also is dedicated to building world-class, mission-driven ventures as managing partner for City Hill Ventures, an impact-minded investment firm he founded in December 2010, and as Venture Partner at ARCH Venture Partners, one of the largest seed and early stage technology venture firms in the U.S. Dr. Lim’s full bio can be accessed here.

Joining Dr. Lim on Erasca’s founding leadership team are business and finance veteran, Gary Yeung, CFA, who serves as the company’s Chief Business Officer, and highly regarded computational biologist, Robert Shoemaker, Ph.D., who is Vice President of Biology.

On December 18, 2018 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO), a clinical-stage biotechnology company specializing in the development of innovative drugs in oncology targeting tumor DNA Damage Response (DDR) to fight resistant cancers, reported having been notified by the European Patent Office (EPO) of its intent to grant the Company a new patent (EP3325623) covering the combination of AsiDNA, Onxeo’s first-in-class agonist of the DNA Damage Response (DDR) , with any PARP inhibitor (PARPi), in all countries of the European Union (EU) (Press release, Onxeo, DEC 18, 2018, View Source [SID1234532120]).

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This new patent protects the intellectual property of Onxeo relating to combined preparations comprising conjugated nucleic acid molecules, including its lead product candidate AsiDNA and a PARP inhibitor, as well as the use of the combined preparations for the treatment of cancer. Under the patent, AsiDNA and the PARP inhibitor may be present in one combined dosage form, or as part of separate dosage forms for sequential administration of the respective drugs. This European patent has a term expiring in mid-2036.

Onxeo has conducted extensive preclinical studies of AsiDNA in combination with various PARPi. These studies show that the combination has a strong synergistic anti-tumor activity in solid tumors, regardless of the genetic mutation status of the tumor. This synergy appears to be a class effect with all PARPi. Combination with AsiDNA could therefore represent an opportunity to expand PARPi indications to HR proficient tumors, which account for approximately 70% of tumors.

"This patent covering the combination of AsiDNA with any PARP inhibitor further reinforces our extensive patent portfolio, which is a key component of the value of AsiDNA. Our strong intellectual property position is an integral part of the Company’s business model, which aims to progress disruptive compounds up to the most valuable clinical inflexion points and then to partner or license them," said Judith Greciet, Chief Executive Officer of Onxeo.

On December 18, 2018 Immedica is reported that from 1 September 2018, it has become the exclusive distributor for the innovative anti-cancer drug Yondelis in the United Kingdom (Press release, Immedica Pharma, DEC 18, 2018, View Source [SID1234555253]). Yondelis is an established treatment for patients suffering from soft tissue sarcomas (STS) and platinum sensitive ovarian cancer. "I believe Immedica will fill an important role as a facilitator for making Yondelis available for patients in the United Kingdom", says Anders Edvell, CEO of Immedica.

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About YONDELIS (trabectedin)
YONDELIS (trabectedin) is a multimodal, synthetically produced antitumor agent, originally derived from the sea squirt, Ecteinascidia turbinata. The drug exerts its activity by targeting the transcriptional machinery and impairing DNA repair. It is approved in close to 80 countries in North America, Europe, South America and Asia for the treatment of advanced soft tissue sarcomas as a single-agent and for relapsed ovarian cancer in combination with DOXIL/CAELYX (doxorubicin HCl liposome injection) in the European Union. Under a licensing agreement with PharmaMar, Janssen Products, L.P. has the rights to develop and sell YONDELIS globally except in Europe, where PharmaMar holds the rights, and in Japan, where PharmaMar has granted a license to Taiho Pharmaceuticals.