ONXEO GRANTS EXCLUSIVE WORLDWIDE LICENSE OF VALIDIVE® DEVELOPED FOR THE TREATMENT OF ORAL SEVERE MUCOSITIS TO MONOPAR THERAPEUTICS

On September 13, 2017 Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen: ONXEO), ("Onxeo" or the "Company"), a clinical-stage biotechnology company specializing in the development of innovative drugs for the treatment of orphan diseases, in particular in oncology, reported the Company has granted a global exclusive license of its product Validive (clonidine mucoadhesive buccal tablet) developed for the treatment of severe oral mucositis induced by radiotherapy or chemotherapy in patients suffering from head and neck cancer to Monopar Therapeutics (Chicago, Illinois, USA), a biopharmaceutical company focused on developing innovative drug combinations to improve clinical outcomes in advanced cancer (Press release, Onxeo, SEP 13, 2017, View Source [SID1234570545]).

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Following the phase II trial, the company had announced that it would not initiate the next clinical steps on its own for this asset but would actively look for an industrial partner to further its development.

Under the agreement, Monopar Therapeutics Inc. receives an exclusive worldwide license to develop, register, commercialize and manufacture Validive. Monopar Therapeutics will drive and fund all remaining development and regulatory activities, the first of these activities being a phase III registration study.

"Severe oral mucositis occurs in the majority of patients treated with radiotherapy/chemotherapy for head and neck cancer but there is no effective prevention or treatment available to date," said Chandler D. Robinson, MD, MBA, MSc, CEO of Monopar Therapeutics Inc. "We are pleased with this agreement and strongly believe in the potential of Validive to answer this large unmet need. The acquisition of a phase III-ready asset is fully aligned with our strategy to build a strong and diversified portfolio of oncology products that will improve clinical outcomes in patients with advanced cancer."

Onxeo is entitled to an immediate $1.0m license fee and to future milestone payments that could reach up to $108m subject to the achievement of the agreed upon milestones, including $15.5m related to regulatory milestones, from phase III to registration. Escalating royalties on sales up to a 2-digit percentage are also part of the agreement.

" It was our stated intention after its successful Phase II to partner Validive prior to initiating any remaining development steps, as it was the best strategy to maximize its value for Onxeo," said Judith Greciet, CEO of Onxeo. "This licensing transaction with Monopar Therapeutics is fully in line with this strategy and further demonstrates our capacity to execute such value-creating deals. We are pleased that Validive will get the opportunity to one day serve the unmet needs of many patients affected with severe oral mucositis, while, on our end, we are focusing all our energy on our pipeline of unique breakthrough compounds in orphan oncology."

Alligator Bioscience signs immunotherapy research collaboration agreement for 4-1BB

On September 13, 2017 Alligator Bioscience (Nasdaq Stockholm: ATORX), a biotechnology company developing antibody-based pharmaceuticals for tumor-directed immunotherapy, reported that a research collaboration agreement has been signed with Professor Ignacio Melero, MD, PhD, Center for Applied Medical Research (CIMA) and Clínica Universidad de Navarra, Spain, to further investigate the biology of 4-1BB (CD137) as a target in cancer immunotherapy (Press release, Alligator Bioscience, SEP 13, 2017, View Source [SID1234538686]).

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Alligator has two pipeline programs targeting 4-1BB, the fully owned monospecific antibody ATOR-1017, and the bispecific antibody ALG.APV-527, co-developed with Aptevo therapeutics.

Under the collaboration agreement, the team led by Professor Melero primarily will investigate the biological effects of 4-1BB activation in various pre-clinical cancer immunotherapy models. Professor Melero has acted as a scientific advisor and collaboration partner for Alligator since 2014.

Professor Melero, principal investigator at the Immunology and Immunotherapy Program of CIMA and codirector of the Immunology Department of Clínica Universidad de Navarra, has contributed extensively to the understanding of the anti-tumor properties of 4-1BB antibodies in cancer. His landmark studies have opened the field of 4-1BB-targeted immunotherapy, and this is now generally considered to be one of the most promising approaches within immuno-oncology.

4-1BB belongs to the so-called TNF receptor superfamily and plays a critical role in immune responses and immunological memory to cancer. CIMA is the University of Navarra’s biomedical research institute. Its mission is to carry out translational research to a high standard of excellence, based on novel biological knowledge and aimed at finding therapeutic solutions to unsolved medical’s needs.

"I am very enthusiastic about the extended collaboration with Alligator. Its 4-1BB drug candidates provide great new treatment opportunities within immuno-oncology", said Professor Ignacio Melero. "The fact that 4-1BB is upregulated on tumor-specific T cells, together with its capacity to promote survival, expansion and functional activity of several immune cells involved in tumor eradication, makes 4-1BB a uniquely appealing target for immunotherapy of cancer."

"We are very fortunate to have Professor Melero as a research collaborator", said Per Norlén, CEO of Alligator Bioscience. "He is a world leading expert in the field of 4-1BB and immuno-therapy and his scientific guidance will be extremely valuable both for our pipeline projects, and to help fulfill our ambition to deliver first and best-in-class products to patients."

For further information:
Cecilia Hofvander, Director Investor Relations & Communications
Phone +46 46 286 44 95
E-mail: [email protected]

This information is information that Alligator Bioscience AB (publ) 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 set out above, at 08:30 a.m. CEST on 13 September 2017.

Polaris Announces Results of a Phase 2 Trial of ADI‑PEG 20 in Relapsed/Refractory or Poor-risk Acute Myeloid Leukemia

On September 12, 2017 Polaris Group reported that its lead therapeutic ADI‑PEG 20 (pegylated arginine deiminase) demonstrated a good safety profile and an efficacy signal as monotherapy in a phase 2 trial in relapsed/refractory or poor-risk acute myeloid leukemia (AML) patients, as reported in the journal Scientific Reports (Press release, Polaris Pharmaceuticals, SEP 12, 2017, View Source [SID1234526282]). The study was conducted at multiple sites in Taiwan and at MD Anderson Cancer Center in the US.

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Among the 21 evaluable patients enrolled in this study, two patients had complete response to the treatment, with response duration of 7.5 and 8.8 months. Seven patients had stable disease in response to the treatment. The treatment appeared to be safe and well tolerated.

"Based on the encouraging efficacy signal from this ADI‑PEG 20 monotherapy study, we have initiated a phase 1 trial combining ADI‑PEG 20 with low-dose cytarabine in poor-risk AML patients," said John Bomalaski, M.D., Executive Vice President, Medical Affairs at Polaris Pharmaceuticals, Inc.

About ADI‑PEG 20

ADI‑PEG 20 is a biologic being developed by Polaris Group to treat cancers carrying a major metabolic defect that renders them unable to internally synthesize arginine. Because arginine is essential for protein synthesis and survival of cells, these cancer cells become dependent upon the external supply of arginine to survive and grow. ADI‑PEG 20 is designed to deplete the external supply of arginine, causing arginine-dependent cancer cells to die while leaving the patient’s normal cells unharmed. Multiple cancers have been reported to have a high degree of arginine-dependency and can potentially be treated with ADI‑PEG 20.

Argos Therapeutics Reports on Interim Analysis of Phase 3 ADAPT Trial Presented at ESMO 2017 Congress

On September 12, 2017 Argos Therapeutics Inc. (NASDAQ:ARGS), an immuno-oncology company focused on the development and commercialization of individualized immunotherapies based on the Arcelis precision immunotherapy technology platform, reported on an update on the interim analysis of data from the ongoing Phase 3 ADAPT clinical trial evaluating Rocapuldencel-T for the treatment of metastatic renal cell carcinoma (mRCC) that was presented on September 11, 2017 by Robert Figlin, MD, Professor and Chairman, Division of Hematology and Oncology at Cedars Sinai Medical Center, and co-principal investigator of the ADAPT trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress in Madrid, Spain (Press release, Argos Therapeutics, SEP 12, 2017, View Source [SID1234520482]).

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A total of 462 patients with previously untreated advanced or metastatic renal cell carcinoma were enrolled in the ADAPT trial and randomized 2:1 between combination treatment with Rocapuldencel-T and sunitinib (combination arm) vs. sunitinib monotherapy (control arm) after undergoing cytoreductive nephrectomy.

As previously reported, as of February 3, 2017, the cut-off date for the most recent interim analysis which was conducted in February 2017, 42.7% of the 307 patients in the combination arm demonstrated an objective response by RECIST criteria, a secondary endpoint in the trial, as compared with 39.4% of the 155 patients in the control arm. Additional data from the trial reported for the first time at the ESMO (Free ESMO Whitepaper) Conference included data related to the duration of tumor response. Patients in the combination treatment arm who demonstrated an objective response had a median duration of response of 8.4 months compared to 6.3 months for patients in the control arm. Additionally, 16% of those patients with an objective response in the combination treatment arm had durable responses lasting at least 30 months compared to 7% of those who had an objective response in the control arm. Also of note, as of the date of the interim analysis, all of the patients in the combination arm who had achieved a duration of response of at least 30 months had maintained those responses through 36 months.

In addition, Dr. Figlin updated immune response data that had been previously reported by the Company, presenting data on 117 patients analyzed for immune response. Samples were collected from patients in the combination arm enrolled at US sites who provided consent for immune monitoring. Of the 117 patients for whom this analysis was completed, 96 (82%) met the criterion for inclusion in the pre-defined subgroup of immune responders, suggesting that Rocapuldencel-T is having its intended effect of stimulating an immune response in the majority of patients. Immune responders are defined as patients who have an increase of more than two standard deviations from the patient-specific baseline in the number of memory T cells (CD8+/CD28+/CD45RA-) at one or more time points. Of note, median overall survival at the time of the February interim analysis had not yet been reached in the subgroup of immune responders (95% CI: 30.1, -). Additionally, consistent with the mechanism of action of Rocapuldencel-T, a statistically significant correlation was observed between the increase from baseline in the number of Rocapuldencel-T-induced memory T cells (CD8+/CD28+/CD45RA-) and overall survival in patients for whom immune response data has been analyzed and who received at least seven doses of Rocapuldencel-T (including both immune responders and non-responders, n=83).

Commenting on the data, Dr. Figlin noted, "Although we did not see a benefit in median overall survival at the February 2017 interim analysis, the data set was relatively immature, with over half of the subjects in both treatment arms still alive. Additionally, for an immunotherapy agent such as Rocapuldencel-T, one might reasonably expect to see a delayed treatment effect, as evidenced by the fact that a statistically significant correlation of the immune response with survival did not emerge until patients had received at least seven doses of Rocapuldencel-T. Thus, median overall survival and other traditional measures of efficacy such as progression-free survival and objective response rate may not be the best endpoints for evaluating the potential benefit of this therapy for patients with limited treatment options, especially in light of the favorable safety and tolerability profile demonstrated to date. Instead, it may be more appropriate to evaluate this agent in light of the potential for a significant "tail-of-the-curve" effect. Thus, my co-principal investigator and I support Argos’ decision to continue the ADAPT trial, and we look forward to reviewing a more mature data set at the next interim analysis, currently planned for the first half of 2018."

As previously reported, the February 2017 interim analysis was conducted by the ADAPT trial’s Independent Data Monitoring Committee (IDMC) after 75% of the originally targeted number of 290 events (deaths) for the analysis of the primary endpoint of overall survival had occurred.

At the time of the analysis, with more than half of the patients still alive in each arm and a median follow-up time of ~20 months, the IDMC concluded that the trial was unlikely to demonstrate a statistically significant improvement in median overall survival in the combination arm and recommended that the trial be discontinued for futility. However, the ADAPT trial principal investigators and Argos considered the data too immature to observe the delayed effects typically associated with immunotherapy and decided to continue the trial pending further review and analysis of the data and discussions with the FDA. In making this determination, Argos considered, among other factors, the degree of maturity of the data set, the mechanism of action of Rocapuldencel-T, which involves the induction of a long-term memory immune response, and the IDMC’s assessment of the safety profile of Rocapuldencel-T. This determination was subsequently further supported by the extended durability of tumor responses in the combination arm, as reported today.

Following the IDMC interim analysis, the Company met with the FDA to discuss the ADAPT trial and the future direction of the Rocapuldencel-T program in April 2017. The FDA agreed with the Company’s decision to continue the ADAPT trial, and further agreed to review a protocol amendment to extend the trial beyond the originally targeted 290 events and a revised statistical analysis plan that the Company plans to submit.

Dr. Figlin’s complete presentation at the ESMO (Free ESMO Whitepaper) 2017 Congress is available for review on Argos’ website at www.argostherapeutics.com. Argos plans to hold a conference call to discuss Dr. Figlin’s presentation on Wednesday, September 20th at 4:30pm ET and will provide logistical information in a subsequent announcement.

Sandoz proposed biosimilar rituximab accepted for review by the FDA

On September 12, 2017 Sandoz, a Novartis Division, and the pioneer and global leader in biosimilars, reported that the US Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) under the 351 (k) pathway for a proposed biosimilar to the reference medicine, Rituxan** (rituximab) (Press release, Novartis, SEP 12, 2017, View Source [SID1234520494]).

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Rituxan** is used to treat blood cancers including non-Hodgkin’s lymphoma (follicular lymphoma and diffuse large B-cell lymphoma) and chronic lymphocytic leukemia, as well as immunological diseases such as rheumatoid arthritis.

"The cost of treating cancer in the US is a major concern for many patients and their families as well as for the healthcare system[3]" said Mark Levick, MD PhD, Global Head of Development, Biopharmaceuticals. "With the FDA acceptance of our regulatory submission for proposed biosimilar rituximab, we plan to deliver patients a high-quality Sandoz biosimilar that, following approval, could help drive healthcare savings and increase competition, while freeing up resources for and supporting patient access in other areas of cancer care including innovative therapies."

The BLA consists of a comprehensive data package that includes analytical, preclinical and clinical data. Clinical studies included a pharmacokinetic/pharmacodynamic (PK/PD) trial in rheumatoid arthritis (ASSIST-RA)[4], and a Phase III confirmatory safety and efficacy study in follicular lymphoma (ASSIST-FL)[5]. Sandoz believes these data provide confirmation that the proposed biosimilar matches the reference medicine in terms of safety, efficacy and quality.

Sandoz is committed to increasing patient access to high-quality biosimilars. As the pioneer and global leader in biosimilars, Sandoz has five biosimilars marketed worldwide, as well as a leading global pipeline. We plan to launch a total of five major oncology and immunology biosimilars between 2017 and 2020. This includes biosimilar rituximab, which was approved by the European Commission for use in Europe in June 2017 (marketed as Rixathon).

Sandoz is well positioned to continue leading the biosimilars industry based on its experience and capabilities in development, manufacturing and commercialization. As a division of Novartis, the first global healthcare company to establish a leading position in both innovative and off-patent medicines, Sandoz benefits strongly from this unique blend of experience and expertise in many different market environments.

Sandoz also continues to champion policy and legislation that enables patients and the healthcare system to benefit from biosimilars. This was demonstrated by the recent US Supreme Court unanimous positive decision related to the Notice of Commercial Marketing (NCM). The Supreme Court ruled that NCM can be provided before FDA approval, accelerating patient access to future US biosimilars by 180 days. The Court also provided additional clarity on how the "patent dance," the process by which biosimilar manufacturers may provide confidential and proprietary information to the manufacturer of the reference medicine in the patent exchange process, will function.