10-Q – Quarterly report [Sections 13 or 15(d)]

Exicure has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Exicure, 2017, SEP 12, 2017, View Source [SID1234521881]).

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Aurigene, a Wholly Owned Subsidiary Of Dr Reddy’s, And Curis Announce CA-170 Program Update Following Data Presented At ESMO 2017

On September 12th, 2017 Aurigene Discovery reported plans to initiate a Phase 2 trial of CA-170, a PDL1-VISTA inhibitor to be conducted at sites in India (Press release, Aurigene Discovery, SEP 12, 2017, View Source [SID1234624587]). This was announced following the presentation of preliminary data from the initial 34 patients with cancer treated in the dose escalation stage of the Phase 1 trial of CA-170 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress by Aurigene’s collaborator and licensee of CA-170, Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development and commercialization of innovative and effective therapeutics for the treatment of cancer. The trial has been conducted in the U.S., South Korea and Spain. The Phase 2 trial is the result of the initial safety data and preliminary evidence of clinical benefit observed in the trial.

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CA-170 is an oral small molecule targeting the immune checkpoints PDL1 and VISTA. Data presented at the ESMO (Free ESMO Whitepaper) 2017 conference represent the initial 34 patients treated to date in the dose escalation Phase 1 trial. 30 patients were naïve to prior immunotherapy treatment, while four patients had experienced prior treatment with approved anti-checkpoint antibodies. No dose limiting toxicities were observed at doses ranging from 50 mg to 800 mg once daily dosing examined thus far. CA-170 demonstrated good oral bioavailability and plasma drug levels were shown to increase in a near-linear manner with increasing doses. Evidence of immune modulation, including an increase in activated CD8+ T cells, was observed in patient blood and tumor biopsy samples examined following treatment. Of the 21 patients evaluable for disease assessment, 13 patients experienced disease stabilization. Four immunotherapy treatment-naïve patients treated with CA-170 experienced shrinkage of their tumors. Six patients remained on drug treatment beyond three months, including all four patients with tumor shrinkages. In addition, seven of the 34 patients remain on study and are continuing with treatment.

"These results are consistent with the observations made in the preclinical setting and further affirm CA-170’s mechanism of action as an oral small molecule checkpoint inhibitor. Based on these initial clinical results, we are excited for the opportunity to expand testing of CA-170, possibly in earlier lines of treatment and in a greater number of immunotherapy treatment-naïve cancer patients," commented Mr. CSN Murthy, Chief Executive Officer of Aurigene. "Together with Curis, we have designed a Phase 2 trial, treating selected populations of patients of interest in the CA-170 program to be treated at major cancer centers in India. Aurigene’s decision to sponsor and fund this trial is further affirmation of our commitment to CA-170 and a reflection of the successful collaboration we have with Curis in multiple development programs. Aurigene has the commercial rights to the program in India and Russia in addition to milestones, royalties other commercial supply rights globally."

"We are pleased with these early results. Evidence of tumor shrinkage and multiple patients remaining on drug treatment for extended periods, along with signals for biomarkers of immune modulation in patient blood and tumor samples, tells us the program continues to move in the right direction. We plan to continue with the dose escalation and continued analysis of patient biopsy samples in the Phase 1 trial," said Ali Fattaey, Ph.D., President and Chief Executive Officer of Curis. "We expect to provide additional updates at upcoming conferences including the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November."

"The ability for cancer patients to administer a potential checkpoint inhibitor on their own as a once daily oral drug is a significant and unique opportunity in our field," added Adil Daud, M.D., investigator in the CA-170 Phase 1 trial and director of Melanoma Clinical Research at the UCSF Helen Diller Family Comprehensive Cancer Center. "These initial clinical results are encouraging and merit continued development."

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.

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.

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.