On June 19, 2019 CARsgen Therapeutics, a clinical-stage company committed to developing Chimeric Antigen Receptor T cell therapies for cancer, reported that one of its leading drug candidates, CT053 fully human BCMA (B-Cell Maturation Antigen)-CAR-T cell for the treatment of patients suffering from relapsed/refractory multiple myeloma (rrMM), has received Investigational New Drug (IND) clearance from the United States Food and Drug Administration (FDA) (Press release, Carsgen Therapeutics, JUN 19, 2019, View Source [SID1234537186]). CT053 has also received IND clearance from the National Medical Products Administration in China four months ago and is the subject of an ongoing phase I clinical trial.

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"At the 5th Annual Immunotherapy in Myeloma Scientific Workshop in Denver, Colorado, CARsgen provided an update of the clinical data of CT053 and showed that as of Feb. 28, 2019, 87.5% of the patients showed overall response to the treatment. 70.8% of the patients had complete response and no grade 3 or higher cytokine release syndrome (CRS) was observed in 24 heavily pre-treated patients with rrMM. The IND clearance of CT053 by the U.S. FDA is of great significance to patients" said Dr. Zonghai Li, founder, CEO and CSO of CARsgen. "According to JAMA Oncology, in 2016, there were about 130,000 cases of myeloma, which means from 1990 to 2016, incident cases of myeloma increased by 126% globally [1] and despite the development of novel therapies, multiple myeloma remains incurable and new treatment options are needed. Our goal is to continue the development of novel, safe and effective immunotherapies. This is our long-standing commitment to cancer patients worldwide."

On June 19, 2019 Nouscom, an immuno-oncology company developing off-the-shelf and personalized cancer neoantigen vaccines, reported the publication of a paper in the online edition of the peer-reviewed journal, Nature Communications, entitled: Adenoviral vaccine targeting multiple neoantigens as strategy to eradicate large tumors combined with checkpoint blockade (D’Alise et al, reference below) (Press release, NousCom, JUN 19, 2019, View Source [SID1234537185]). The authors describe results from preclinical studies investigating the immunological potency and efficacy of a vaccine based on an adenovirus vector derived from non-human Great Apes (GAd) that encodes multiple neoantigens.

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The study demonstrated that a single administration of Nouscom’s GAd vaccine can eradicate large tumors in mouse models when combined with anti-PD1 or anti-PD-L1 treatment and triples the efficacy of such checkpoint inhibitors in established tumors. Nouscom’s GAd vaccine accommodates multiple neoantigens, up to 31 in these studies, and demonstrates strong and broad CD8+ and CD4+ neoantigen-specific T cell responses following vaccination. The results highlight the potential of these viral vector vaccines for development as personalized cancer vaccines.

Dr. Elisa Scarselli, Chief Scientific Officer and Co-Founder of Nouscom, said: "We are very encouraged by these preclinical results. These exciting data confirm our hypothesis that our GAd vaccine synergizes with checkpoint inhibitors. It also establishes that anti-tumor efficacy positively correlates with the breadth and potency of T cell responses induced by vaccination, including the expansion of the intratumoral T cell repertoire. Our findings have a potential translational impact, suggesting that the changes in intratumoral T cell repertoire induced by the vaccination might be an indicator of treatment efficacy."

Dr. Scarselli added, "These findings provide compelling preclinical evidence that a vaccine encoding multiple neoantigens in combination with anti-PD1 or anti-PD-L1 may be an efficacious approach for the personalized treatment of cancers. We look forward to evaluating our approach with NOUS-PEV, a personalized cancer vaccine, in human trials, which we expect to start in 2020."

References

A.M. D’Alise et al. Adenoviral vaccine targeting multiple neoantigens as strategy to eradicate large tumors combined with checkpoint blockade, Nature Communications 2019.

Online publication: View Source

On June 19, 2019 Medicenna Therapeutics Corp. ("Medicenna" or the "Company") (TSX: MDNA;OTCQB: MDNAF), a clinical stage immuno-oncology company, is reported that Dr. Fahar Merchant, Chairman, President and CEO will present an overview of the Company at the Raymond James Life Sciences and MedTech Conference today, Wednesday, June 19th at 8:00 AM ET at the Lotte New York Palace hotel in New York City (Press release, Medicenna Therapeutics, JUN 19, 2019, View Source [SID1234537184]). Dr. Merchant will also be available to meet with investors on a one-on-one basis during the conference.

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This presentation will be made available with a live webcast and may be accessed on the Events & Presentations page by visiting Medicenna’s website at View Source This webcast will be available for 7 days following the presentation.

On June 19, 2019 Kymera Therapeutics Inc., a biotechnology company pioneering targeted protein degradation to create breakthrough medicines for patients, reported that it will present new preclinical data demonstrating its first-in-class oral IRAK4 protein degraders cause tumor regression in MYD88-mutant B cell lymphoma (Press release, Kymera Therapeutics, JUN 19, 2019, View Source [SID1234537183]). Data will be shared during an oral presentation at the 15th International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland on Thursday, June 20 at 5:45 PM CEST.

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"We are highly encouraged by the data, which strongly supports clinical advancement of our orally active IRAK4 protein degraders in MYD88-driven B cell malignancies, both as monotherapy and in combination with drugs targeting complementary pathways," said Jared Gollob, MD, CMO, Kymera Therapeutics. "We look forward to sharing our findings and engaging with an international group of lymphoma experts at this year’s ICML meeting to define the path forward into the clinic in 2020."

Activating mutations in MYD88 are frequent across multiple subsets of aggressive B cell lymphomas, including activated B cell-like diffuse large B cell lymphoma (ABC-DLBCL, 30-40%), primary central nervous system lymphoma (30-70%), and primary extranodal lymphoma (45-75%), as well as Waldenström Macroglobulinemia (>90%). Myddosome signaling triggered by MYD88 is dependent on both the kinase activity and scaffolding function of IRAK4. Kymera is using a chemical knockdown strategy to develop heterobifunctional small molecule IRAK4 protein degraders such as KYM-001 for the treatment of B cell malignancies driven by MYD88 mutations.

ICML Study Highlights
ABSTRACT #083: "KYM-001, a first-in-class oral IRAK4 protein degrader, induces tumor regression in xenograft models of MYD88-mutant ABC-DLBCL alone and in combination with BTK inhibition"

Presented by Duncan Walker, PhD, VP of Oncology, Kymera Therapeutics

Kymera’s IRAK4 degrader achieved potent and selective E3 ligase-dependent degradation of IRAK4 in both MYD88 mutant and MYD88 wild-type (WT) ABC-DLBCL cell lines.
Cell viability was impacted in MYD88 mutant but not WT ABC-DLBCL cell lines, whereas an IRAK4 kinase inhibitor had no effect.
Orally dosed KYM-001 caused in vivo tumor regression in a dual MYD88/CD79 mutant OCI-LY10 mouse xenograft model of ABC-DLBCL associated with ≥75% IRAK4 knockdown in tumors.
Subtherapeutic doses of KYM-001 in combination with the BTK inhibitor ibrutinib increased OCI-LY10 apoptosis in vitro and drove tumor regression in vivo in the OCI-LY10 mouse xenograft model.

On June 19, 2019 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company studying topsalysin (PRX302), a first-in-class, pore-forming protein, in late-stage clinical trials for the treatment of patients with urological diseases, reported that it has received formal scientific advice from the European Medicines Agency (EMA) regarding a proposed design of a Phase 3 clinical trial to evaluate the potential of topsalysin as a targeted focal therapy to treat patients with intermediate risk localized prostate cancer (Press release, Sophiris Bio, JUN 19, 2019, View Source [SID1234537182]).

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"Based on the safety profile of topsalysin in 451 patients in our clinical development program along with the efficacy seen in our Phase 2 studies in localized prostate cancer, we approached the EMA with our proposed study design for a single Phase 3 trial to support registration in Europe, and we are pleased to have now obtained formal feedback from the Agency," said Randall Woods, president and CEO of Sophiris. "We believe that data from a single Phase 3 trial, if successful, will be sufficient to support market approval in Europe."

The Phase 3 study design, agreed upon by the EMA, will enroll patients with a confirmed diagnosis of intermediate risk disease. Approximately 700 men who meet the eligibility criteria will be equally randomized to receive a single administration of either topsalysin or placebo. The primary endpoint for the study will be the proportion of patients at 12 months who have failed treatment, defined as histological progression of disease resulting in the need for alternative intervention, per an independent central adjudication panel.

Webcast scheduled for today at 9:30 a.m. Eastern Time

The Sophiris management team will host a conference call and webcast today, June 19, at 9:30 a.m. Eastern Time to review the key details of the proposed Phase 3 clinical trial design and to address the potential commercial opportunity for topsalysin, along with Professor Hashim Ahmed, Faculty of Medicine Department of Surgery & Cancer, Chair in Urology, Imperial College of London & Imperial College Healthcare NHS Trust and a member of the Scientific Advisory Board at Sophiris.

A live audio webcast will be accessible on the "Investor Relations" page of the Sophiris corporate website at www.sophirisbio.com. A replay will be available at the same location.

About Localized Prostate Cancer

Prostate cancer is the second most common form of cancer in men in the United States with an estimated 175,000 new cases in 2019. Approximately 77 percent of patients in the United States are diagnosed with localized disease. Research has shown that patients with early, localized disease have a low likelihood of the cancer spreading beyond the confines of the prostate; however, many men with clinically-significant localized disease choose to undergo radical treatment. Radical therapies include surgery to remove the entire prostate and/or radiation. Potential toxicities from radical treatments can be significant and permanent and include erectile dysfunction, urinary incontinence and rectal toxicity.

About Topsalysin

Topsalysin (PRX302), an innovative, "First-in-Class" transmembrane pore-forming protein, was genetically modified to be activated only by enzymatically-active PSA, which is produced in large quantities within the prostate of men with prostate cancer. The targeted focal treatment of prostate cancer is in line with current treatment trends for solid tumors such as breast and liver, where the goal is to remove the tumor and preserve as much of the organ and organ function as possible.

Topsalysin has the potential to provide a targeted focal therapy for the ablation of localized prostate cancer lesions while potentially avoiding many of the complications and side effects associated with whole gland radical treatments. The increasing use of multiparametric magnetic resonance imaging (mpMRI) and advances in software to co-register previously obtained mpMRI images with real-time three-dimensional ultrasound images enables urologists to more accurately locate tumors within the prostate when taking biopsies. This increases the accuracy with which men with clinically significant lesions are identified. It also enables the injection of an ablative agent, such as topsalysin, directly into previously identified clinically significant tumors located within the prostate.