On November 20, 2019 Sierra Oncology, Inc. (Nasdaq: SRRA), a late-stage drug development company focused on the development and commercialization of momelotinib, a JAK1, JAK2 & ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis, reported that it has launched the MOMENTUM clinical trial for patients with myelofibrosis (Press release, Sierra Oncology, NOV 20, 2019, View Source [SID1234551535]). The randomized double-blind global Phase 3 trial is designed to confirm the efficacy of momelotinib on myelofibrosis symptoms, transfusion independence and splenomegaly, as compared to danazol. The trial is targeting enrollment of 180 myelofibrosis patients who are symptomatic, anemic and have been treated previously with a JAK inhibitor.

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"I am pleased to act as Chief Investigator for this important global trial for the myelofibrosis patient community," said Dr. Srdan Verstovsek, MD, PhD, Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center, Houston, Texas. "JAK inhibitors remain the cornerstone of myelofibrosis treatment but new options in this class are needed for the majority of patients who have difficulty tolerating the currently approved agents due to the cytopenias they can exacerbate or induce. Momelotinib could become a suitable alternative for many patients previously treated with a JAK inhibitor due to its ability to positively address all three hallmarks of myelofibrosis – symptoms, anemia and an enlarged spleen. Critically, momelotinib has consistently demonstrated positive anemia benefits in its prior clinical trials. This anemia benefit is biologically-driven, via potent inhibition of the ACVR1/hepcidin axis, a mechanism that is unique in the JAK inhibitor class."

"The launch of the MOMENTUM trial is a key milestone in our global registration strategy for momelotinib, aimed at bringing this important and differentiated therapy to patients with myelofibrosis," said Dr. Barbara Klencke, Chief Development Officer of Sierra Oncology. "MOMENTUM is designed to confirm the array of benefits observed in prior Phase 3 studies, where momelotinib demonstrated a unique ability to improve anemia and reduce transfusion dependency in patients with myelofibrosis, while also providing clinically comparable benefits on constitutional symptoms and enlarged spleens to other JAK inhibitors. To support our proposed study timeline, global clinical trial sites are anticipated to be activated over the coming months with top-line data from MOMENTUM anticipated in the fourth quarter of 2021."

"The launch of MOMENTUM is a major step in advancing Sierra’s vision of becoming a commercial company. Momelotinib is a promising late stage asset that targets a sizable unaddressed global market with a clear path forward to registration. Importantly, momelotinib’s clinical potential has been previously demonstrated in two large, completed Phase 3 clinical trials, which informed the design of MOMENTUM," said Dr. Nick Glover, President and CEO of Sierra Oncology. "As recently announced, Sierra has secured capital that we expect to be sufficient to execute our development strategy into the second half of 2022, providing us with the financial runway to advance momelotinib towards its anticipated commercialization. Moreover, we also recently announced that we had amended our Asset Purchase Agreement (the "Amendment") with Gilead Sciences, Inc. ("Gilead"). Pursuant to the terms of the Amendment, and subject to certain conditions, Gilead will become a stockholder in Sierra in exchange for substantive reductions in the annual royalty rates payable by Sierra to Gilead upon commercialization of momelotinib. This Amendment will meaningfully benefit Sierra’s stockholders should momelotinib prove commercially successful."

About Momelotinib
Momelotinib is a potent, selective and orally-bioavailable JAK1, JAK2 & ACVR1 inhibitor with a differentiated therapeutic profile in myelofibrosis encompassing robust constitutional symptom improvements, a range of meaningful anemia benefits, including eliminating or reducing the need for frequent blood transfusions, and comparable spleen control to ruxolitinib. More than 1,200 subjects have received momelotinib since clinical studies began in 2009, including more than 800 patients treated for myelofibrosis.

Chronic, progressive anemia is a key hallmark of myelofibrosis and transfusion dependence is the most important negative prognostic indicator of reduced survival in this disease. Approximately 60% of patients are anemic and 45% are transfusion dependent within one year of diagnosis, with most patients ultimately progressing to transfusion dependency. Unfortunately, currently approved JAK inhibitor therapies can induce or worsen anemia, exacerbating this significant unmet medical need in anemic myelofibrosis patients.

The marked systemic inflammation seen in myelofibrosis leads to increased ACVR1 activity which in turn increases secretion of hepcidin, resulting in perturbed iron homeostasis and an iron-restricted anemia. Momelotinib’s inhibition of ACVR1 in addition to JAK1 and JAK2, unique amongst the JAK inhibitor class, results in notable reductions of both hepcidin and inflammation, restoring iron homeostasis and RBC production, thereby alleviating anemia and transfusion dependency.

The SIMPLIFY-1 trial was a double-blind, active-controlled Phase 3 study in which 432 patients received randomized treatment with momelotinib or ruxolitinib for 24 weeks (JCO. 2017;35:3844–50). In addition to a significant reduction in splenomegaly and improvements in constitutional symptoms, previously reported analyses of the SIMPLIFY-1 data demonstrated that patients in the momelotinib arm achieved nominal-statistical significance for all anemia endpoints tested, including a higher rate of transfusion independence (p < 0.001) and lower rates of transfusion dependence (p = 0.019) at Week 24, compared to patients on ruxolitinib.

Retrospective analyses of SIMPLIFY-1 transfusion data, to be presented in a poster at ASH (Free ASH Whitepaper) 2019, demonstrate that the odds of momelotinib patients remaining transfusion free are nearly 10-times higher than those for ruxolitinib treated patients.

Momelotinib is an investigational drug that is not approved for any use in any country. The U.S. Food and Drug Administration has granted Fast Track designation to momelotinib for the treatment of patients with intermediate/high-risk myelofibrosis who have previously received a JAK inhibitor.

Momelotinib is wholly owned by Sierra Oncology and is protected by patents anticipated to provide potential exclusivity to 2040 in the United States and Europe (including Patent Term Extension or Supplementary Protection Certificate).

About the MOMENTUM Phase 3 Clinical Trial for Patients with Myelofibrosis:
The MOMENTUM Phase 3 clinical trial is a randomized double-blind trial designed to enroll 180 myelofibrosis patients who are symptomatic and anemic, and who have been treated previously with a JAK inhibitor. Patients will be randomized 2:1 to receive either momelotinib or danazol. Danazol has been selected as an appropriate treatment comparator given its use to ameliorate anemia in myelofibrosis patients, as recommended by NCCN and ESMO (Free ESMO Whitepaper) guidelines. After 24 weeks of treatment, patients on danazol will be allowed to crossover to receive momelotinib.

The Primary Endpoint of the trial is the Total Symptom Score (TSS) response rate of momelotinib compared to danazol at Week 24 (99% power; p-value < 0.05).

Secondary and exploratory endpoints include:

Transfusion Independence (TI) rate at Week 24 (key secondary: >90% powered; p-value < 0.05),
Splenic response rate (SRR) at Week 24 (>90% powered; p-value < 0.05),
Duration of TSS response to Week 48,
Other measures of anemia benefit, including Transfusion Dependence response rate and various measures of cumulative transfusion burden,
Patient Reported Outcome measures of fatigue and physical function.

On November 20, 2019 Kymera Therapeutics Inc., a biotechnology company pioneering targeted protein degradation to discover breakthrough therapies for patients, reported Kymera Co-founder, President and Chief Scientific Officer Nello Mainolfi, PhD, has been promoted to President and Chief Executive Officer effective immediately (Press release, Kymera Therapeutics, NOV 20, 2019, View Source [SID1234551551]). Mainolfi will lead Kymera as the company enters its next stage of growth, advancing a series of novel degrader drugs into clinical development as well as expanding the impact of its innovative Pegasus platform.

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"On behalf of entire Board of Directors, I’m excited to recognize Nello Mainolfi as the next CEO of Kymera Therapeutics, especially at a time of exceptional growth, promising data delivery, and premier partnerships," said Bruce Booth, DPhil, Chairman of the Kymera Board of Directors and partner at Atlas Venture. "Since the inception of Kymera, Nello has demonstrated exemplary leadership and capabilities, and provided strategic direction to advance the development of our powerful new drug discovery engine from target identification into development. With a pipeline of drug candidates advancing into the clinic, we have full confidence that Nello is the best person to successfully position the company and realize the potential of this modality."

Mainolfi started his drug discovery career at the Novartis Institutes for Biomedical Research, leading teams to identify novel potential medicines that have entered clinical development across a series of disease areas. Before co-founding Kymera with Booth in 2016, Mainolfi was an Entrepreneur in Residence at Atlas Venture and had previously led discovery research at cancer metabolism startup Raze Therapeutics.

"I would like to thank the Board of Directors and the entire Kymera team for this unique opportunity," said Mainolfi. "I’ve had the privilege of contributing to the scientific and corporate evolution of Kymera and I look forward to continuing to foster its research & development progress and ambition. I’m excited to help Kymera deliver on our mission of inventing a new class of protein degrader medicines for patients."

On November 20, 2019 Immunomic Therapeutics, Inc., a privately held clinical stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms, reported that Duane Mitchell, MD, Ph.D., a University of Florida professor of neurosurgery and co-director of the Preston A. Wells Jr. Center for Brain Tumor Therapy, and his team will present new clinical data at the 2019 Society for Neurology (SNO) Annual Meeting being held in Phoenix, Arizona Nov. 20-24, 2019 (Press release, Immunomic Therapeutics, NOV 20, 2019, View Source [SID1234551572]).

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The data being presented at the SNO meeting are from a Phase 2 clinical trial (ATTAC-II) evaluating the efficacy of autologous CMV pp65-LAMP RNA pulsed dendritic cell vaccines mixed with GM-CSF and administered during cycles of adjuvant dose-intensified temozolomide (NCT02465268). The results demonstrate that a CMV pp65-LAMP RNA-pulsed DC vaccination was associated with positive immunologic and clinical response in a patient with MGMT unmethylated midline glioblastoma (GBM).

"The new clinical data to be presented demonstrates the potential impact of our UNITE technology platform and will help validate our therapeutic approach utilizing vaccines to treat difficult cancers like glioblastoma," said Dr. Teri Heiland, Chief Scientific Officer of Immunomic Therapeutics, Inc. "We are encouraged by the immunological response shown with this patient and we look forward to Dr. Mitchell’s presentation of these positive findings at the SNO meeting."

SNO posters – presentation details:

Title: "(ATIM-15) Sustained complete radiographic response and prolonged systemic immune activation in a patient with MGMT unmethylated midline glioblastoma receiving CMV pp65-LAMP RNA-pulsed dendritic cell vaccines"

Category: Adult Clinical Trials – Immunologic

Date and Time: Saturday, Nov 23rd, 5:00 PM – 7:00 PM

Location: Ballroom Lawn, JW Marriott Desert Ridge, Phoenix, Arizona

Title: "(ATIM-34) Single-cell RNA sequencing reveals dynamic immune response changes in glioblastoma patient with durable complete response to CMV pp65-LAMP RNA-pulsed dendritic vaccines
Category: Adult Clinical Trials – Immunologic

Date and Time: Friday, Nov 22nd, 7:30 PM – 9:30 PM

Location: Ballroom Lawn, JW Marriott Desert Ridge, Phoenix, Arizona

The abstracts for University of Florida’s posters at SNO can be found on the following meeting site: View Source and also have been published online in the journal "Neuro-Oncology" by Oxford Academic and can be found here: View Source

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On November 20, 2019 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported the pricing of its previously announced underwritten public offering (Press release, Checkpoint Therapeutics, NOV 20, 2019, View Source [SID1234551519]). Checkpoint is offering 13,400,000 shares of its common stock, par value $0.0001 per share, at a price to the public of $1.27 per share. In connection with the offering, Checkpoint has also granted the underwriters a 45-day option to purchase up to an additional 2,010,000 shares of common stock offered in the public offering, at the same public offering price per share, less underwriting discounts and commissions.

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National Securities Corporation, a wholly owned subsidiary of National Holdings Corporation (NasdaqCM: NHLD), is acting as sole book-running manager for the offering and H.C. Wainwright & Co., LLC is acting as lead manager for the offering. Lake Street Capital Markets, LLC is acting as financial advisor to Checkpoint.

The Company expects to receive gross proceeds from the offering of $17,018,000, before deducting underwriting discounts and commissions and other offering-related expenses. Assuming the full exercise of the underwriter’s option to purchase additional shares, gross proceeds would be $19,570,700.

Checkpoint intends to use the net proceeds from the offering primarily to support the continued development of cosibelimab, including an ongoing Phase 1 clinical trial in checkpoint therapy-naïve patients with selected recurrent or metastatic cancers, including ongoing cohorts intended to support one or more Biologics License Application submissions, and for general corporate purposes.

The offering is expected to close on or about November 22, 2019, subject to customary closing conditions.

A shelf registration statement on Form S‐3 (File. No. 333‐221493) (the "Registration Statement") relating to the shares of common stock being offered was filed with the U.S. Securities and Exchange Commission (SEC) and was declared effective on December 1, 2017. The offering is being made only by means of a prospectus. A preliminary prospectus supplement and accompanying prospectus describing the terms of the offering have been filed with the SEC. A final prospectus supplement to the base prospectus describing the final terms of the offering will be filed with the SEC and, when available, may be obtained from National Securities Corporation, Attn: Charles Wanyama, 200 Vesey Street, 25th Floor, New York, New York 10281, telephone: (212) 417-3634, or by email at [email protected]; or the on the SEC’s website at View Source

This press release shall not constitute an offer to sell or a solicitation of an offer to buy securities of the Company, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale is not permitted.

On November 20, 2019 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on harnessing the body’s immune system in the fight against cancer, reported its collaboration with Idaho Urologic Institute, PA (IUI) in the Cchek early cancer detection study (Press release, Anixa Biosciences, NOV 20, 2019, View Source [SID1234551536]). IUI will provide patient samples for Anixa’s ongoing prostate cancer study, including support of clinical validation of Anixa’s Cchek Prostate Cancer Confirmation test.

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IUI is one of several sites across the U.S. participating in this study. Dr. Lisa Parillo will serve as the Principal Investigator for the study at IUI.

Dr. Amit Kumar, Chief Executive Officer of Anixa, stated, "We are pleased to have IUI join our expanding group of Cchek collaboration partners. Our large and growing team of influential collaborators has been key to the development of our artificial intelligence driven liquid biopsy for early cancer detection. We look forward to IUI’s participation as we continue the clinical validation phase of the Cchek Prostate Cancer Confirmation test, in preparation for commercial launch later this year."

About Cchek
Cchek is an early cancer detection technology, which measures a patient’s immunological response to a malignancy by analyzing immune system cells in peripheral blood. The goal is to utilize the technology to determine a patient’s cancer status from a simple blood draw, eliminating the need for a biopsy, which can be an expensive, painful and invasive procedure. Further, conventional methods using current cancer screening tests often lack accuracy and reliability. Anixa’s orthogonal approach using flow cytometry coupled with artificial intelligence provides an alternative method with greater affordability, efficacy and efficiency. To date, Anixa has successfully used Cchek to detect the presence of 20 different cancers including lung, colon, breast and prostate. The robust cancer detection performance of Cchek makes it a platform from which multiple cancer diagnostic tests may be developed. The first such test, a prostate cancer confirmation test, is slated for commercial launch by the end of 2019.