On March 3, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused pharmaceutical company, reported the pricing of its previously announced registered underwritten public offering of 6,250,000 shares of its common stock at a price to the public of $24.00 per share (Press release, Karyopharm, MAR 3, 2020, View Source [SID1234555121]). The gross proceeds to Karyopharm from the offering, before deducting the underwriting discounts and commissions and other estimated offering expenses, are expected to be $150.0 million. The offering is expected to close on or about March 6, 2020, subject to customary closing conditions. In addition, Karyopharm has granted the underwriters a 30-day option to purchase up to 937,500 additional shares of its common stock.

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J.P. Morgan, Morgan Stanley and Jefferies are acting as joint book-running managers for the offering. RBC Capital Markets, Baird and H.C. Wainwright & Co. are acting as co-managers for the offering.

Karyopharm intends to use the net proceeds of the offering (i) to maintain and grow the infrastructure to support the continued commercialization of selinexor in the United States, including further developing our sales, marketing and market access functions along with related general and administrative capabilities; (ii) to support continued clinical development of selinexor in hematologic malignancies and solid tumors; (iii) to conduct activities to support regulatory submissions for oral selinexor as a potential second line therapy for patients with relapsed or refractory multiple myeloma and as a potential new treatment for patients with relapsed/refractory diffuse large B-cell lymphoma; (iv) for conducting clinical trials of two of our pipeline drug candidates in oncology, eltanexor, a second-generation SINE compound, and KPT-9274, a dual acting p21-activated kinase 4 (PAK4) allosteric modulator and nicotinamide phosphoribosyltransferase (NAMPT) inhibitor; and (v) for working capital and other general corporate purposes.

An automatically effective shelf registration statement relating to the shares of common stock offered in the public offering described above was filed with the Securities and Exchange Commission (SEC) on February 26, 2020. The securities may be offered only by means of a written prospectus, including a prospectus supplement, forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from J.P. Morgan Securities LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at [email protected]; Morgan Stanley & Co. LLC, 180 Varick Street, 2nd Floor, New York, NY 10014, Attention: Prospectus Department; or Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by email at [email protected], or by phone at (877) 821-7388.

On March 3, 2020 VBI Vaccines Inc. (NASDAQ: VBIV) ("VBI"), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported an update on Part A of the ongoing Phase 1/2a study of VBI-1901, the company’s cancer vaccine immunotherapeutic candidate, for the treatment of patients with recurrent glioblastoma (GBM) (Press release, VBI Vaccines, MAR 3, 2020, View Source [SID1234555138]). For patients who had an immunologic response to the vaccine, considered to be vaccine responders, the 12-month overall survival (OS) rate was 83% (n=5/6), compared to 33% for vaccine non-responders (n=3/9). Similarly, among patients evaluable for response and survival in Part A, vaccine responders saw a 6.25-month improvement in median OS (14.0 months) compared to vaccine non-responders (7.75 months). VBI-1901 continues to be safe and well-tolerated at all doses tested, with no safety signals observed.

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"We remain encouraged by the data from this ongoing Phase 1/2a study of VBI-1901 in the recurrent GBM setting, a setting in which it has historically been incredibly difficult to show any benefit, especially with a monotherapy," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "The standard of care treatment in the recurrent GBM setting is not well defined, however, commonly-used regimens, including chemotherapy and VEGF-A inhibitors as monotherapy and in combination, have demonstrated 12-month OS rates below 50% with median OS around 8 to 12 months1. Moreover, these regimens often cause significant toxicity, poorly affecting quality of life for the individual. While the data generated for VBI-1901 is early, we continue to be optimistic as we work hard to provide any meaningful benefit to patients who currently have few treatment options."

Expanded immunologic, tumor imaging, and clinical data from the Phase 2a part of the study are expected in Q2 2020 – data from the VBI-1901 + GM-CSF arm – and Q4 2020 – data from the VBI-1901 + AS01B arm. Additionally, efforts are underway to define biomarkers that may help identify patients more likely to respond to VBI-1901.

About the Phase 1/2a Study Design
VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 38 patients with recurrent GBM:

Phase 1 (Part A)
Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) in recurrent GBM patients, with any number of prior recurrences.
This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg.
Enrollment completed in December 2018.
Phase 2a (Part B)
Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase.
This phase is a two-arm study, enrolling 10 first-recurrent GBM patients in each arm, assessing 10.0 µg of VBI-1901 in combination with either GM-CSF or GSK’s proprietary AS01B adjuvant system as immunomodulatory adjuvants.
Enrollment in both study arms is ongoing.
VBI-1901 is administered intradermally when adjuvanted with GM-CSF and will be administered intramuscularly when adjuvanted with AS01B adjuvant system. Patients will receive the vaccine immunotherapeutic every four weeks until tumor progression.

Additional information, including a detailed description of the study design, eligibility criteria, and investigator sites, is available at ClinicalTrials.gov using identifier NCT03382977.

On March 3, 2020 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, reported as of February 2020, a patient from the Phase 1 clinical trial for the treatment of glioblastoma multiforme ("GBM"), conducted by Reata Pharmaceuticals, Inc., remains cancer free. GBM is an aggressive type of brain cancer and, currently, there are no effective therapies approved to treat this disease (Press release, CNS Pharmaceuticals, MAR 3, 2020, View Source [SID1234555155]).

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In the Phase 1 trial, 44% of patients experienced a statistically significant improvement in progression-free survival. This 44% disease control rate was based on 11 patients (out of 25 evaluable patients) with stable disease, plus responders. One patient experienced a durable complete response, which is defined by the National Cancer Institute as the disappearance of all signs of cancer in response to treatment. This initial trial was conducted in 2006 and as of February 2020, the patient has remained cancer free for over 13 years.

"We are delighted to provide this update on Berubicin’s potential capabilities given the unmet need for this aggressive disease," commented John M. Climaco, Chief Executive Officer of CNS Pharmaceuticals. "We are looking forward to initiating our Phase 2 clinical trial in the second half of this year with our partner, WPD Pharmaceuticals, Inc. In addition, we expect WPD Pharmaceuticals, Inc. to commence a first-ever Phase 1 trial in children later this year. As previously announced, the pediatric trial will be conducted at Children’s Memorial Health Institute, the largest pediatric hospital in Poland." WPD Pharmaceuticals, Inc. was founded by Dr. Waldemar Priebe, the founder of the Company.

About Berubicin
Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc.

On March 3, 2020 Sunesis Presented the corporate presentation (Press release, Sunesis, MAR 3, 2020, View Source [SID1234555100]).

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On March 3, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s lead drug candidate, tipifarnib, for the treatment of adult patients with relapsed or refractory angioimmunoblastic T-cell lymphoma (AITL), follicular T-cell lymphoma (FTCL) and nodal peripheral T-cell lymphoma with T follicular helper (TFH) phenotype (Press release, Kura Oncology, MAR 3, 2020, View Source [SID1234555122]).

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"This important designation from the FDA comes just two months after tipifarnib was awarded Fast Track for the treatment of patients with HRAS mutant head and neck squamous cell carcinomas (HNSCC)," said Bridget Martell, M.A., M.D., Acting Chief Medical Officer of Kura Oncology. "We believe that this designation reflects tipifarnib’s significant potential in these devastating disease settings, and we are now actively preparing to initiate a second registration-directed trial of tipifarnib in advanced nodal lymphomas of TFH phenotype, including AITL."

Fast Track designation is granted by the FDA for products that are intended for the treatment of serious or life-threatening disease or conditions, which demonstrate the potential to address an unmet medical need. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application.

In December 2019, Kura reported updated clinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting showing robust and durable activity from tipifarnib as a monotherapy in relapsed or refractory AITL. The data demonstrated an objective response rate (ORR) of approximately 50% in a heavily pre-treated patient population, with a median of three prior regimens. Additionally, enhanced anti-tumor activity was observed in patients who carried mutations in the killer-cell immunoglobulin-like receptor, or KIR, a CXCL pathway-associated biomarker. These patients had an ORR of 70% and a complete response rate of 40%.

About T-Cell Lymphomas

Peripheral T-cell lymphomas compromise up to 20% of all aggressive non-Hodgkin lymphomas and consist of many different subtypes of fast-growing lymphomas, representing approximately 20,000 incident cases per year worldwide. Outcomes for these patients are poor, with 5-year survival of approximately 30%. Although several drugs have been approved in the relapsed and/or refractory setting, none has led to a survival benefit. In addition, the National Comprehensive Cancer Network guidelines currently recommend clinical trials for these patients. Significant advances in the genetic landscape of T-cell lymphomas have led to revisions to the World Health Organization classification and the introduction of new entities. As a result, cases of AITL, an aggressive form of T-cell lymphoma frequently characterized by high levels of CXCL12 expression, are now unified with FTCL under the classification of nodal lymphomas of TFH phenotype.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent, selective farnesyl transferase inhibitor in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients, showing compelling and durable anti-cancer activity in certain patient subsets. However, no molecular mechanism of action was determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next-generation sequencing and emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. The Company has received multiple issued patents for tipifarnib in the U.S. and foreign countries, including one issued by the U.S. Patent and Trademark Office in September 2019 that further extends Kura’s exclusivity to the use of any farnesyl transferase inhibitor for the treatment of CXCL12-expressing peripheral T-cell lymphoma and acute myeloid leukemia.