On March 28, 2020 Ryvu Therapeutics (WSE: RVU), a clinical-stage biopharmaceutical company developing novel small molecule therapies that address emerging targets in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation (ODD) to Ryvu’s SEL120, for the treatment of patients with acute myeloid leukemia (AML) (Press release, Ryvu Therapeutics, MAR 28, 2020, View Source [SID1234555967]).

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SEL120 is an oral, selective inhibitor of CDK8 kinase which is implicated in the development of hematological malignancies and solid tumors. A clinical phase 1b study of SEL120 is currently enrolling in 6 investigational sites in USA, investigating safety and preliminary efficacy of SEL120 in treatment of patients with relapsed or refractory AML or high-risk myelodysplastic syndrome (HR-MDS).

"The FDA’s granting orphan drug designation to SEL120, is a significant encouragement for advancement of our clinical strategic plan addressing the unmet medical needs in the area of AML treatment, a disease where patients still face poor prognosis" said Setareh Shamsili, MD, PhD, Chief Medical Officer and Executive VP at Ryvu Therapeutics. "SEL120 has shown strong proof of concept in the preclinical studies and has received a strategic support from The Leukemia & Lymphoma Society (LLS) through its Therapy Acceleration Program (TAP). SEL120 may have the potential to offer an important therapeutic benefit in AML and in particular to those refractory/relapsed AML patients with the poorest prognosis, worldwide."

Orphan drug status (ODD) is intended to advance drug development for rare diseases. The FDA provides ODD to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. The FDA’s orphan drug designation allows the drug for the designated indication to be eligible for requesting a seven-year period of U.S. marketing exclusivity upon approval of the drug, as well as potential of other development assistance and financial incentives: View Source

About SEL120

SEL120 is an oral, selective inhibitor of CDK8, a kinase which is a part of the mediator complex and is essential for the activity of super-enhancers important to the regulation of RNA transcription. CDK8 kinase is implicated in the development of hematological malignancies and solid tumors. SEL120 was discovered with the Ryvu Therapeutics discovery engine platform and is currently in Phase 1b study in adult patients with AML or HR-MDS who are refractory to treatment or have relapsed after previous therapies. Patients are enrolled in the study independent of specific tumor mutational burden. The study will also assess pharmacokinetic and pharmacodynamic parameters of SEL120.

SEL120 has received support from The Leukemia & Lymphoma Society Therapy Acceleration Program (TAP), a strategic initiative to partner directly with innovative biotechnology companies and leading research institutions to accelerate the development of promising new therapies for blood cancers. More information about TAP program is available at: View Source

About Acute Myeloid Leukemia

Acute myelogenous leukemia or acute myeloid leukemia (AML) is a heterogenous hematological malignancy involving the clonal expansion of myeloid blasts in the bone marrow and peripheral blood with possible spread to liver and spleen. It is estimated that there are around 90 000 cases of AML worldwide each year, and American Cancer Society estimates that approximately 20 000 of these in U.S. only. The median age at diagnosis is 66 years, with 54% patients over 65 years, and 33% over 75 years. Of those diagnosed at a later age, the diagnosis is often associated with underlying myelodysplastic syndromes (MDS), sometimes linked to cancer chemotherapy and radiotherapy exposure.

On March 27, 2020 Bonnie J Addario Lung Cancer Foundation reported the Food and Drug Administration approved durvalumab (IMFINZI, AstraZeneca) in combination with etoposide and either carboplatin or cisplatin as first-line treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) (Press release, Bonnie J Addario Lung Cancer Foundation, MAR 27, 2020, View Source [SID1234555996]).

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Efficacy of this combination in patients with previously untreated ES-SCLC was investigated in CASPIAN, a randomized, multicenter, active-controlled, open-label, trial (NCT03043872). The evaluation was based on the comparison of patients randomized to durvalumab plus chemotherapy vs. chemotherapy alone. The major efficacy outcome measure was overall survival (OS). Additional efficacy outcome measures were investigator-assessed progression-free survival (PFS) and objective response rate (ORR), per RECIST v1.1.

Median OS was 13.0 months (95% CI: 11.5, 14.8) in the durvalumab plus chemotherapy arm compared with 10.3 months (95% CI: 9.3, 11.2) in the chemotherapy alone arm (hazard ratio 0.73; 95% CI: 0.59, 0.91; p=0.0047).

Investigator-assessed PFS (96% of total planned events) showed a HR of 0.78 (95% CI: 0.65, 0.94), with median PFS of 5.1 months (95% CI: 4.7, 6.2) in the durvalumab plus chemotherapy arm and 5.4 months (95% CI: 4.8, 6.2) in the chemotherapy alone arm. The investigator-assessed confirmed ORR was 68% (95% CI: 62%, 73%) in the durvalumab plus chemotherapy arm and 58% (95% CI: 52%, 63%) in the chemotherapy alone arm.

The most common adverse reactions (≥20%) in patients with ES-SCLC were nausea, fatigue/asthenia, and alopecia.

For ES-SCLC, durvalumab is to be administered prior to chemotherapy on the same day. The recommended durvalumab dose when administered with etoposide and either carboplatin or cisplatin is 1500 mg every 3 weeks prior to chemotherapy and then every 4 weeks as a single agent.

View full prescribing information for IMFINZI.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

FDA granted this application priority review and granted durvalumab orphan drug designation for this indication. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

On March 27, 2020 Merrimack Pharmaceuticals, Inc. (the "Company") reported that it has entered into an Asset Purchase Agreement (the "Asset Purchase Agreement") with Celator Pharmaceuticals, Inc. (the "Buyer"), pursuant to which the Buyer agreed to purchase certain assets (the "Transferred Assets") relating to certain of the Company’s preclinical nanoliposome programs (the "Transaction") (Filing, 8-K, Merrimack, MAR 27, 2020, View Source [SID1234555983]). The Company and the Buyer completed the Transaction simultaneously with the execution of the Asset Purchase Agreement. Under the terms of the Asset Purchase Agreement, the Buyer agreed to pay to the Company upfront cash payment of $2.25 million, and also agreed to reimburse the Company for certain specified expenses and to assume certain liabilities with respect to the acquired assets.

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Pursuant to the Asset Purchase Agreement, the Company assigned to the Buyer the previously disclosed intellectual property license agreement among the Company, Ipsen S.A. and Ipsen Biopharm Ltd. (together with Ipsen S.A., "Ipsen"), dated as of April 3, 2017, pursuant to which Ipsen granted the Company licenses to certain patents. On March 27, 2020, the Company and the Buyer also entered into an intellectual property license agreement pursuant to which the Buyer granted to the Company an exclusive license to certain specified know-how included in the Transferred Assets and an exclusive sublicense to the patents which are the subject of the license agreement with Ipsen, in each case to exploit certain specified nanoliposome products.

Both the Company and the Buyer have agreed to indemnify the other for losses arising from certain breaches of the Asset Purchase Agreement and for certain other potential liabilities, subject to certain limitations. The Asset Purchase Agreement also contains customary representations and warranties and covenants. The assertions embodied in those representations and warranties were made solely for purposes of the Asset Purchase Agreement and may be subject to important qualifications and limitations agreed to by the Company and the Buyer in connection with negotiating its terms. Moreover, the representations and warranties may be subject to a contractual standard of materiality that may be different from what may be viewed as material to stockholders or may have been used for the purpose of allocating risk between the Company and the Buyer rather than establishing matters as facts. For the foregoing reasons, no person should rely on such representations and warranties as statements of factual information at the time they were made or otherwise.

The foregoing description of the Asset Purchase Agreement does not purport to be complete and is qualified in its entirety by reference to the terms of the Asset Purchase Agreement, a copy of which is filed as Exhibit 2.1 hereto and incorporated herein by reference.

On March 27, 2020 Guangzhou XlifeSciences, a partner of GenScript ProBio (GenScript’s CDMO segment) reported that the early phase clinical trial of TAEST16001 project has been started (Press release, GenScript, MAR 27, 2020, View Source [SID1234555958]). GenScript ProBio extends congratulations on this.

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This clinical trial project is China’s first TCR-T project targeted at solid tumor therapy, marking a milestone in immunotherapy in China. In 2019, XlifeSciences entrusted TCR-T products with complete independent intellectual property rights to GenScript ProBio. It is the first cooperation project between the parties, where GenScript ProBio works on plasmid and virus process development and clinical manufacturing and will be further responsible for commercial manufacturing.

"We congratulate XLifeSciences on the successful launch of TAEST16001 early phase clinical trial, which marks a milestone in the industry," said Dr. Brian Min, CEO of GenScript ProBio. "We are honored to offer technology and manufacturing support in such a meaningful project. With a one-stop high-quality plasmid and virus platform, GenScript ProBio facilitates XLifeSciences to complete all pre-clinical CMC research for IND filing. We will continue to follow up on the clinical trial. We wish that the clinical project proceeds smoothly so as to relieve patients from pain as soon as possible. We are optimistic about the prospects of the project. As China’s first company that has built the plasmid facility for gene and cell therapy, GenScript ProBio has well-established technological platform and quality system, and is capable of providing quality services to more customers.

On March 27, 2020 Seneca Biopharma, Inc. (Nasdaq: SNCA), a biopharmaceutical company focused on developing novel treatments for diseases of high unmet medical need, reported its financial results for the year ended December 31, 2019 (Press release, Seneca Biopharma, MAR 27, 2020, View Source [SID1234555956]).

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Business Highlights for the Year Ended December 31, 2019

In early 2019, the Company shifted its operating strategy and focus away from the development of our neural stem cell treatments and initiated an out-licensing effort to partner these programs, while seeking to in-license or acquire novel therapeutics that are complementary to our current technologies and could benefit from our development experience.

On July 17, 2019, we effected a 1-for-20 reverse stock split of our common stock;

In July of 2019, we completed an underwritten public offering of our securities resulting in gross proceeds of approximately $7.5 million;

On October 28, 2019, we changed our name from Neuralstem, Inc. to Seneca Biopharma, Inc.

Financial Results for the Year Ended December 31, 2019

Cash Position and Liquidity: At December 31, 2019, cash was approximately $5.1 million as compared to approximately $5.8 million at December 31, 2018. The $0.7 million decrease is due to cash used in operations of approximately $7.3 million partially offset by the proceeds from our July 2019 underwritten offering.

Operating Loss: Operating loss for the year ended December 31, 2019 was $8.6 million compared to a loss of $8.3 million for 2018. The increase in operating loss for the year was primarily due to the absence of revenue and a small increase in research and development expenses. General and administrative expenses totaled approximately $4.6 million during the two periods.

Net Loss: Net loss for the year ended December 31, 2019 was $8.4 million, or $3.80 per share, compared to a loss of $4.9 million, or $7.80 per share on a post-reverse stock-split basis, for 2018. The change in net loss was primarily attributed to non-cash related changes in the fair value of our liability classified stock purchase warrants.

R&D Expense: Research and development expense for the year ended December 31, 2019 was $4.1 million as compared to $4.0 million for the year ended December 31, 2018. The increase was primarily attributable to an increase in external consulting services engaged in the technical evaluation of our internal programs as well as the evaluation of certain potential assets we considered for acquisition. These costs were partially offset by lower clinical trial expenditures as we wound down clinical activates.

G&A Expense: General and administrative expense were approximately $4.6 million for both 2019 and 2018. As noted above, in early 2019, the Company shifted its operating strategy and initiated an out-licensing effort to partner our neural stem cell treatments while seeking to acquire novel therapeutics with the potential to be complementary to our current technologies and that could benefit from our development experience. Associated with this shift in strategic focus, our G&A expenses in 2019 reflect an enhanced internal management structure including individual consultants in key roles.

Liquidity: In January 2020, the Company entered into an agreement with certain accredited investors from our July 2019 underwritten offering. Under this agreement the exercise price of certain warrants issued in the July offering were reduced from $2.70 to $1.36 to induce the immediate cash exercise of such warrants. The Company received approximately $6.8 million in net proceeds. We believe that the proceeds from the warrant exercise, along with our cash as of December 31, 2019, will be sufficient to fund our planned operations for more than 12 months.

"With the proceeds from the January 2020 transaction, we have the capital to build out our management team and continue our initiative of evaluating new therapeutic products for development as well as seeking partners for our promising neural stem-cell therapeutic NSI-566" commented Dr. Kenneth Carter, Seneca’s Executive Chairman. "On this front, our China-based subsidiary is readying a new facility which will be utilized to complete activities for our China-based stroke trial from which we are expecting top line data by the end of this year. In addition, we recently held a discussion with the US FDA regarding a path forward for our ALS program. We will provide additional information on these programs when certain activities are concluded."