On April 8, 2021 Secura Bio, Inc. (Secura Bio) – (www.securabio.com), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, reported that it has completed enrollment, with 101 patients, into the PRIMO study, which is evaluating COPIKTRA for the treatment of patients with relapsed or refractory Peripheral T-cell Lymphoma (PTCL) (Press release, Secura Bio, APR 8, 2021, https://www.prnewswire.com/news-releases/secura-bio-announces-enrollment-completion-of-the-copiktra-duvelisib-study-primo-in-peripheral-t-cell-lymphoma-301264723.html [SID1234577765]). The completion of study enrollment is an important milestone in the continued development of COPIKTRA to treat T-cell lymphomas, a disease category for which it is not currently indicated.

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The PRIMO study is a global, multi-center, open-label, parallel cohort registration-directed Phase 2 study. In the dose optimization portion of the study, patients were randomized to receive COPIKTRA 25mg twice daily (cohort 1) or COPIKTRA 75mg twice daily continuously (cohort 2) until disease progression or unacceptable toxicity. Based on the dose optimization results, an expansion group was added in which COPIKTRA was dosed at 75mg twice daily for two cycles, followed by 25mg twice daily, until disease progression or unacceptable toxicity. The primary endpoint in the expansion phase of the study is independent review committee assessed overall response rate (ORR). Secondary endpoints include duration of response (DOR) and safety.

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and gamma pathways, which are involved in the proliferation and sustenance of malignant cells.

"Secura Bio will continue to aggressively support the development of COPIKTRA in the treatment of T-cell malignancies, because these patients often have limited therapeutic options and generally poor outcomes, and because PI3K inhibition appears to be a relevant, safe and promising mechanism of action" said Dr. David Cohan, Chief Medical Officer of Secura Bio.

For the treatment of PTCL, COPIKTRA monotherapy has received Fast Track status and Orphan Drug Designation, and investigations are imminently planned in combination with other proven anti-cancer agents.

"Secura Bio now has two meaningful oncology drugs with novel modes of action that offer the potential to build a broad portfolio of indications in hematologic and solid malignancies. We will also fully explore the combination of COPIKTRA with FARYDAK (panobinostat), Secura Bio’s pan-HDAC inhibitor" said Joseph M. Limber, President and CEO of Secura Bio.

Interim results of the PRIMO study are expected to be published later in 2021.

On April 8, 2021 Morphogenesis, Inc. ("Morphogenesis"), a clinical stage biotechnology company specializing in the development of cell and gene therapy products to treat chronic diseases such as cancer, reported that the United States Food and Drug Administration (FDA) has granted the Company’s lead clinical stage candidate, IFx-Hu2.0, Fast Track drug designation for the treatment of patients with advanced skin cancer (Press release, Morphogenesis, APR 8, 2021, View Source [SID1234578994]). Specifically, these are patients with unresectable or metastatic cutaneous melanoma who have not responded to, or have stopped responding to, FDA-approved immune checkpoint inhibitors. Most advanced skin cancer patients who receive immune checkpoint inhibitors do not respond to these therapies. Based on the data amassed to date and its mechanism of action, IFx-Hu2.0 has the potential to change that. The FDA previously granted Orphan Drug Designation to IFx-Hu2.0 for the same indication.

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Fast Track is a designation issued by the FDA for products that have the potential to treat patients with a serious disease and who have an unmet medical need. Morphogenesis provided the FDA with epidemiologic data as well as pre-clinical and clinical evidence to support IFx-Hu2.0’s mechanism of action. IFx-Hu2.0 is an immunomodulator that as well as initiating a significant anti-tumor response in previously untreated patients, also has the potential to re-sensitize patients to immune checkpoint inhibitors. More people have skin cancer than all the other primary cancers in the United States combined. Advanced melanoma’s aggressiveness and high mortality rate make it the deadliest type of skin cancer.

"We believe that being able to initiate a broad anti-tumor response in late-stage melanoma patients with a 30-second intralesional injection of IFx-Hu2.0 fulfills an unmet medical need of these patients," said Patricia D. Lawman, PhD, Chief Executive Officer of Morphogenesis.

"Fast Track designation for IFx-Hu2.0 signifies the FDA’s recognition that patients suffering from this life-threatening disease remain in dire need of effective treatment options even with all the therapies that are being developed for melanoma. IFx-Hu2.0 holds promise to effectively treat these patients," said Michael J.P. Lawman, PhD, President of Morphogenesis.

The FDA is committed to facilitating the development and to expediting the review of IFx-Hu2.0 to treat patients with advanced skin cancer, thereby potentially addressing a significant unmet medical need. Morphogenesis is advancing the clinical development of IFx-Hu2.0, a highly differentiated cancer immunotherapy drug candidate, especially since IFx-Hu2.0 is now eligible for accelerated approval and priority review if relevant criteria are met.

"With IFx-Hu2.0’s Fast Track designation, we look forward to having expert guidance and frequent communication with the FDA throughout the entire drug development and review process," said Christopher Konig, PharmD, Regulatory Affairs Manager at Morphogenesis. "In the past, this quality and frequency of communication with the FDA has led to earlier drug approval and access by patients."

On April 8, 2021 Silverback Therapeutics, Inc. (Nasdaq: SBTX) ("Silverback"), a clinical-stage biopharmaceutical company leveraging its proprietary ImmunoTAC technology platform to develop systemically delivered, tissue-targeted therapeutics for the treatment of cancer, chronic viral infections and other serious diseases, reported that it will present preclinical data on its second product candidate, SBT6290, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2021 Annual Conference, taking place virtually April 10-15 (Press release, Silverback Therapeutics, APR 8, 2021, View Source [SID1234577749]). The data highlight the ability of SBT6290, a systemically administered Nectin4-directed TLR8 ImmunoTAC product candidate, to selectively activate myeloid cells in the tumor microenvironment, a promising approach to overcome resistance mechanisms associated with most current immunotherapies.

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"SBT6290 showcases the versatility of our ImmunoTAC platform and along with our lead program SBT6050, exemplifies our goal to target difficult-to-treat solid tumors subset by subset. We are building on learnings from SBT6050, leveraging the same TLR8 linker-payload, which is highly transferrable across different targeting antibodies," said Valerie Odegard, Ph.D., president & chief scientific officer of Silverback Therapeutics. "These preclinical data show that SBT6290 activates human myeloid cells in a Nectin4-dependent manner and that a SBT6290 mouse surrogate confers single agent anti-tumor activity in preclinical studies. We will continue to evaluate SBT6290 in GLP toxicology studies and anticipate submitting an IND for this program in the fourth quarter of 2021."

SBT6290 comprises a selective TLR8 agonist conjugated to a Nectin4-specific monoclonal antibody. Nectin4 is a cell surface adhesion molecule that is overexpressed in multiple solid tumor types including urothelial, triple negative breast, squamous cell head and neck, and non-small cell lung cancers, with limited expression in normal tissues.

The abstract is now available on the AACR (Free AACR Whitepaper) Annual Meeting website. Details of the poster presentation are as follows:

Title: SBT6290, a Systemically Administered Nectin4-Directed TLR8 ImmunoTAC Product Candidate, is Designed for Tumor-localized Activation of Myeloid Cells

Abstract Number: 1858

Session Category: Immunology

Session Title: Therapeutic Antibodies, Including Engineered Antibodies

Virtual Poster Session Date and Time: The poster will be made available on the AACR (Free AACR Whitepaper) website and Silverback website on April 10, 2021 at 8:30 a.m. ET

On April 8, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, reported that it will host a virtual R&D day for investors and analysts on Tuesday, June 8, 2021, from 12:30 p.m. to 2:30 p.m. EDT (Press release, NeuBase Therapeutics, APR 8, 2021, View Source [SID1234577768]).

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During the event, NeuBase will present new data, along with an in-depth review of the Company’s pipeline of drug candidates – including in Huntington’s disease, myotonic dystrophy type 1, and a new oncology program for a target that has previously been thought of as undruggable – as well as an introduction to the expanded management team.

Additional details will be made available prior to the event. The event will be webcast live on NeuBase’s website at View Source Following the live webcast, a replay will be available on the Company’s website and archived for approximately 90 days.

On April 8, 2021 Ventus Therapeutics, Inc., a biotechnology company utilizing rational structure-based drug design to discover small-molecule medicines for high value and elusive targets, reported a $100 million Series B financing led by RA Capital Management with participation from a select syndicate including BVF Partners L.P., Casdin Capital, Cormorant Asset Management, Fonds de solidarité FTQ, and Alexandria Venture Investments (Press release, Ventus Therapeutics, APR 8, 2021, View Source [SID1234577718]). Founding investor Versant Ventures also participated in the round, as did existing investor GV (formerly Google Ventures).

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"This support from strong crossover investors will enable the next stage of evolution for Ventus as we build the resources to leverage the full potential of our platforms and product pipeline," said Marcelo Bigal, M.D., Ph.D., president and CEO of Ventus. "We have made rapid and substantial progress over the last twelve months, including the emergence of the ReSOLVE platform and expansion of our pipeline. Ventus is on an exciting growth trajectory, as we continue to unlock high-value targets that have been elusive in the past and advance our lead products for patients with devastating diseases."

Ventus’ drug discovery platforms combine computational technologies with leading-edge structural biology, protein engineering, biophysics and chemistry. Using two proprietary platforms, Ventus expands the set of drug targets accessible to small-molecule medicines by discovering and validating novel binding sites invisible to other approaches.

Proceeds from the Series B will be used to advance Ventus’ pipeline of novel medicines to treat diseases in multiple therapeutic areas and expand platform capabilities. The company’s most advanced programs are focused on critical targets of the innate immune system. Additional programs in autoimmune, inflammation, oncology and neurology indications create a diversified pipeline with opportunities for both partnerships and internal growth.

While the targets of Ventus’ lead program and other pipeline programs are undisclosed, the company’s second most advanced molecule targets NLRP3, a key mediator of pro-inflammatory cytokines IL-1b and IL-18. NLRP3 is implicated in many autoimmune diseases and other conditions characterized by aberrant inflammation. Ventus’ platforms enabled the stable expression of inactive subunits of NLRP3 and determination of the precise dynamic solvation structure of NLRP3’s binding pocket. Based on these breakthroughs, Ventus identified and optimized novel and differentiated NLRP3 inhibitors, including high potency and quality brain-penetrant molecules as well as peripherally restricted compounds.

Concurrent with this financing, Josh Resnick, M.D., managing director at RA Capital Management, will join the Ventus Board of Directors.

"Ventus is at the forefront of advances in structure-based drug design," said Dr. Resnick. "We are excited that the company has proven its ability to drug challenging targets and look forward to continued progress by Ventus’ pipeline as the company seeks to bring new options to patients."