BeyondSpring Announces Final Positive Data from the PROTECTIVE-1 Phase 3 CIN Program of Plinabulin as a Single Agent Compared to Pegfilgrastim at the American Society of Clinical Oncology (ASCO) Annual Meeting

On June 10, 2021 BeyondSpring Inc. (the "Company" or "BeyondSpring") (NASDAQ: BYSI), a global biopharmaceutical company focused on the development of innovative cancer therapies, reported presentation of data from the PROTECTIVE-1 Phase 3 clinical study of plinabulin for prevention of chemotherapy-induced neutropenia (CIN) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held on June 4 – 8, 2021 (Press release, BeyondSpring Pharmaceuticals, JUN 10, 2021, https://beyondspringpharma.com/beyondspring-announces-final-positive-data-from-the-protective-1-phase-3-cin-program-of-plinabulin-as-a-single-agent-compared-to-pegfilgrastim-at-the-american-society-of-clinical-oncology-asco-annua/?utm_source=rss&utm_medium=rss&utm_campaign=beyondspring-announces-final-positive-data-from-the-protective-1-phase-3-cin-program-of-plinabulin-as-a-single-agent-compared-to-pegfilgrastim-at-the-american-society-of-clinical-oncology-asco-annua [SID1234585696]).

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The poster titled "Head-to-Head Comparison of Single Agent (SA) Plinabulin (Plin) versus Pegfilgrastim (Peg) for the Prevention of Chemotherapy-Induced Neutropenia (CIN) in the Phase 3 Trial PROTECTIVE-1," was presented at 9:00 a.m. ET on June 4, 2021, at Lung Cancer Poster Session (Abstract #547) is the PROTECTIVE-1 Phase 3 data comparing plinabulin versus pegfilgrastim. Data further supports plinabulin’s fast onset of action and CIN prevention benefit in week 1 following chemotherapy with results including clinically meaningful endpoints for reduction of febrile neutropenia (FN), hospitalization and bone pain.

Primary endpoint of demonstrating non-inferiority of single agent plinabulin versus single agent pegfilgrastim was met: DSN (days of severe neutropenia) cycle 1, non-inferiority margin of 0.65 day criterion between plinabulin and pegfilgrastim was met.
Comparable or numerically better clinical sequelae of CIN for plinabulin vs. pegfilgrastim:
FN Infection Antibiotics
Use

Hospitalization Docetaxel Discontinue Docetaxel Delay
Plin (n=52) 0% 7.69% 15.4% 3.84% 13.5% 3.85%
Peg (n=53) 1.89% 15.1% 13.2% 1.89% 26.4% 5.66%
Significant improvement in bone pain and platelet count: Less bone pain (p=0.01) and less thrombocytopenia (p<0.0001 on D15) compared to pegfilgrastim.
Same day convenience of use: Plinabulin is infused on the same day (Day 1) as chemotherapy, 30 minutes after chemotherapy with 30 minutes of intravenous infusion, whereas pegfilgrastim on the next day (Day 2).
"The dedicated program of plinabulin in CIN prevention was comprised of four studies to show the unique profile of plinabulin: PROTECTIVE-1 Phase 2 and Phase 3 and PROTECTIVE-2 Phase 2 and Phase 3, which were designed in consultation with the FDA to explore the CIN benefit of novel agent plinabulin. We are very pleased to announce that all four studies were positive and met their primary and key secondary endpoints," said Ramon Mohanlal M.D., Ph.D., Chief Medical Officer and Executive Vice President of Research and Development at BeyondSpring. "All these four studies support the combination regimen of plinabulin and G-CSF for an intended broad label to prevent CIN in all solid tumors and all chemotherapy, which is included in our NDA filing."

The Company has submitted New Drug Applications (NDA) for plinabulin in combination with pegfilgrastim for the prevention of CIN, in both the U.S. and China. The U.S. FDA accepted the NDA with Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of November 30, 2021.

About Plinabulin

Plinabulin, BeyondSpring’s lead drug, is a selective immunomodulating microtubule-binding agent (SIMBA). It is a novel, intravenous infused, patent-protected drug that is NDA filed for CIN prevention in the U.S. and China and has a fully enrolled pivotal Phase 3 anti-cancer study for non-small cell lung cancer (NSCLC). Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anticancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells, and the second is early-onset action in CIN prevention after chemotherapy by boosting the number of hematopoietic stem/progenitor cells (HSPCs). Plinabulin received breakthrough designation from both U.S. and China FDA for CIN prevention indication. As a "pipeline in a drug," plinabulin is being broadly studied in various immuno-oncology regimens to determine if it can boost the effects of the PD-1/PD-L1 antibodies and re-sensitize PD-1/PD-L1 antibody resistant patients.

About Plinabulin CIN NDA Program

The Plinabulin and G-CSF combination NDA program is comprised of four studies: PROTECTIVE-1 Phase 2 and Phase 3 and PROTECTIVE-2 Phase 2 and Phase 3.

PROTECTIVE-1 Phase 2 (4arm randomized study, NSCLC, docetaxel, n=55): Plinabulin single agent (5 mg/m2, 10 mg/m2, or 20 mg/m2, day 1 dose), pegfilgrastim (6 mg, day 2 dose). Plinabulin 20 mg/m2 was selected as the Phase 3 dose.
PROTECTIVE-1 Phase 3 (2 arm randomized, double-blind study, NSCLC, breast cancer, prostate cancer, docetaxel, n=105 patients): Plinabulin single agent (40 mg fixed dose – equivalent to 20 mg/m2, day 1 dose) vs. pegfilgrastim (6 mg, day 2 dose). Primary endpoint DSN cycle is met with non-inferiority. Plinabulin showed week 1 early onset of action and was effective in prevention of clinical outcomes, such as FN and hospitalization, and chemo dose reduction and delay.
PROTECTIVE-2 Phase 2 (7 arm randomized study, breast cancer, TAC, n=115 patients): Plinabulin single agent (10 mg/m2, 20 mg/m2, or 30 mg/m2, day 1 dose) – 20 mg/m2 selected; Plinabulin (20 mg/m2, day 1 dose) + pegfilgrastim (1.5 mg, 3 mg, or 6 mg, day 2 dose); pegfilgrastim (6 mg, dose 2 dose). Plinabulin (20 mg/m2) and pegfilgrastim (6 mg) was selected to be Phase 3 dose.
PROTECTIVE-2 Phase 3 (2 arm randomized, double-blind study, breast cancer, TAC, 221 patients – Pivotal study): Plinabulin (40 mg fixed dose – equivalent to 20 mg/m2, day 1 dose) + pegfilgratstim (6 mg, day 2 dose) vs. pegfilgrastim (6 mg, day 2 dose). The combination is superior to pegfilgrastim alone, which met primary endpoint of prevention of grade 4 neutropenia and key secondary endpoints, with superior benefit in reducing the incidence and severity of febrile neutropenia (FN) and hospitalization, with better quality-of-life (QoL)
About CIN

CIN remains a severely unmet medical need and is the primary cause for the 4D’s (Decrease, Delay, Discontinue dose and Downgrade regimen) that compromise carefully selected cancer treatment regimens. Treatment or prevention of CIN with G-CSF has been the standard of care since Neupogen was approved in 1991. The main benefit of G-CSF treatment, however, is in Week 2 after chemotherapy. Week 1 after chemotherapy is considered the "neutropenia vulnerability gap" where over 75% of CIN-related clinical complications occur, including febrile neutropenia, infection, hospitalization and death. Plinabulin is the first drug seeking FDA approval that has the potential to fill this gap. Combining plinabulin and G-CSF may maximize the protection of patients for the full cycle of chemotherapy, as demonstrated in the PROTECTIVE-2 Phase 3 registration study.

Each year in the U.S., 110,000 patients receiving chemotherapy are hospitalized after developing CIN, a severe side effect that increases the risk of infection with fever (also called FN). Due to the COVID-19 pandemic, the updated National Comprehensive Cancer Network (NCCN) guidelines expanded the use of prophylactic G-CSFs, including pegfilgrastim, from high-risk patients only (chemo FN rate >20%), to include intermediate-risk patients (FN rate between 10-20%), to reduce the number of hospital/ER visits related to CIN. The revision of the NCCN guidelines effectively increases the addressable market of patients to approximately 467,500 cancer patients in the U.S. annually.

Viewpoint Molecular Targeting™ Commences Patient Enrollment in Phase 1 Imaging Study for VMT01 Program for Metastatic Melanoma

On June 10, 2021 Viewpoint Molecular Targeting, Inc. ("Viewpoint" or the "Company"), a radiopharmaceutical company developing precision lead-212-based α-particle oncology therapeutics and complementary diagnostic imaging agents, reported it has initiated patient enrollment and screening in its Phase 1 imaging study evaluating VMT01 for the treatment of metastatic melanoma (Press release, Viewpoint Molecular Targeting, JUN 10, 2021, https://viewpointmt.com/viewpoint-molecular-targeting-commences-patient-enrollment-in-phase-1-imaging-study-for-vmt01-program-for-metastatic-melanoma/ [SID1234585490]).

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Melanoma is a cancer of the skin and the most aggressive form of skin cancer. While a disease that is identified early (when confined to the outer layers of the skin) can be cured by surgery, melanoma that migrates to distant sites in the body (metastatic melanoma) is difficult to treat. Despite the introduction of new treatments for metastatic melanoma, according to the American Cancer Society, the five-year survival is approximately 25%.

"The start of the VMT01 imaging study represents a significant milestone for the Company. The study tests Viewpoint’s investigational imaging agents in subjects with advanced melanoma to accelerate therapeutic drug development and enable image-guided treatment. We are pleased to be working alongside Mayo Clinic to advance this novel theranostic approach to radiopharmaceutical therapy against metastatic melanoma," commented Frances L. Johnson, MD, Chief Executive Officer and Co-Founder of Viewpoint Molecular Targeting. "Based on the extremely encouraging preclinical data seen to date, we believe that our targeted alpha-particle therapies have the potential to significantly enhance treatment responses in metastatic melanoma."

The Phase 1 first-in-human, crossover design study of Positron Emission Tomography (PET) and Single-Photon Emission Computerized Tomography (SPECT) scanning will enroll approximately 10 subjects with Stage III/IV unresectable melanoma. The primary endpoint of the study is safety and biodistribution. Secondary endpoints of the study are molecular target validation and image quality. The current VMT01 Phase 1 imaging study is being conducted at Mayo Clinic in Rochester, MN, and a future therapy study is planned. Provisional results for the imaging study are targeted for Q4 2021. Following the results of the imaging trial, the Company plans to initiate a multisite Phase 1/2a therapy study of VMT01 for the treatment of metastatic melanoma.

Viewpoint’s VMT01 program is intended to address an unmet clinical need with the use of a new imaging agent to guide Viewpoint’s radiopharmaceutical therapy against metastatic melanoma. This image-guided approach is often referred to as "theranostics." Using information guided by the low-risk medical imaging scan, a treatment plan utilizing the VMT01 ligand is designed to deliver the power of alpha-particle radiation specifically to melanoma tumors, while minimizing risk to unaffected organs and tissues. VMT01 represents a unique way to treat metastatic melanoma that has been vetted as scientifically sound by rigorous peer review and has the potential to be transformative for melanoma patients.

Labcorp and OmniSeq Launch Insight, Next-Generation Sequencing Platform to Advance Precision Oncology

On June 10, 2021 Labcorp (NYSE: LH), a leading global life sciences company, and OmniSeq, a CAP-accredited, molecular diagnostic innovation of Roswell Park Comprehensive Cancer Center, reported the launch of OmniSeq INSIGHTsm, a comprehensive genomic and immune profiling, tissue-based test that integrates next-generation sequencing (NGS) technology (Press release, OmniSeq, JUN 10, 2021, View Source [SID1234585347]). The test is designed to advance precision oncology and improve patient outcomes, as part of Labcorp’s commitment to empower better health decisions for patients through the addition of cutting-edge diagnostic tools in precision medicine.

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"There continue to be significant advancements in the oncology space, including the identification of new biomarkers and therapies at an unprecedented pace," said Prasanth Reddy, M.D., MPH, senior vice president and oncology head at Labcorp and a triple board-certified oncologist. "This new test provides up-to-date, evidence-based treatment recommendations and incorporates current guidelines into a report that is easy to interpret, allowing clinicians to make the best treatment choices for their patients, including potential clinical trials."

OmniSeq INSIGHT is a pan-cancer, solid tumor test that combines two different treatment paradigms— genomic and immune profiling, including tumor mutational burden, microsatellite instability, and immune gene expression, which enhances the characterization of the unique genomic biomarkers in a patient’s tumor. The results from the test are presented in an easy-to-read report, summarizing all FDA-approved therapies and immunotherapies that match the patient’s tumor profile. This enables clinicians to provide the most up-to-date treatments for their patients, in addition to identifying clinical trials for which patients may be eligible within 200 miles of their homes. The report provides evidence-based recommendations specific to the individual test results through the use of a proprietary database sourced from numerous biologic and scientific literature, including National Comprehensive Cancer Network (NCCN) guidelines, giving clinicians access to the most advanced treatment options.

OmniSeq INSIGHT is available to U.S.-based clinicians exclusively through Labcorp, and across Canada through Dynacare, a Labcorp company. The test is also available to global biopharmaceutical companies exclusively through Labcorp Drug Development. This can help reduce development time and costs while maximizing patient stratification by accelerating biomarker selection in preclinical and clinical development, and in targeted therapeutic and companion diagnostic development.

Labcorp Drug Development has a proven track record of providing a range of solutions and can offer the scalability required, backed by global expertise, regulatory guidance and informatics systems that are tailored to the biopharmaceutical partner’s unique development needs.

"We are excited to advance our partnership with Labcorp, continuing to provide increased access to oncology care for patients leveraging Labcorp’s extensive footprint and oncology experience," said Margot Schoenborn, CEO of OmniSeq. "We are making this test available globally to biopharmaceutical customers for incorporation into their clinical trials for biomarker stratification, and to support new precision medicine oncology approaches to drug and companion diagnostic development."

OmniSeq INSIGHT is NYS CLEP approved and leverages Labcorp’s broad national coverage, including in-network with most major health plans and 1,600 contractual relationships with plans, payers and other health care organizations.

Labcorp and OnmiSeq Launch Insight, Next-Generation Sequencing Platform to Advance Precision Oncology

On June 10, 2021 Labcorp (NYSE: LH), a leading global life sciences company, and OmniSeq, a CAP-accredited, molecular diagnostic innovation of Roswell Park Comprehensive Cancer Center, reported the launch of OmniSeq INSIGHTsm, a comprehensive genomic and immune profiling, tissue-based test that integrates next-generation sequencing (NGS) technology (Press release, OmniSeq, JUN 10, 2021, View Source [SID1234583930]). The test is designed to advance precision oncology and improve patient outcomes, as part of Labcorp’s commitment to empower better health decisions for patients through the addition of cutting-edge diagnostic tools in precision medicine.

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"There continue to be significant advancements in the oncology space, including the identification of new biomarkers and therapies at an unprecedented pace," said Prasanth Reddy, M.D., MPH, senior vice president and oncology head at Labcorp and a triple board-certified oncologist. "This new test provides up-to-date, evidence-based treatment recommendations and incorporates current guidelines into a report that is easy to interpret, allowing clinicians to make the best treatment choices for their patients, including potential clinical trials."

OmniSeq INSIGHT is a pan-cancer, solid tumor test that combines two different treatment paradigms— genomic and immune profiling, including tumor mutational burden, microsatellite instability, and immune gene expression, which enhances the characterization of the unique genomic biomarkers in a patient’s tumor. The results from the test are presented in an easy-to-read report, summarizing all FDA-approved therapies and immunotherapies that match the patient’s tumor profile. This enables clinicians to provide the most up-to-date treatments for their patients, in addition to identifying clinical trials for which patients may be eligible within 200 miles of their homes. The report provides evidence-based recommendations specific to the individual test results through the use of a proprietary database sourced from numerous biologic and scientific literature, including National Comprehensive Cancer Network (NCCN) guidelines, giving clinicians access to the most advanced treatment options.

OmniSeq INSIGHT is available to U.S.-based clinicians exclusively through Labcorp, and across Canada through Dynacare, a Labcorp company. The test is also available to global biopharmaceutical companies exclusively through Labcorp Drug Development. This can help reduce development time and costs while maximizing patient stratification by accelerating biomarker selection in preclinical and clinical development, and in targeted therapeutic and companion diagnostic development.

Labcorp Drug Development has a proven track record of providing a range of solutions and can offer the scalability required, backed by global expertise, regulatory guidance and informatics systems that are tailored to the biopharmaceutical partner’s unique development needs.

"We are excited to advance our partnership with Labcorp, continuing to provide increased access to oncology care for patients leveraging Labcorp’s extensive footprint and oncology experience," said Margot Schoenborn, CEO of OmniSeq. "We are making this test available globally to biopharmaceutical customers for incorporation into their clinical trials for biomarker stratification, and to support new precision medicine oncology approaches to drug and companion diagnostic development."

OmniSeq INSIGHT is NYS CLEP approved and leverages Labcorp’s broad national coverage, including in-network with most major health plans and 1,600 contractual relationships with plans, payers and other health care organizations.

Valo Health to go public via Khosla-backed SPAC, gaining $500M-plus

On June 10, 2021 Valo Health reported that unveiled plans to go public by merging with a special purpose acquisition company formed by Khosla Ventures(Press release, Valo Health, JUN 10, 2021, View Source [SID1234583927]).

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The combined company will have about $750 million in the bank when the deal closes sometime in the third quarter. That includes $250 million from Valo, $333 million from the SPAC, Khosla Ventures Acquisition Co., and $168.5 million through a private financing from the likes of Khosla Ventures, Koch Disruptive Technologies and Flagship Pioneering.

Khosla Ventures Acquisition Corp., or KVAC for short, raised $300 million in its Nasdaq IPO in March and went on the hunt for one or more companies "addressing large market opportunities with highly differentiated proprietary technology" to merge with or acquire, the SPAC said in a securities filing. It could have gone into several other sectors, such as food and agriculture or climate, but ultimately landed on Valo, a biotech using computational technology to transform drug development and trim years off R&D timelines.

RELATED: Valo Health grabs $110M top-up, bringing series B to $300M

The proceeds from the deal will propel Valo’s preclinical and clinical programs, develop its software platform and support "new and existing growth initiatives," Valo said in a statement Wednesday.

Valo revealed its first four preclinical programs in January when it closed the first $190 million of its series B round. Those comprise cancer treatments targeting a mixture of known targets and new targets: PARP1, NAMPT, USP28 and HDAC3. Two of its programs have since entered the clinic, the company said in the statement, though it did not specify which ones. And that’s not all—its bench is much deeper than that, with 15 prioritized preclinical programs in total across oncology and neurodegeneration as well as cardiovascular, metabolic and renal diseases, according to the statement.

The company’s pipeline is based on its Opal Computational Platform, an end-to-end drug discovery and development platform. As the company develops its prospects, the Opal technology actively learns, so the company isn’t just advancing new medicines but also improving its discovery and development engine at the same time, said Valo CEO David Berry in a previous interview.

In the case of its PARP1 program, Valo used the platform to create a PARP inhibitor that could cross the blood-brain barrier without sacrificing efficacy and target PARP1 specifically rather than both PARP1 and PARP2, Berry said.

RELATED: Roivant catches the SPAC wave to Wall Street in $611M deal

It hopes to improve upon PARP inhibitors such as AstraZeneca and Merck’s Lynparza and Zejula from GlaxoSmithKline’s Tesaro, which have seen success in breast and ovarian cancers but don’t get into the brain. That stops them from being effectively used to treat cancers that spread to the brain as well as cancers that start in the brain.

Previously known as Integral Health, Valo launched in September 2020, acquiring Forma Therapeutics’ hit discovery capabilities for $2.5 million upfront, $17.5 million in installments and $10 million in equity. It had also acquired Numerate, which gave Valo the engineers who created a platform with more than 30,000 models and 70 trillion molecules.