On June 22, 2022 Biogen (NASDAQ:BIIB) and Happify Health (www.HappifyHealth.com), the Intelligent Healing Company, reported a collaboration to provide a digital solution for patient education and engagement, powered by artificial intelligence (AI), to support people living with multiple sclerosis (MS) (Press release, Biogen, JUN 22, 2022, View Source [SID1234616185]). Through this collaboration, Biogen and Happify Health will help people with MS manage their care journey, improve their wellbeing, learn about treatment options, consult with experts, and connect with other people in the MS community for support.

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More than 2.3 million people live with MS globally, including over 1 million people in the United States, according to the National MS Society. Women are two to three times more likely to develop MS than men1.

"This collaboration is one of many ways we are focused on supporting patients by meeting them where they are. The combination of Biogen’s expertise in neuroscience and our commitment to digital health, along with Happify Health’s AI and digital therapeutic capabilities will help drive a connected and comprehensive experience that allows patients to feel supported throughout their journey," said Jason Hawbecker, Head of Strategic Partnerships & Collaborations at Biogen. "We are excited about the potential of this partnership to provide overall support for the MS community."

The Happify Health solution, which brings together a digital tool configured for MS and a care community (Kopa), offers holistic health and wellness support, with access to neurology providers (doctors, nurse practitioners, physicians assistants and nurses) as well as mental health specialists, dietitians, and rehabilitation professionals. The online community that recently launched provides a place for those living with MS to ask experts questions and discuss the physical and mental challenges they are facing with patients in similar stages. Happify Health uses its proprietary AI technology to personalize content to patients’ life stages, symptoms and interests.

"It can be challenging for people living with MS to stay up-to-date with the latest treatments, interpret new symptoms, or investigate changes in their health that may or may not be related to MS," said Ofer Leidner, President of Happify Health. "We are providing the MS community with a solution configured to accommodate their specific needs, as well as opportunities to engage with experts, receive curated content, and discuss disease management with other community members. We are very excited to partner with Biogen on this opportunity given their leadership in the MS space."

Biogen, a global leader in developing treatments for people living with serious neurological and neurodegenerative diseases, will provide support for the MS platform, including educational content and resources for the patient care journey. The company is a global leader in treating MS with a portfolio of medicines to treat relapsing forms of MS, characterized by periods where symptoms subside, with full or partial recovery, and there is no disease progression between attacks.

In addition to psoriasis and maternal health, MS is the third therapeutic area to be addressed through Happify Health’s unique SequenceTM approach. Sequences are configured to support specific medical conditions and weave together components like evidence-based digital therapeutics, online communities, and coaching in one unified platform, with recommendations tailored for each individual.

On June 22, 2022 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that the results from a small trial of just 18 rectal cancer patients in complete remission using an immunotherapy called dostarlimab are "impressive," whilst acknowledging there’s more work to be done (Press release, Propanc, JUN 22, 2022, View Source [SID1234616184]). Chief Scientific Officer and Co-Founder, Dr Julian Kenyon MD, MB, ChB, believes that the field’s biggest challenge remains that immunotherapies work inconsistently across cancers. Oncologists estimate a response rate of 20% across cancer types, according to the Wall Street Journal ("WSJ"). The drugs can wipe out cancers from some people, but fail to work for others. It is also uncertain whether the cancer may eventually return once a patient is in remission, even after a prolonged period of time.

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Immunotherapies like dostarlimab, known as a checkpoint inhibitor, seek to inhibit key regulators of the immune system that when stimulated, reduces the body’s immune response to fight cancer. Given that immunotherapies target specific gene sequences, it often means they can encounter resistance, due to mutations that occur and genetic variation even within the primary tumor of a patient. As a result, Dr Cercek, from Memorial Sloan Kettering, who conducted the study for dostarlimab, estimates only 10% of rectal cancer patients and about 4% of all cancers will respond to treatment, according to the WSJ.

"For many cancers, multiple factors can drive growth, making it hard to effectively match one biomarker, or a particular gene sequence, to a single drug. On the other hand, a therapeutic approach like our lead product candidate, PRP, which alters the characteristics of the cancer cell, by enforcing it to express proteins it normally wouldn’t, means the treatment is less likely to encounter resistance through mutations, which is what we have observed in the lab as well as in clinical practice," said Dr Julian Kenyon.

In addition to specifically selecting the 18 rectal cancer patients according to their genetic biomarker, the trial included patients that were pre-metastatic, where tumors were locally advanced in one area, but not spread to other organs. This means patients identified with metastatic cancer were excluded from the trial. Therefore, the treatment and prevention of metastatic cancer, the main cause of patient death for sufferers, still remains the unsolved, final frontier. Cancer stem cells, which are the cells responsible for spreading to other parts of the body, remains a key focus for Dr Kenyon.

Dr Kenyon said, "PRP is a proenzyme treatment that targets and eradicates cancer stem cells by altering multiple pathways of a cancerous cell rather than a single genetic sequence. We’ve observed that once they are treated with PRP, the effects are irreversible and are more easily recognizable by the immune system, therefore potentially improving the response rates of standard approaches like immunotherapy, to overcome advanced cancers. We look forward to testing the utility of PRP with these approaches as we further advance into the clinic."

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include kidney, ovarian, breast, brain, prostate, colorectal, lung, liver, uterine and skin cancers.

On June 22, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation to MB-106, Mustang’s CD20-targeted, autologous CAR T cell therapy for the treatment of Waldenstrom macroglobulinemia ("WM" or "Waldenstrom"), a rare type of B-cell non-Hodgkin lymphoma ("B-NHL") (Press release, Mustang Bio, JUN 22, 2022, View Source [SID1234616183]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Center ("Fred Hutch") to treat patients with relapsed or refractory B-NHLs and chronic lymphocytic leukemia ("CLL").

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The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent from intellectual property protection.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "We are very pleased to receive Orphan Drug Designation from the FDA, as it is an important regulatory milestone for Mustang’s MB-106 program for the treatment of Waldenstrom macroglobulinemia, a rare B-NHL with a significant unmet medical need. We look forward to dosing the first patient in our multicenter Phase 1/2 clinical trial evaluating the safety and efficacy of MB-106 for relapsed or refractory B-NHL and CLL under Mustang’s IND shortly. In the Phase 1 portion of this trial, MB-106 dose escalation will proceed in three separate arms, and Waldenstrom patients will be included in the indolent lymphoma arm in parallel with accrual of patients to the aggressive lymphoma and CLL arms."

MB-106 data presented earlier this month at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress continue to demonstrate high efficacy and a very favorable safety profile across all five dose levels. The overall response ("ORR") was 96% across all dose levels and all indications (n=26). In particular, the 100% complete response rates by PET scan of patients with WM (n=2) as well as of patients with B-NHL previously treated with CD19-directed CAR T cell therapy (n=2) underscore the potential for MB-106 to treat these patient populations with high unmet needs. Durable responses were observed in a wide range of hematologic malignancies including follicular lymphoma, CLL, diffuse large B-cell lymphoma and WM. The possible outpatient administration of this therapy makes it potentially even more compelling. Currently no CAR T therapy is specifically approved for WM.

Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About Waldenstrom Macroglobulinemia
Waldenstrom macroglobulinemia ("WM"), also known as lymphoplasmacytic lymphoma, is a rare type of non-Hodgkin lymphoma ("NHL"), a malignant disorder of the bone marrow and lymphatic tissues. The proliferation of cancer cells can crowd out normal cells in these tissues, leading to low levels of red blood cells, white blood cells, and platelets which, in turn, causes fatigue, shortness of breath, infections, bruising, and bleeding. In addition, the cancer cells make large amounts of the large antibody protein immunoglobulin M, or IgM, which cause the blood to become thick. This hyperviscosity of the blood affects its flow through the smaller blood vessels, leading to some of the other manifestations of the disease, such as visual and neurological symptoms. WM is a rare disorder with an incidence of approximately 3 per million people per year, and 1,400 new cases are diagnosed in the U.S. each year. The median age at diagnosis is 70 years.

About MB-106 (CD20-targeted autologous CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and was exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody. MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the multicenter Phase 1/2 clinical trial that is now open to enrollment under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on these trials can be found at View Source using the identifier NCT05360238 for the Mustang multicenter trial and NCT03277729 for the ongoing trial at Fred Hutch.

On June 22, 2022 ProMIS Neurosciences Inc. (TSX: PMN) (OTCQB: ARFXF) ("ProMIS" or the "Company"), a biotechnology company focused on the discovery and development of antibody therapeutics targeting misfolded proteins such as toxic oligomers, implicated in the development of neurodegenerative diseases, reported that it has amended its articles to authorize the issuance of 70,000,000 Series 1 Preferred Shares ("Series 1 Shares") which it intends to use in settlement of its outstanding debentures (Press release, ProMIS Neurosciences, JUN 22, 2022, View Source [SID1234616182]).

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Holders of Series 1 Shares will be entitled to participate, on an as-converted basis, in dividend payments only if the Company declares, pays or sets aside any dividends on shares of any other class or series of capital stock. The Series 1 Shares are not subject to mandatory redemption or other redemption provisions and do not confer any voting rights or privileges to the holders. Holders of the Series 1 Shares shall be entitled to a liquidation preference in an amount per share equal to US$0.10.

Series 1 Shares are convertible at the option of the holder, in a 1:1 ratio, into the Company’s Common Shares ("Common Shares"). All outstanding Series 1 Shares shall automatically convert into Common Shares, at the effective conversion rate, upon the closing of one or more sales of equity securities resulting in at least US$30 million of gross proceeds to the Company.

On June 22, 2022 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for VBI-1901, a bivalent gB/pp65 immunotherapeutic vaccine candidate for the treatment of glioblastoma (GBM) (Press release, VBI Vaccines, JUN 22, 2022, View Source [SID1234616181]). In June 2021, the FDA also granted Fast Track Designation for VBI-1901 for the treatment of recurrent GBM in patients with first tumor recurrence.

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"This orphan drug designation is another significant milestone for our VBI-1901 program, and it underscores the urgency of our effort to develop meaningful new treatment options for patients with this devastating cancer," said Jeff Baxter, President and CEO of VBI. "As recently presented at ASCO (Free ASCO Whitepaper), we continue to see strong tumor response data and improvements in overall survival data compared to historical controls in the Phase 2a study of VBI-1901. With this orphan drug status, we look forward to working closely with the FDA and clinical investigators to build on that data, advancing the potential of this program to be a valuable part of the fight against GBM."

Though classified as a rare disease, GBM is the most common primary brain cancer with approximately 14,000 new cases diagnosed in the United States each year, and a low median overall survival of 15-18 months after diagnosis of primary GBM.1 Glioblastomas are stage IV brain tumors – they are an exceptionally aggressive form of brain cancer, with high recurrence rates and a five-year survival rate around 10%.2 Standard of care in the frontline setting includes surgical resection, chemotherapy, and radiation therapy. There is no effective standard of care in the recurrent setting – median overall survival in this patient population is approximately eight months.3

About FDA Orphan Drug Designation

The FDA’s Office of Orphan Products Development grants orphan drug designation to investigational drugs and biologics intended to prevent, diagnose, or treat rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified clinical trials, exemptions from certain FDA fees, and the potential for seven years of post-approval marketing exclusivity.

About FDA Fast Track Designation

The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to: 1) treat serious or life-threatening conditions, and 2) demonstrate the potential to address unmet medical needs. A therapeutic that receives Fast Track Designation is eligible for some or all of the following: 1) more frequent meetings with FDA to discuss the development plan and data needed to support approval, 2) more frequent written communication from FDA relating to the design of the proposed clinical trials and use of biomarkers, 3) Accelerated Approval and Priority Review, if relevant criteria are met, and 4) Rolling Review, which means the company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, instead of waiting until all sections of the application are completed.

Fast Track Designation was granted to VBI-1901, adjuvanted with granulocyte macrophage colony-stimulating factor (GM-CSF), for the treatment of first-recurrent GBM in June 2021.

About VBI-1901 and GBM

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 14,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

To learn more about VBI’s ongoing Phase 1/2a study and the INSIGhT trial, visit clinicaltrials.gov (Respective Identifiers: NCT03382977 and NCT02977780).