Case-Coulter Translational Research Partnership awards $1.1 million in funding and support for promising biomedical engineering university technologies

On December 1, 2020 The Case-Coulter Translational Research Partnership (CCTRP) reported that it has announced more than $1.1 million in funding and other support for six biomedical technologies (Press release, Case Western Reserve University, DEC 1, 2020, View Source [SID1234572021]).

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The six Case Western Reserve projects were selected for full program funding, which ranges from $50,000 to $200,000 each. Several additional pilot projects have or will be awarded funding by the end of the year. All projects are partnerships between a clinician and a biomedical engineer, and are focused on solving areas of unmet healthcare needs.

The 14-year-old program—a collaboration between Case Western Reserve University and the Wallace H. Coulter Foundation—provides direct funding and support services to help campus research teams advance products from their laboratories to the marketplace, where they can improve patient care.

Funding supports steps involved in preparing projects for potential commercialization, such as demonstrating technical feasibility, analyzing the business opportunity and assessing market feasibility. Projects must have the potential to advance to a commercial entity within 12 to 30 months.

More than two-dozen companies have emerged from the partnership’s support, and licensing arrangements have enabled three-dozen technologies to reach patients. For each dollar the partnership has invested, the projects have garnered an additional $25 of investment.

"The Case-Coulter Translational Research Partnership continues to be a cornerstone, filling an essential gap to transition university biomedical technologies from research to products, where they can significantly improve the health of our society," said Robert Kirsch, the Allen H. and Constance T. Ford Professor and chair of the university’s Department of Biomedical Engineering.

"As a group, the quality of the evaluated technologies continues to improve each year, demonstrating the robustness of the biomedical research-based technology pipeline," said Stephen Fening, CCTRP director. "We had many more proposals that were deserving of inclusion into the program than we were able to accommodate, making the selection process more challenging than ever."

The six projects selected and their inventors are:

10-liter Scale Production of BG34-200 Immunotherapeutic Under cGMP Guidelines
Mei Zhang, assistant professor of biomedical engineering, and Alex Huang, professor of pediatrics and pathology.

A significant portion of patients with solid tumor cancers do not respond to immunotherapies due to a lack of T-cell-inflamed tumor microenvironment. This novel plant-derived non-toxic BG34-200 molecule can be intravenously injected to modulate macrophages and create a tumor microenvironment that is vital for the generation of antitumor T-cell responses. The team is launching a clinical trial targeting canine metastatic osteosarcoma to collect key and gap data in preparation for a first human clinical trial targeting pediatric osteosarcoma.

Drug-Free Targeted Prostate Cancer Treatment with TNT – Targeted Nanobubble Therapy
Agata Exner, professor of radiology and biomedical engineering, Jim Basilion, professor of radiology and biomedical engineering, and Lee Ponsky, professor of urology

Drug-free, low-toxicity prostate cancer treatment using nanobubbles (NBs) are targeted to the prostate specific membrane antigen (PSMA) biomarker overexpressed on prostate tumor cells. The nanobubbles are injected into the bloodstream and specifically seek out only the cancer cells. Once inside the target cell, the NBs remain trapped and can be excited with an ultrasound pulse. Exposure to ultrasound results in collapse of the bubbles, leading to a highly focused mechanical disruption of the cancer cells and cell death. The approach, which we call TNT (targeted nanobubble therapy) can fit into the existing clinical work-flow and can be carried out with standard clinical ultrasound equipment. TNT can treat tumors without severe side effects, as it will be effective only when NBs are sonicated and will destroy only the cancer cells and not the surrounding healthy cells.

TraumaChek: A Field-deployable Dielectric Coagulometer for Comprehensive Assessment of Trauma-induced Coagulopathy
Anirban Sen Gupta, professor of biomedical engineering, Pedram Mohseni, professor of electrical engineering and biomedical engineering, and Sanjay Ahuja, professor of pediatrics

TraumaChek is a multichannel, handheld blood-coagulation analyzer for early, rapid and comprehensive assessment of trauma-induced coagulopathy. It is designed to guide hemorrhage control, transfusion and resuscitative management of trauma at the point-of-injury by first responders and at the point-of-care by hospital clinicians.

Minimally Invasive Interfascicular Nerve Stimulation (MiiNS) System for Chronic Pain Management
Dustin Tyler, the Kent H. Smith Professor of Biomedical Engineering, Emily Graczyk, research assistant professor of biomedical engineering, and Jennifer Sweet, professor of neurological surgery

This is a drug-free technology to provide targeted, comfortable, worry-free relief to people suffering from long-term pain. The discomfort and emotional stress from pain affects a person’s activity, sleep and ability to live a healthy life, leading to other serious health problems. Their Minimally Invasive Interfascicular Nerve Stimulation (MiiNS) technology provides a targeted, personally customized and comfortable treatment without side effects, addiction or surgical procedures. MiiNS can be implanted by a doctor during a simple office visit to provide long-lasting pain relief.

BAFF CAR-NK Cells for Therapy of B Cell Malignancies
Reshmi Parameswaran, assistant professor of medicine, and Pallavi Tiwari, assistant professor of biomedical engineering

BAFF CAR-­NK cells can specifically kill B cell cancers in a very effective manner with minimum side effects. This is a potential curative therapeutic strategy for patients who are not responding to current treatment methods.

PhotoSorb: Safe and Long-lasting Sunscreen
Vijay Krishna, assistant staff in Cleveland Clinic’s Department of Biomedical Engineering, and Edward Maytin, staff in Cleveland Clinic’s Department of Dermatology

Every year, more than a million new cases of skin cancer, including melanoma, are diagnosed in the United States. The primary cause is exposure to ultraviolet radiation (UV) from sunlight. Sunscreens can block UV, but increasing concerns about the health and environmental risks of chemical sunscreens now on the market underscores an urgent need for safer, more effective alternatives. A team from biomedical engineering and dermatology at Cleveland Clinic is developing a novel sunscreen (PhotoSorb) that appears to be safer and more stable than current sunscreens, and also has the potential to actually prevent skin cancers.

Regulus Therapeutics Announces Private Placement of Equity

On December 1, 2020 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that it has entered into a definitive securities purchase agreement in connection with a private placement to existing institutional and other accredited investors as well as new institutional investors (Press release, Regulus, DEC 1, 2020, View Source [SID1234572020]). Upon the closing of the financing, which is anticipated to occur on December 3, 2020, the Company expects to receive gross proceeds of approximately $19.4 million, not including any proceeds that may be received upon exercise of warrants. The closing of the financing is subject to customary closing conditions. H.C. Wainwright & Co. is acting as the exclusive placement agent for the financing.

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Under the securities purchase agreement, the investors have agreed to purchase approximately 24.3 million shares of the Company’s Common Stock ("Common Stock") and accompanying warrants to purchase up to an aggregate of approximately 18.2 million shares of Common Stock, at a combined purchase price of $0.7158 per share and accompanying warrant to purchase 0.75 of a share of Common Stock, except that the price per share for executive officers and directors participating as investors in the financing is $0.7239 per share and accompanying warrant to purchase 0.75 of a share of Common Stock. Certain investors have also agreed to purchase, in lieu of shares of Common Stock, an aggregate of approximately 272,970 shares of non-voting Class A-3 convertible preferred stock at a price of $6.22 per share, and accompanying warrants to purchase an aggregate of up to approximately 2.0 million shares of Common Stock at a price of $0.125 for each share of Common Stock underlying the warrants. Each share of non-voting Class A-3 convertible preferred stock will be convertible into 10 shares of Common Stock, subject to certain beneficial ownership conversion limitations. The warrants will be exercisable for a period of five years following the date of issuance and will have an exercise price of $0.7464 per share, subject to proportional adjustments in the event of stock splits or combinations or similar events. The closing is expected to occur on December 3, 2020, subject to customary closing conditions.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state.

Sierra Oncology to Host KOL Panel Event to Discuss Updated Phase 3 Myelofibrosis Data Presented at ASH

On December 1, 2020 Sierra Oncology, Inc. (SRRA), a late-stage biopharmaceutical company on a quest to deliver targeted therapies that treat rare forms of cancer, reported it will host an analyst and investor event on Wednesday, December 16, 2020 at 10:00 am ET (Press release, Sierra Oncology, DEC 1, 2020, View Source [SID1234572014]). The event will feature three leading myelofibrosis experts:

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Jean-Jacques Kiladjian, MD, PhD, Saint-Louis Hospital; Paris Diderot University
Ruben Mesa, MD, Director of the Mays Cancer Center, home to UT Health San Antonio, MD Anderson Cancer Center
Srdan Verstovsek, MD, PhD, University of Texas; MD Anderson Cancer Center
The call will include an overview of momelotinib data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, a panel discussion moderated by Barbara Klencke, MD, Chief Development Officer of Sierra Oncology, and an open question & answer session with attendees.

Analyst & Investor Event and Webcast Information

Date and Time: Wednesday, December 16, 2020, 10:00 am ET

To register, please click here.

The presentation will be webcast live, and an archive of the presentation will be accessible after the event through the Sierra Oncology website: www.SierraOncology.com.

About Momelotinib

Momelotinib is a selective and orally bioavailable JAK1, JAK2 and ACVR1 inhibitor currently under investigation for the treatment of myelofibrosis. Myelofibrosis results from dysregulated JAK-STAT signaling and is driven by constitutional symptoms, splenomegaly (enlarged spleen) and progressive anemia.

Momelotinib is currently under investigation in the MOMENTUM clinical trial, a global, randomized, double-blind Phase 3 study for symptomatic and anemic myelofibrosis patients. Top-line data are anticipated in H1 2022. The U.S. Food & Drug Administration has granted Fast Track designation for momelotinib.

Ipsen highlights new strategic priorities and provides mid-term financial outlook

On December 1, 2020 Ipsen (Euronext: IPN; ADR: IPSEY), a specialty-care focused biopharmaceutical group, reported that it will host a virtual Capital Markets Day to highlight the Group’s new strategic priorities with the aim of driving continued growth and bringing transformative medicines to patients (Press release, Ipsen, DEC 1, 2020, View Source [SID1234572012]).

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David Loew, Chief Executive Officer of Ipsen stated: "Our new Group strategy positions Ipsen for long-term success by focusing together for patients and society. We will reinforce our commitment to Oncology, Rare Disease and Neuroscience by strengthening and accelerating our external innovation efforts and pipeline in clearly-defined segments. Through prioritization and collaboration, we will drive efficiencies to support investment in our pipeline. We are building on a strong foundation of engaged employees, agile development capabilities and global commercial footprint. I am energized to execute on our key strategic priorities to create long-term value for all stakeholders."

Bring the full potential of innovative medicines to patients

Ipsen is focused on maximizing the value of its current Specialty Care product portfolio through commercial excellence and geographic expansion. The Group aims to maximize its core brands Somatuline (lanreotide), Decapeptyl (triptoreline) and Dysport (botulinum toxin type A) and capture the full potential of its innovative oncology products Cabometyx (cabozantinib) and Onivyde (irinotecan liposome injection). If approved, the launch of palovarotene will be a key milestone to bring this medicine to patients with FOP and strengthen Ipsen’s presence in Rare Disease.

A strategic review of the Consumer Healthcare business is proceeding.

Build a high-value sustainable pipeline

Ipsen’s priority is to build a sustainable pipeline to drive long-term growth. Recent initiatives have prioritized the pipeline on the highest potential opportunities and progressed the transformation of the R&D organization. Ipsen is strengthening its external innovation efforts by targeting differentiated medicines in its three core therapeutics areas of Oncology, Rare Disease and Neuroscience, with a broader disease and modality scope than previously defined, and across all stages of clinical development.

Deliver efficiencies to enable targeted investment & growth

Ipsen is committed to generating efficiencies through a focused and agile operating model. Leveraging smart spending, streamlined operations, manufacturing efficiencies and optimizing digitalization, the Group will be able to reinvest in R&D and external innovation to fuel future growth.

Boost culture of collaboration & excellence

Patients and society are at the core of Ipsen’s mission, starting with fully engaged employees and a culture of accountability to perform and compete in the long-term. Ipsen is highly committed to its corporate social responsibility (CSR) initiatives which are centered around employees, community and the environment, as reflected throughout the organization and in the inclusion of responsibility metrics in management compensation.

Mid-term financial outlook and capital allocation strategy

Ipsen provides its financial outlook for the period 2020-20241:

Group Net Sales CAGR2 between +2% and +5%3,assuming potential additional indications
Commitment to invest in R&D supported by SG&A efficiencies
Lower SG&A as a percentage of net sales driven by further focus and optimization
Higher R&D as a percentage of net sales driven by external innovation strategy
External innovation is Ipsen’s number one priority for capital allocation. In support of its external innovation strategy, Ipsen expects to generate by 2024 a cumulative €3bn4 of firepower for pipeline expansion, excluding the sale of any assets.

Webcast

Ipsen will host a video webcast of the Capital Markets Day on Tuesday, 1 December 2020 from 2:00 p.m. to 5:00 p.m. CET (GMT+1) available at www.ipsen.com. Participants should log in to the webcast approximately 5 to 10 minutes prior to its start. No reservation is required to participate in the conference call.

Webcast link: View Source

Please note this event will be streamed live. No dial-in number available.

A recording will be available for 14 days on Ipsen’s website.

1 Ipsen is on track to deliver its previous 2022 financial targets and is committed now to a new 2024 financial outlook

2 Compound Annual Growth Rate

3 At constant exchange rates and scope

4 Based on Net Debt remaining below 2.0x EBITDA

Nordic Nanovector completes patient enrolment into Phase 1 trial of Betalutin® in Diffuse Large B Cell Lymphoma

On December 1, 2020 Nordic Nanovector ASA (OSE: NANO) reported that it has completed enrolment into the LYMRIT 37-05 Phase 1 clinical trial of Betalutin (177Lu lilotomab satetraxetan) in patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) not eligible for autologous stem cell transplantation (ASCT) (Press release, Nordic Nanovector, DEC 1, 2020, View Source [SID1234571999]).

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Eighteen DLBCL patients were enrolled into the trial at clinical trial sites in the US and Europe and were dosed with three escalating treatment doses of Betalutin (10MBq/kg, 15MBq/kg and 20MBq/kg). A preliminary data readout is expected in H1’2021.

As announced in April 2020, LYMRIT 37-05 will be paused pending analysis of these data, which is expected to inform plans for the further development of Betalutin in R/R DLBCL.

Nordic Nanovector’s primary focus is the timely completion of the pivotal Phase 2b PARADIGME trial of Betalutin in 3rd-line follicular lymphoma (3L FL).

Christine Wilkinson Blanc, Chief Medical Officer of Nordic Nanovector, said: "The completion of recruitment into this dose-finding study in patients with DLBCL is an important milestone. DLBCL remains a significant indication with a large unmet medical need. The data analysis from this trial will form the basis of our considerations for the further development of Betalutin in DLBCL and more broadly across non-Hodgkin’s lymphoma."

The LYMRIT 37-05 study is a Phase 1 open-label, single-arm, dose-escalation study in DLBCL designed to determine the dose to be recommended for further studies in DLBCL and assess the safety, tolerability, pharmacokinetic profile and preliminary anti-tumour activity of a single administration of Betalutin. More information on this study can be found at www.clinicaltrials.gov (NCT02658968).

DLBCL is an aggressive form of non-Hodgkin’s Lymphoma (NHL) that accounts for 30% of all NHL cases1,2. The number of diagnosed incident cases of DLBCL in the seven major markets (US, key five European markets and Japan) was 64,172 in 2018 and is expected to grow to 74,927 in 20283.

Approximately 40% of DLBCL patients relapse after first-line combination treatment with rituximab and chemotherapy. These patients have few therapeutic options, with high-dose chemotherapy and autologous stem cell transplant (ASCT) achieving long-term remissions in only a minority of patients4. Relapsed DLBCL therefore remains a serious unmet medical need.

References

Siegel R, Miller K and Jemal A. Cancer Statistics, 2019. CA Cancer J. Clin. 2019;69(1):7-34
View Source
Non-Hodgkin’s Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL), 2020, Decision Resources Group, Clarivate
Liu Y, Barta SK. Diffuse large B-cell lymphoma: 2019 update on diagnosis, risk stratification, and treatment. Am J Hematol. 2019 May;94(5):604-616.