On February 9, 2022 Global pharma major, Lupin Limited (Lupin) reported that it hasentered into a distribution agreement with Medis for Lupin’sorphan drug NaMuscla (mexiletine) (Press release, Lupin, FEB 9, 2022, View Source [SID1234607945]). Medis will commercialize NaMuscla for the symptomatic treatment of myotonia in adults with non-dystrophic myotonic (NDM) disorders in Central and Eastern European countries.NaMuscla is the first and only licensed product for this indication .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

NDM disorders are a group of rare, inherited neuromuscular disorders which is characterized by the inability to relax muscles following voluntary contraction. NaMuscla reduces myotonia symptoms in people with NDM, resulting in a significant improvement in quality of life and other functional and clinical outcomes for patients1. NaMuscla, which has been designated orphan drug status, received EU marketing authorization in December 20182.

Under the agreement announced today, Medis will initially focus on the commercialization of NaMuscla in the Central and East European countries, namely Croatia, Czech Republic, Hungary, Slovakia, and Slovenia in the first phase. Lupin will continue commercialization of NaMuscla in Germany, France, and the UK.

"The distribution agreement representsan important milestone for Lupin as we continue the roll out of NaMuscla across Europe. We know that collaborating with partners which are highly focused in their territories means patients receive medicines in the most efficient way," said Thierry Volle, President EMEA, Lupin.

"At Medis, we are very excited to partner with Lupin and are further committed to using our expertise in comprehensive commercialisation to provide new, innovative treatment options like NaMuscla that address patients’ unmet needs. For us, each patient counts," said Martina Perharič, CEO of Medis. "As a pioneer in full-service pharmaceutical distribution for the CEE region, we have gained extensive knowledge of the complex markets in the region. This allows us to launch NaMuscla quickly and effectively in selected countries and provide excellent support to our partner Lupin."

Today, around 1,000 people in Central and Eastern Europe living with NDM have limited access to a licensed treatment for myotonia that can reduce the daily burden of this disabling, lifelong symptom3-5. Limited access leads to inconsistent medication supply, administrative challenges, and associated financial burdens. Coupled with low awareness and limited clinical experience among healthcare professionals due to the rare nature of the disease, may result in significant harm to patients4.

Lupin has recruited the first study participants in a pediatric trial as part of the pediatric investigation plan for NaMuscla in children (NCT04624750) and a post-authorization study to address long-term safety and treatment effects on patient-reported outcomes in adults (NCT04616807).

Notes for Editors

About Myotonic Disorders and Non-Dystrophic Myotonias (NDM)

Myotonic disorders are a group of heterogeneous, inherited, neuromuscular disorders characterized by a shared symptom called myotonia. Myotonia can be described as an inability to relax a contraction of skeletal muscle which originates from a voluntary muscular contraction such as shaking someone’s hand and blinking, or everyday activities such as walking across a street and climbing stairs.

Non-dystrophic myotonias (NDM) are a sub-set of rare (prevalence of 1:100,0003), inherited, myotonic disorders which are caused by mutations within ion channels in the sarcolemma membrane of skeletal muscles. Non-dystrophic myotonias exhibit both sodium and chloride channelopathies which result in altered membrane excitability6. For patients with NDM, myotonia is the most prominent symptom and demonstrates different phenotypes in subgroups of NDM disorders, and can affect different parts of the body, such as legs, arms, or facial muscles, more severely6.

Myotonia in NDM patients has an onset in childhood and persists across their lifetime. Patients perceive that myotonia increases in severity over time, impacting daily life. Myotonia is described by patients in a variety of ways (stiffness, cramps, pain, difficulty releasing a fist, or difficulty swallowing or eating) which can contribute to substantial delays in diagnosis and treatment, leading to decreased patient quality-of-life and often significant disability4, 7.

About NaMuscla (mexiletine)

NaMuscla is the first and only antimyotonic agent licensed to treat symptomatic myotonia in adults with non-dystrophic myotonic disorders in Europe8. In randomized controlled trials, NaMuscla (167 to 500 mg/day) has been shown to significantly reduce myotonia compared to placebo, reducing skeletal muscle hyperexcitability through its use-dependent, voltage-gated, sodium channel blocking actions which are independent of the cause of channel function. This resulted in an improvement in patient quality-of-life and other functional outcomes, with gastro-intestinal discomfort reported as the most common adverse event, demonstrating NaMuscla to be safe and well tolerated1, 2, 8.

On February 9, 2022 Checkmate Pharmaceuticals, Inc. (Nasdaq: CMPI) ("Checkmate"), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, reported the appointment of Alan Bash as President and Chief Executive Officer, effective March 1, 2022 (Press release, Checkmate Pharmaceuticals, FEB 9, 2022, View Source [SID1234607897]). Mr. Bash, who will also join the Board of Directors, will succeed Alan Fuhrman, who has served as interim President and Chief Executive Officer since October 2021. Mr. Fuhrman will continue to serve Checkmate as a member of the Board of Directors.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"I am very pleased to welcome Alan Bash as President and CEO. With over two decades of leadership experience at Bristol Myers Squibb, Mr. Bash has extensive expertise translating innovative science into meaningful advances for patients, notably driving the successful launch and commercialization of Opdivo and Yervoy," said Mike Powell, Chairman of the Board of Directors. "Under Alan’s leadership, Checkmate will continue developing novel treatments for patients with difficult-to-treat cancers. The Company’s lead program vidutolimod, continues to progress in multiple clinical studies and towards registration in refractory melanoma. I want to thank Mr. Fuhrman for his outstanding stewardship as interim President and CEO over these past months."

"I am honored to be joining an experienced management team, an accomplished Board and a Company built on the potential to improve the lives of those living with cancer," said Mr. Bash. "Vidutolimod combines a powerful, unique immune activator, CpG-A DNA, with an innovative delivery platform, that has demonstrated potential best-in-class efficacy in certain patients whose tumors were non-responsive to approved immunotherapies."

Mr. Bash’s appointment follows the recent additions of Jon Wigginton, M.D. and Joy Yan, M.D., Ph.D. to the Checkmate Board of Directors. These appointments provide complementary leadership and immuno-oncology expertise and position Checkmate well to advance the development of vidutolimod and unlock its value potential.

Mr. Bash will join Checkmate from Bristol Myers Squibb ("BMS"), where he currently serves as Senior Vice President, Commercial Model Innovation, and prior to that, Senior Vice President, Commercial for Immunology and Fibrosis, partnering with Research & Development to advance early and late-stage assets in the integrated BMS and Celgene portfolios. He has a deep expertise in immuno-oncology, having led the U.S. launch of the PD-1 checkpoint inhibitor, Opdivo; serving as franchise lead for metastatic melanoma; and leading the launch of the first FDA-approved checkpoint combination. He also served as Worldwide Vice President for BMS’s lung cancer franchise and led the product team for a targeted treatment for head and neck cancer and colorectal cancer. He earned his BA from Georgetown University and his MBA from Columbia Business School.

On February 9, 2022 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that it will webcast its Quarterly Update Conference Call for the fourth quarter 2021 on Tuesday, February 22 at 4:30 p.m. ET / 1:30 p.m. PT. Dr. Helen Torley, president and chief executive officer, will lead the call. On the same date, Halozyme will release financial results for the fourth quarter and year ended December 31, 2021, following the close of trading (Press release, Halozyme, FEB 9, 2022, View Source [SID1234607913]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To register for this conference call, please use this link: View Source After registering, you will receive an email confirmation that includes dial in details and unique conference call codes for entry. Registration is open through the live call. However, to ensure you are connected for the full call, we suggest registering a day in advance or at minimum 10 minutes before the start of the call.

The call will be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit the Investor’s page of www.halozyme.com approximately 15 minutes prior to the call to register, download and install any necessary audio software. A telephone replay will be available for two weeks after the call by dialing (800) 770-2030 (domestic callers) or (647) 362-9199 (international callers) using replay ID number 44868.

On February 9, 2022 NANOBIOTIX (Euronext: NANO – NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported the publication of a peer-reviewed case study by researchers at The University of Texas MD Anderson Cancer Center in Clinical and Translational Radiation Oncology (Press release, Nanobiotix, FEB 9, 2022, View Source [SID1234607928]). The case study reports on the first patient experience of treatment with potential first-in-class radioenhancer, NBTXR3, in pancreatic ductal adenocarcinoma (PDAC; pancreatic cancer).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Given the nature of NBTXR3 as a therapeutic candidate with potentially broad applicability across solid tumor indications and therapeutic combinations, Nanobiotix and MD Anderson are collaborating to expand development of NBTXR3 beyond the Company’s priority pathways in locally advanced head and neck squamous cell carcinoma and immunotherapy. This ongoing phase I pancreatic cancer study is one of five active phase I or phase II studies currently being conducted as part of the collaboration.

The phase I clinical trial is designed to determine the safety profile of NBTXR3 activated by radiotherapy (RT) for patients with locally advanced or borderline resectable PDAC, and the recommended phase II dose (RP2D) for future efficacy evaluation. The case study in Clinical and Translational Radiation Oncology reports on the first patient ever to receive local endoscopic delivery of NBTXR3 to a deep visceral tumor. The patient is a 66-year-old male with unresectable, locally advanced PDAC who received local endoscopic delivery of NBTXR3 followed by intensity modulated RT. CT imaging demonstrated no visible leakage of the radioenhancer outside of the injected tumor. At initial follow-up evaluation, the lesion remained radiographically-stable, the patient did not demonstrate treatment-related toxicity, and the report concluded that the treatment was feasible.

PDAC is one of the leading causes of cancer-related death in the world today. For patients with PDAC who are not eligible for surgery, RT has been shown to improve local disease control. However, safely delivering therapeutic doses of radiation remains challenging due to off-target toxicities in surrounding healthy tissues. While systemic radiosensitizers have been evaluated in this indication, these agents increase the sensitivity of both tumor tissues and healthy tissues. In contrast, due to the potential of NBTXR3 to increase the dose of RT within the tumor without increasing the dose in surrounding healthy tissues, the radioenhancer is being evaluated as a new option to address this urgent unmet need.

"There is a critical need to develop an effective therapy to improve treatment outcomes while mitigating off-target toxicities for patients with locally advanced pancreatic cancer," said Eugene Koay, MD, PhD, associate professor of Radiation Oncology at MD Anderson. "The initial feasibility for NBTXR3 injection outlined in this case study provides an important step forward as we determine the recommended phase II dose and the potential for the radioenhancer to help patients with this challenging disease."

Nanobiotix expects to establish the recommended phase II dose for NBTXR3 in pancreatic cancer in 2022.

About NBTXR3
NBTXR3 is a novel, potentially first-in-class oncology product, composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. The product candidate’s physics-based mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly, with immune checkpoint inhibitors.

NBTXR3 is being evaluated in locally advanced head and neck squamous cell carcinoma (HNSCC) as the primary development pathway. The company-sponsored phase I dose escalation and dose expansion study has produced favorable safety data and early signs of efficacy. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy.

Nanobiotix has also prioritized an Immuno-Oncology development program—beginning with a Company sponsored phase I clinical study, evaluating NBTXR3 activated by radiotherapy in combination with anti-PD-1 checkpoint inhibitors for patients with locoregional recurrent or recurrent/metastatic HNSCC and for patients with lung or liver metastases from any primary cancer eligible for anti-PD-1 therapy, either naïve or resistant to prior PD-1 (either primary or secondary as per SITC (Free SITC Whitepaper) criteria).

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in strategic collaborations to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several phase I and phase II studies to evaluate NBTXR3 across tumor types and therapeutic combinations. In 2021, the Company entered into an additional strategic collaboration agreement with LianBio to support its global phase III study in Asia along with four future registrational studies.

On February 9, 2022 Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease with three ongoing Phase 1 clinical trials, reported that company management will participate in the 11th Annual SVB Leerink Global Healthcare Conference being held February 14-18, 2022 (Press release, Shattuck Labs, FEB 9, 2022, View Source [SID1234607946]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Presentation Details
Conference: 11th Annual SVB Leerink Global Healthcare Conference
Format: Corporate Presentation
Presenter: Taylor Schreiber, M.D., Ph.D., Shattuck’s Chief Executive Officer
Date: February 18, 2022
Time: 10:40 a.m. EST

A live webcast of the presentation will be available on the Events & Presentations section of the Company’s website. A replay of the webcasts will be archived for up to 90 days following the presentation date.