Corporate Presentation dated September 13, 2021

On September 13, 2021, Moleculin Biotech, Inc. Presented the Corporate presentation (Press release, Moleculin, SEP 13, 2021, View Source [SID1234587664]).

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HUTCHMED Receives Breakthrough Therapy Designation in China for Amdizalisib (HMPL-689) for Treatment of Relapsed or Refractory Follicular Lymphoma

On September 13, 2021 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM: HCM; HKEX: 13) reported that the Center for Drug Evaluation of China’s National Medical Products Administration ("NMPA") has granted Breakthrough Therapy Designation ("BTD") to amdizalisib (HMPL-689), a highly selective and potent PI3Kδ inhibitor, for the treatment of relapsed or refractory follicular lymphoma ("FL"), a subtype of non-Hodgkin’s lymphoma ("NHL") (Press release, Hutchison China MediTech, SEP 13, 2021, View Source [SID1234590538]).

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NMPA grants BTD to new drugs that treat life-threatening diseases or serious conditions for which there are no effective treatment options, and where clinical evidence demonstrates significant advantages over existing therapies. Drug candidates with BTD may be considered for conditional approval and priority review when submitting a New Drug Application (NDA). This indicates that the development and review of amdizalisib for relapsed or refractory FL may be expedited, to address patients’ unmet needs more quickly.

Christian Hogg, CEO of HUTCHMED, said, "The granting of BTD to amdizalisib by the NMPA underscores the promising clinical value of this highly selective and potent PI3Kδ inhibitor. There is a clear need for new therapies in this treatment setting, particularly with regard to specific toxicities and suboptimal efficacy with existing treatments across different lymphoma subtypes. We look forward to important clinical data on amdizalisib being presented at the ESMO (Free ESMO Whitepaper) Congress next week and are continuing to accelerate global development of this novel therapy."

Updated preliminary results from the ongoing Phase Ib expansion study in China will be presented as a Proffered Paper at the 2021 ESMO (Free ESMO Whitepaper) (European Society for Medical Oncology) Congress on September 20, 2021. To date, amdizalisib has been shown to be well tolerated, exhibiting dose-proportional pharmacokinetics ("PK"), a manageable toxicity profile, and single-agent clinical activity in relapsed/refractory B-cell lymphoma patients. Additional details may be found at clinicaltrials.gov, using identifier NCT03128164.

HUTCHMED has initiated an extensive, globally-focused clinical development pathway for amdizalisib. In April 2021, HUTCHMED initiated a Phase II registration study in China for amdizalisib in approximately 100 patients with relapsed or refractory FL and approximately 80 patients with marginal zone lymphoma ("MZL"). The trial is being conducted in over 35 sites in China. Additional details may be found at clinicaltrials.gov, using identifier NCT04849351.

Amdizalisib is also being evaluated in an ongoing Phase I/Ib study in the U.S. and Europe in patients with relapsed or refractory NHL (NCT03786926).

About PI3Kδ and NHL
PI3Kδ (phosphoinositide 3-kinase delta) is a lipid kinase that controls the activation of several important signaling proteins. Upon an antigen binding to B-cell receptors, PI3Kδ can be activated through the Lyn and Syk signaling cascade. The abnormal activation of B-cell receptor signaling is closely related to the development of B-cell type hematological cancers, which represent approximately 85% of all NHL cases. Therefore, PI3Kδ is considered a promising target for drugs that aim to treat certain hematologic cancers.

FL accounts for approximately 17% of NHL. In 2020, there were an estimated 16,000 and 13,000 new cases of FL in China and the U.S., respectively[i],[ii],[iii]. Patients with relapsed or refractory FL do not have curative treatment options and have a high unmet need for optimal therapeutic options.

About Amdizalisib
Amdizalisib (HMPL-689) is a novel, selective and potent oral inhibitor targeting the isoform PI3Kδ. Amdizalisib’s PK properties are favorable with good oral absorption, moderate tissue distribution and low clearance in preclinical PK studies, suggesting a low risk of drug accumulation and drug-to-drug interaction. Because of its high target selectivity and optimal PK profile, amdizalisib has the potential to demonstrate an optimal benefit-risk profile in this class.

HUTCHMED currently retains all rights to amdizalisib worldwide.

The World Health Organization grants the name ecubectedin to PharmaMar’s anti-tumor compound PM14

On September 13, 2021 PharmaMar (MSE:PHM) reported that the World Health Organization (WHO) has confirmed the International Nonproprietary Name (INN) of its investigational anti-tumor compound PM14 as
ecubectedin (Press release, PharmaMar, SEP 13, 2021, View Source [SID1234596743]).

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The WHO has published a list of recommended INNs on its website, including this new name for PharmaMar’s active ingredient, which means that it can now be used officially.

Ectubectedin is currently in Phase I-II clinical development, both as a single agent and in combination with other drugs for the treatment of solid tumors. The fourongoing clinical trials with this compound are being conducted in 12 hospitals in Spain, France, the United Kingdom and the United States.

Ecubectedin is a compound that acts by specifically inhibiting RNA synthesis and active transcription of protein-coding genes.

Y-mAbs Announces Priority Review of BLA for DANYELZA® (naxitamab-gqgk) in China

On September 13, 2021 Y-mAbs Therapeutics, Inc. ("Y-mAbs" or the "Company"), NASDAQ: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that its partner SciClone Pharmaceuticals (Holdings) Limited ("SciClone Pharmaceuticals") has been granted priority review of the Biologics License Application ("BLA") for DANYELZA (naxitamab-gqgk) for the treatment of patients with relapsed/refractory high-risk neuroblastoma by the Center for Drug Evaluation ("CDE") of China’s National Medical Products Administration ("NMPA") (Press release, Y-mAbs Therapeutics, SEP 13, 2021, View Source [SID1234587598]).

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"We are pleased to see SciClone Pharmaceuticals receive priority review of the DANYELZA BLA in China. Priority review in China is intended to expedite review and approval, which means that we could potentially be facing approval and launch of DANYELZA in China as soon as the first quarter of 2022," commented Thomas Gad, founder, Chairman and President at Y-mAbs.

Dr. Claus Moller, Chief Executive Officer, continued, "DANYELZA was recently prescribed for the first time in China, and we were excited to learn that the first patient had received treatment at the Lecheng Branch of Hainan Women and Children’s Medical Center in Hainan Boao Lecheng International Medical Tourism Pilot Zone."

Researchers at MSK developed DANYELZA, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the product.

About DANYELZA (naxitamab-gqgk)

DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

According to New Multinational Survey, Healthcare Providers Believe Immunotherapy Has Potential to Positively Impact Earlier-Stage Cancer Treatment Landscape Across Tumor Types

On September 13, 2021 Bristol-Myers Squibb reported that Results of a new multinational survey of healthcare providers revealed that the majority of participants expect immunotherapy to have a positive impact on the treatment landscape for patients with earlier-stage cancers in the adjuvant (after surgery), neo-adjuvant (before surgery) and peri-operative (both before and after surgery) settings, if approved by regulatory bodies (Press release, Bristol-Myers Squibb, SEP 13, 2021, View Source [SID1234587616]). The survey, commissioned by Bristol Myers Squibb (NYSE: BMY), included over 250 oncologists, surgeons and specialists in the U.S., Japan, Germany, Italy and France who currently treat patients with stage I-III disease across eight different types of cancer. While healthcare providers surveyed are more satisfied with current treatments in cancers where earlier options are well established, they do not always use treatment before or after surgery, and the vast majority of respondents express enthusiasm for the potential of immunotherapy in earlier-stage cancers.

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"Cancer recurrence often marks the transition from curable to incurable disease and can be life-altering for patients, which is why we continually investigate ways to improve upon the standard of care," said Michele Maio, M.D., Ph.D., Director of Medical Oncology and Immunotherapy, University Hospital of Siena. "Optimizing cancer treatment in its early phases, before the disease returns or spreads, represents a significant opportunity and unmet need. Research with immunotherapy in these settings is growing, and the survey results announced today reinforce that a majority of healthcare providers surveyed are enthusiastic about its future potential."

Current Treatment Approaches in Earlier Stages of Cancer

Today, treatment in the neoadjuvant, adjuvant or peri-operative settings may consist of chemotherapy, radiation, targeted therapy, chemoradiation therapy, and increasingly in a subset of tumors, immunotherapy. The survey identified a number of trends on the current use, satisfaction and drivers of treatment choices in earlier-stage cancers.

HCPs surveyed do not always use treatment beyond surgery: The majority of HCPs surveyed report that they "sometimes" use neoadjuvant (62%), adjuvant (55%) or peri-operative (54%) treatments for patients with earlier stages of cancer, highlighting the opportunity for earlier intervention.
HCPs surveyed are more satisfied with current treatments in cancers where earlier options are well established :Six out of 10 or more survey respondents are "very" or "fairly" satisfied with current neoadjuvant (67%), adjuvant (70%) and peri-operative (61%) treatment options as a whole. However, satisfaction varies by tumor* and is highest among cancers with well-established therapies, like breast cancer (87% satisfaction in the neoadjuvant/adjuvant settings) and melanoma (77% satisfaction in adjuvant). In kidney and liver cancers, satisfaction is noticeably lower (less than 35% satisfaction with neoadjuvant, adjuvant and peri-operative options), signaling the need for additional research.
HCPs surveyed use immunotherapy in earlier stages (either as approved therapies or in clinical trials), but not as often as other treatments : Currently, respondents report more experience using chemotherapy (85%, 86% and 73% for neoadjuvant, adjuvant and peri-operative, respectively) than immunotherapy (48%, 65% and 39%, respectively), likely reflecting that immunotherapy remains under investigation in a number of tumor types and only recently emerged as an approved option in others.
The Potential of Immunotherapy for the Future of Earlier-Stage Treatment

To better understand the future landscape of neoadjuvant, adjuvant and peri-operative treatment, the survey explored HCPs’ perceptions of immunotherapy and found:

Many HCPs surveyed see potential for a positive impact with immunotherapy in earlier stages of disease: Participants see the greatest potential for positive outcomes in melanoma (92% report positive potential impact in the adjuvant setting), lung cancer (89% in the neoadjuvant setting) and bladder or urothelial cancer (84% in the adjuvant setting).*
HCPs surveyed believe the potential benefits of immunotherapy align with what currently drives treatment preferences in earlier settings : Selecting from a list, HCPs surveyed state the most important potential benefits of immunotherapy as longer overall survival (64%), increased disease-free, event-free or recurrence-free survival (57%) and maintenance of quality of life (54%). These responses align with the factors HCPs surveyed report as most important in making treatment decisions in patients with operable tumors (long-term survival, prevention of relapse or recurrence and quality of life).
HCPs surveyed cite the need for more data as a leading barrier to adoption of immunotherapy in earlier stages of cancer : From a list, surveyed participants selected the need for long-term and overall survival data as leading barriers to adoption of immunotherapy in earlier stages of cancer (53% and 50%, respectively), reinforcing the importance of ongoing research and follow-up analyses.
"Over the past decade, immunotherapy research has evolved, starting with a focus on metastatic cancers, and more recently, expanding to explore the role of these treatments in earlier stages of the disease," said Jonathan Cheng, senior vice president, head of Oncology Development, Bristol Myers Squibb. "We hope that by addressing cancer in earlier stages, when the immune system may be more responsive and intact, immunotherapy may have the potential to prevent recurrence and ultimately lead to patients living longer. Oncologists, surgeons and specialists who responded to this survey are similarly optimistic about the potential of bringing immunotherapy into earlier stages of cancer."

*Tumor-specific results are based on responses from HCPs who currently treat these types of cancer, a subset of the full sample.

About the Survey

On behalf of Bristol Myers Squibb, Ipsos MORI carried out an online survey on treatment perceptions and practices in earlier stages of cancer. A total of 256 healthcare providers across five countries (France n=50, Germany n=50, Italy n=50, U.S. n=56 and Japan n=50) chose to take part in the online survey. Fieldwork took place between June 3 and July 2, 2021. Respondents included medical oncologists, surgeons (general, thoracic, breast, respiratory and gastroenterological surgeons) and specialists (urologists, dermatologists, pulmonologists, gastroenterologists and otolaryngologists) who treat patients with stage I-III disease across eight different cancer types (bladder/urothelial cancer, breast cancer, gastroesophageal cancers, head and neck cancer, kidney cancer, liver cancer, lung cancer and melanoma). A quota was set to obtain a minimum of 25 medical oncologists in France (n=28), Germany (n=29), Italy (n=29) and U.S. (n=25). The sample included a mix of hospital-, university- and community-based HCPs. The respondents were sampled from pre-existing panels of self-selecting HCPs, managed by M3 and SHC.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.