On March 4, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported the publication of pre-clinical data highlighting the potential of its alternating 2-vector, intravenously administered cancer therapeutics in the peer-reviewed journal, Cell Reports Medicine (Press release, Hookipa Pharma, MAR 4, 2021, View Source [SID1234576053]). The publication, which is available online now, will appear in the 16 March print issue.

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"The pre-clinical data published in Cell Reports Medicine underscore the potential of our engineered arenavirus platform to redefine success in cancer immunotherapy. Specifically, our alternating 2-vector approach delivered a substantial tumor-specific response, resulting in tumor cures and long-term anti-tumor immunity in a pre-clinical setting," said Joern Aldag, Chief Executive Officer at HOOKIPA. "The data in this peer-reviewed publication provide the scientific substantiation for the ongoing clinical trial of the alternating 2-vector therapy for Human Papillomavirus 16-positive (HPV16+) cancers as well as for advancing to prostate cancer."

Pre-clinical data featured in the article showed that intravenous, alternating administration of two different replicating arenaviral vectors that express the same antigen induces potent T cell response, exceeding 50% of the circulating T cell pool, and robust anti-tumor activity. The anti-tumor activity and very high T cell generation were demonstrated both with onco-viral antigens and also with a cancer self-antigen, illustrating the ability of the arenaviral platform to break tolerance.

Other key highlights from the paper include:

Single-vector and alternating 2-vector therapy did not induce vector-neutralizing antibodies, supporting repeated intravenous administration
Mice that cleared tumors after therapy were protected from tumor re-challenge
Expanding on the data observed with single-vector therapy, alternating 2-vector therapy induced an even higher T cell response and more efficient tumor control
The study on which this publication is based, was conducted and led by an international group of researchers at the University of Basel.

HOOKIPA is evaluating its single-vector and alternating 2-vector technologies in the ongoing Phase 1/2 clinical trial of its lead oncology candidates, HB-201 and HB-202. HB-201 and HB-202 use the LCMV and PICV arenaviral backbones, respectively, while expressing the same antigen, an E7/E6 fusion protein derived from HPV16. Interim Phase 1 monotherapy data on HB-201 for the treatment of advanced HPV16+ cancers showed promising anti-tumor activity and favorable tolerability. Data demonstrated responses and stable disease in head and neck cancer patients who failed prior standard of care therapy, platinum therapy, PD(L)1 inhibitor, or both. Initial data on HB-201 and HB-202 as a replicating 2-vector therapy are anticipated by mid-2021. HOOKIPA’s HB-300 program for prostate cancer also uses the LCMV and PICV arenaviral backbones directed against three validated antigens for prostate cancer: PAP, PSA, and PSMA.

On March 4, 2021 Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in the development and commercialization of treatments for rare and orphan diseases, reported that Michael F. Cola, President and Chief Executive Officer, will present at two upcoming virtual conferences (Press release, Cerecor, MAR 4, 2021, View Source [SID1234576051]).

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H.C. Wainwright Global Life Sciences Conference
Date: March 9-10, 2021
Pre-Recorded Formal Presentation will be made available on Tuesday, March 9, 2021 at 7:00 AM ET

Oppenheimer’s 31st Annual Healthcare Conference
Date: Wednesday, March 17, 2021
Time: 8:00 AM ET

A live webcast of the presentations can be accessed under "News/Events" page in the Investors section of the Company’s website at www.cerecor.com.

On March 4, 2021 Apollomics, Inc., an innovative biopharmaceutical company committed to the discovery and development of mono- and combination- oncology therapies, reported that the first patient has been successfully enrolled into a Phase 1 clinical trial of APL-106 (uproleselan injection) for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) in China (Press release, Apollomics, MAR 4, 2021, View Source [SID1234576050]). In February of this year, the two Phase 1 study sites were initiated.

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The Phase 1 clinical trial is a part of the Phase 1 and Phase 3 bridging clinical study of APL-106 in combination with chemotherapy in adults with relapsed or refractory AML. Its principal investigator is Professor Jianxiang Wang of the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College. The primary objective of the Phase 1 is to study the pharmacokinetic (PK) characteristics of APL-106 in Chinese subjects with relapsed or refractory AML and to evaluate the safety and tolerability of APL-106 in combination with chemotherapy.

About APL-106 (uproleselan injection)

APL-106 (uproleselan injection) is an innovative drug discovered and developed by GlycoMimetics. Uproleselan (yoo’ pro le’ sel an) is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells, thereby disrupting the mechanism of leukemic cell resistance within the bone marrow microenvironment. In 2017, the U.S. FDA granted Breakthrough Therapy Designation to uproleselan for the treatment of adults with relapsed or refractory acute myeloid leukemia. Apollomics licensed uproleselan from GlycoMimetics in January 2020. Apollomics has the rights to clinical development, production and commercial sales in the Chinese market (Mainland China, Hong Kong, Macau and Taiwan).

In September 2020, APL-106 received the approval of the National Medical Products Administration (NMPA) through the issuance of the "Clinical Trial Drug Approval Notification". This approval enables the conduct of Phase I and Phase III bridging clinical study of APL-106 in combination with chemotherapy in adults with relapsed or refractory acute myeloid leukemia. In January 2021, APL-106 was also granted Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of the NMPA.

About Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) is a cancer of the blood and bone marrow. It is an aggressive disease that causes the bone marrow to produce immature cells that are unable to carry out their normal function and develop into leukemia cells. In the U.S., there are approximately 20,000 new cases of AML each year, and the 5-year survival rate is 28.7%1. The annual incidence of AML in China in 2019 is approximately 26,9002, and relapsed/refractory AML has an extremely poor prognosis.

On March 4, 2021 Aileron Therapeutics (NASDAQ:ALRN) reported that Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer, will give a corporate presentation at the 2021 H.C. Wainwright Global Life Sciences Conference, which is taking place March 9 – 10, 2021 (Press release, Aileron Therapeutics, MAR 4, 2021, View Source [SID1234576049]). Dr. Aivado’s presentation will include an overview of Aileron’s upcoming Phase 1b placebo-controlled trial of ALRN-6924 in patients with advanced p53-mutated non-small cell lung cancer, anticipated to begin in the second quarter of 2021, including the company’s target enrollment expansion announced earlier this week.

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A webcast of the presentation will be available on demand under the Investors and Media section of Aileron’s website at View Source beginning March 9, 2021 at 7:00 a.m. EST. A replay of the webcast will be archived on Aileron’s website for 90 days following the event.

On March 4, 2021 Kyowa Kirin reported that The National Institute for Health and Care Excellence (NICE) its final appraisal document (FAD) for POTELIGEO (mogamulizumab) announcing that POTELIGEO will not be made available on the NHS1 in England and Wales. Mogamulizumab is a treatment for adults living with rare blood cancers, mycosis fungoides (MF) and Sézary syndrome (SS), two subtypes of Cutaneous T-Cell Lymphoma (CTCL), who have received at least one prior systemic therapy (Press release, Kyowa Hakko Kirin, MAR 4, 2021, View Source [SID1234576032]).2 Kyowa Kirin is disappointed with this decision but remains committed to finding a solution for people living with MF and SS to have access to the medicine and will continue to work with NICE to find a resolution.

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Richard Johnson, Northern Cluster General Manager, responsible for the UK at Kyowa Kirin, commented: "We are disappointed that, despite feedback from the patient and clinical community together with extensive evidence provided, the appraisal committee’s decision is negative. We fully support the recently published UK Rare Disease Framework and specifically priority 4 on "improving access to specialist care, treatments and drugs". This is critically important given that many people with rare diseases, including those with CTCL, face challenges to access safe, high quality care and treatments." He added: "Kyowa Kirin remains committed to supporting adults with MF and SS. The company will do all it can to ensure people with these debilitating haematological malignancies and eligible for these treatments have access to mogamulizumab."

Professor Sean Whittaker, Professor of Cutaneous Oncology at the School of Basic and Medical Biosciences, Kings College London, and Consultant dermatologist, Guy’s and St Thomas’ NHS Foundation Trust, added: "MAVORIC is the largest randomised controlled trial completed in Cutaneous T-Cell Lymphoma, a rare form of non-Hodgkin’s lymphoma, and the only trial to have included a significant proportion of patients with the advanced leukaemic stage of disease, Sézary syndrome, for which we have no consistently effective therapies. Patients with advanced stages of CTCL represent an unmet clinical need at present and effective therapies such as mogamulizumab are urgently needed to enable consolidation with stem cell transplantation. NICE’s decision is very disappointing for patients with this rare malignancy."

MF and SS are two forms of Cutaneous T-Cell Lymphoma (CTCL), which is a serious and potentially life-threatening form of cancer that affects the skin.3 People living with the condition have a substantially reduced quality of life.4 Additionally, there is a significant impact on quality of life for those caring for an individual living with CTCL.5 CTCL is treatable but not curable and there is a clear unmet need for new treatment options.

Stephen Scowcroft, Director of Operations and External Affairs at Lymphoma Action commented: "We are deeply disappointed by this decision and the effect it will have on people living with these rare and debilitating haematological cancers. We know that there is a real need for effective treatments for people living with mycosis fungoides and Sézary syndrome as there are currently limited treatment options and this can have a significant impact on a person’s quality of life, daily function and social interactions. We believe patients should have access to the best care and there continues to be a need for effective treatments for people living with mycosis fungoides and Sézary syndrome. We call on the relevant stakeholders, NICE and the company, to continue to discuss the options and to work with the patient community to find a resolution."

About POTELIGEO (mogamulizumab)

Mogamulizumab is a first-in-class humanised monoclonal antibody (mAb) directed against CC chemokine receptor 4 (CCR4), a protein consistently expressed on cancerous cells seen in both MF and SS;6,7,8 once mogamulizumab binds to CCR4, it increases attraction of immune cells from the immune system to destroy the cancerous cells.9

Mogamulizumab has been shown to offer benefits to many patients with MF and SS.10 The MAVORIC trial compared the efficacy of mogamulizumab with vorinostat in previously treated people with relapsed or refractory mycosis fungoides or Sézary syndrome, two types of Cutaneous T-cell lymphoma (CTCL).10 Patients taking mogamulizumab experienced control over their disease for more than twice as long as those taking the comparator treatment, vorinostat*1 (7.7 months vs 3.1 months of median progression free survival), the primary endpoint of the trial.10 Levels of adverse events were similar between the two treatment groups.10

About Mycosis Fungoides (MF) and Sézary Syndrome (SS)

MF and SS are characterised by localisation of cancerous white blood cells called T lymphocytes (T cells), to the skin.11,12 These cancerous T cells consistently express a protein called CC-chemokine receptor 4 (CCR4), which enables them to move from the blood to the skin.6,7,8 When these cancerous T cells move to the skin, they can create a localised inflammatory immune skin response, commonly resulting in visible skin symptoms of red patches or plaques 6,13,14,15,16 which can resemble psoriasis or eczema.11

MF and SS can affect the skin, blood, lymph nodes (part of the body’s immune system which is spread throughout the body) and internal organs.17 All four areas of the body are used to assess disease stage18,19 and clinically significant involvement of the blood, particularly in more advanced disease, is linked with increased morbidity and an overall reduction in patient survival.18,20,21

Due to its likeness to more common skin conditions such as eczema and psoriasis,11 CTCL can take, on average, between 2 and 7 years for individuals to receive a confirmed diagnosis.22 It is critical for doctors to diagnose CTCL as early as possible as the patient’s prognosis can be affected if the disease progresses to later stages.23 Whilst most individuals that present with early stage do not progress to a more severe stage,24 patients with advanced disease have significantly poorer outcomes with only around half of patients (52%) surviving for just 5 years.18

CTCL is a rare disease that affects 0.7 per 100,000 patients across the UK.25 The annual incidence of MF in Europe is estimated to be between 1 in 110,000 to 1 in 350,000.26 The annual incidence of SS is 1 in 10,000,000.27 Together they represent approximately 65% of all cases of CTCL.17