On October 14, 2021 OncoNano Medicine, Inc. reported that results from a Phase 2 study of its lead clinical development candidate, pegsitacianine, presented as a late- breaking oral presentation at the 2021 World Molecular Imaging Congress (WMIC) (Press release, OncoNano Medicine, OCT 14, 2021, View Source [SID1234591223]). This late- breaking oral presentation by Dr. Jason Newman of the University of Pennsylvania Health System revealed the fluorescent nanoprobe, pegsitacianine, provided real-time surgical imaging in a tumor-agnostic manner for the accurate identification of malignant tissue in the operating room.

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Pegsitacianine is an intraoperative fluorescence imaging agent under development by OncoNano Medicine for the detection of cancerous tissue in patients undergoing removal of their solid tumor. Relying on an ultra pH-sensitive activation mechanism of OncoNano’s ON-BOARDTM platform, pegsitacianine exists in a fluorescently dark ( "Off") state at physiological pH but transitions rapidly to a fluorescently "On" state in the presence of the elevated acidic tumor microenvironment. Pegsitacianine’s unique mechanism of action provides it with the potential to function as a tumor agnostic imaging agent compatible with most clinical cameras designed for ICG imaging across a variety of solid tumor types. Pegsitacianine has previously been studied in a Phase 1 clinical trial where breast, head and neck, colorectal, and esophageal cancers were successfully imaged following an intravenous dose of pegsitacianine.

"The Phase 2 study results for pegsitacianine presented at the recent World Molecular Imaging Congress indicate this fluorescent nanoprobe has the potential to change the current surgical paradigm," said Dr. Newman. "With the real-time feedback that pegsitacianine provides, we could now have a tool that offers real-time surgical imaging on margin status and provides a greater degree of confidence in achieving complete tumor resection. Given the unique activation mechanism of targeting a universally dysregulated tumor metabolic signature, pegsitacianine could be beneficial in aiding surgical oncologists across multiple tumor disciplines."

The recently completed Phase 2 study (NCT03735680) was a non-randomized, open-label, multi-center study being conducted at three U.S. sites. This exploratory study further confirmed the selected dose-schedule, expanded upon the drug’s safety profile and increased the number of tumor types in which pegsitacianine imaging was shown to be feasible. Together, the results demonstrated that pegsitacianine is well-tolerated in patients and provides the surgeon with accurate, real-time feedback in the operating room. Intraoperative imaging observations demonstrated a high correlation with the pathological assessments of collected tissues, and imaging can be reliably performed using multiple FDA-cleared NIR imaging devices. Results from evaluable patients demonstrated favorable sensitivity (100%) and specificity (92%) values, as well as a strong negative predictive value (100%) for detecting histologically normal tissue that was collected as part of standard of care surgery.

"The high sensitivity and specificity valuesfrom the Phase 2 trial not only corroborated our Phase 1 observations to further support the use of OncoNano’s ON-BOARDTM platform in surgical imaging, but also highlight the potential benefit of this pH-activatable platform for tumor-targeted delivery of therapeutic payloads with an increased therapeutic window," commented Jinming Gao, PhD, OncoNano’s Chief Scientific Officer.

On October 14, 2021 Purple Biotech Ltd. (the "Registrant" or the "Company") reported that the Company entered into a settlement agreement with Lupin Ltd. and its subsidiary Lupin Pharmaceuticals, Inc. (collectively, "Lupin"), resolving the Company’s patent litigation against Lupin (Press release, Purple Biotech, OCT 14, 2021, View Source [SID1234591220]). The litigation concerned Lupin’s filing of an Abbreviated New Drug Application (ANDA) seeking approval from the U.S. Food and Drug Administration (FDA) to market a generic version of the Company’s commercial drug CONSENSI. This agreement with Lupin allows Lupin to enter the market on March 1, 2030 and earlier, under certain circumstances.

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On October 14, 2021 Kyowa Kirin International PLC (Kyowa Kirin), a wholly owned subsidiary of Kyowa Kirin Co., Ltd., reported a new study of real world data showing higher response rates than previously seen in people living with cutaneous T-cell lymphoma (CTCL) treated with POTELIGEO (mogamulizumab) (Press release, Kyowa Hakko Kirin, OCT 14, 2021, View Source [SID1234591218]). The findings, from a retrospective observational study of French CTCL patients, showed that the best overall response rate in the real-life study was 58.5 per cent, compared to 35 per cent seen in the MAVORIC trial.1,2 The MAVORIC study was the pivotal Phase 3 trial that investigated treatment with POTELIGEO compared to an active comparatora in previously treated adult patients with mycosis fungoides (MF) and Sézary syndrome (SS), two types of CTCL.

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Professor Martine Bagot of Saint-Louis Hospital, Université de Paris, France said: "The purpose of the study was to assess, for the first time, the efficacy and safety of mogamulizumab in a real-life setting. We saw a higher response in the real-life study compared to the MAVORIC trial results. The increase was also seen in both the MF and SS patient groups, compared to MAVORIC. As both the real world data and MAVORIC recruited a broadly similar set of patients, this new data underlines the value of mogamulizumab in treating these CTCL patients, whose disease causes a severe impact on their functioning, emotional and social wellbeing."

Data was collected and analysed from medical records of 124 MF and SS patients treated with POTELIGEO between February 2014 and March 2020, from 14 specialist treatment centres in France. The best overall response rate (ORR) seen in the real-life study was 47.0% in MF patients and 68.3% in SS patients. In contrast, the ORRb in MAVORIC was 21% and 37%, respectively.1,2 Of the 124 patients who received POTELIGEO treatment in the study, 44% were MF patients and 56% were SS patients. This compares to 56% and 44% in MAVORIC, respectively. In the real-life study, 49.6% of patients were at disease stage IVA1, compared to 39% in MAVORIC.1,2

POTELIGEO was also shown to have a favourable safety profile, in the real life study the most common drug-related treatment-emergent adverse events (TEAEs) were lymphopenia and asthenia. Commonly occurring TEAEs seen in both the real life study and MAVORIC were rash and infusion-related reactions.1,2

Abdul Mullick, President of Kyowa Kirin International, said: "Kyowa Kirin is committed to improving outcomes for patients with rare diseases, whose needs are often underserved. It’s heartening to see this new real world data, which reinforces what we’ve seen previously in clinical trials. Kyowa Kirin is dedicated to improve the lives of patients with CTCL, which is a chronic, debilitating disease and to deliver on our purpose, to make people smile."

MF and SS are subtypes of CTCL, a rare type of non-Hodgkin’s lymphoma that can affect the skin, blood, lymph nodes and internal organs.3 It is associated with disfiguring skin lesions, intractable itching, sleep disturbance, and psychosocial problems, CTCL has a serious negative effect on quality of life.4

The new data was presented at a poster session at the EORTC meeting in Marseille, France, which is being held on 14-16 October 2021.

a The comparator in the MAVORIC trial was vorinostat, a histone deacetylase inhibitor (HDACi) and FDA-approved standard of care option for treatment of CTCL that is licensed in the US for the treatment of cutaneous manifestations in patients with CTCL who have progressive, persistent or recurrent disease on or following two systemic therapies. Vorinostat is not licensed for use in Europe.

b MAVORIC ORR included only those patients with confirmed global response at two (or more) successive evaluations at least 8 weeks apart).

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

Cutaneous T-cell lymphoma (CTCL) is a cancer of white blood cells (lymphocytes) that presents in the skin.5 CTCLs are rare, serious and potentially life-threatening forms of non-Hodgkin lymphoma (NHL).2 Malignant T cells in CTCL circulate in the blood and migrate to the skin6 causing lesions and itching.7

The annual incidence of CTCL in Europe is around 5.2 new cases per 100,000 population8 and it affects around 240 people per million in the region at any one time.5 The two best-studied types of CTCL are mycosis fungoides (MF) and Sézary syndrome (SS).9 Together, MF and SS account for around two thirds of all CTCLs.10,11

MF accounts for approximately 60% of all CTCLs,10 is typically indolent and is characterised by skin symptoms including patches or plaques, skin redness and tumours.12
SS is much rarer, accounting for around 5% of CTCLs,6 and is more aggressive13 with high levels of blood involvement by definition.10 It causes very severe itching, total body redness (erythroderma), intense scaling of the skin and frequent hair loss. 7
It can typically take between three and six years from CTCL disease onset for a patient to be diagnosed, in some cases it can take decades.10,12 This is sometimes due to the fact that CTCL presents very similarly to other benign skin conditions (e.g. eczema and psoriasis)7; this can mean many patients experience a long, frustrating journey before diagnosis.

CTCL can be very distressing for patients and has a significant negative life-long impact upon many aspects of a patient’s life.12,14 CTCL causes chronic skin impairment with itching, pain, recurrent infections, and disfiguring skin lesions, as well as sleep disturbance and psychosocial problems.9,12 Even in the early stages of CTCL, the disease can affect patients’ ability to meet the needs of their family, interfere with their job, and limit normal daily activities.14

About POTELIGEO (mogamulizumab) and the MAVORIC Trial

Malignant T lymphocytes in MF and SS, consistently express a molecule called CC chemokine receptor 4 (CCR4).2 POTELIGEO is a humanised monoclonal antibody that selectively binds to CCR4-expressing cells15 and helps destroy them by harnessing the body’s immune system.2 POTELIGEO is a systemic therapy, administered by intravenous infusion weekly for the first five weeks, then subsequently every two weeks.15 The registrational Phase 3 MAVORIC trial was the largest randomised study of systemic therapy in CTCL. It evaluated the safety and efficacy of POTELIGEO versus vorinostat in patients who had failed at least one previous systemic therapy.2

Following a positive opinion from Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), the European Commission (EC) granted marketing authorisation for POTELIGEO in November 2018 for the treatment of adult patients with MF or SS who have received at least one prior systemic therapy.15

Important Prescribing information and Safety Information

Refer to the full Summary of Product Characteristics (SmPC) for prescribing information and the full safety information: View Source

On October 14, 2021 Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, reported the appointment of Theresa Heah, M.D., MBA as Chief Medical Officer and President of Kriya’s newly launched ophthalmology division, Kriya Ophthalmology (Press release, Kriya Therapeutics, OCT 14, 2021, View Source [SID1234591217]). Dr Heah will be responsible for advancing Kriya’s current pipeline of ophthalmology gene therapies and continuing to expand Kriya’s ophthalmology portfolio through its in-house R&D and business development engines.

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Kriya Ophthalmology is focused on the discovery and development of transformative gene therapies for ocular diseases with high unmet need. By leveraging Kriya’s computationally enabled vector design, data analytic and manufacturing technologies, Kriya Ophthalmology is uniquely positioned to accelerate the development of best-in-class gene therapies for rare and prevalent ocular diseases. Kriya Ophthalmology is currently advancing therapeutic programs in uveitis, geographic atrophy and additional undisclosed disease areas.

"Gene therapy has the potential to deliver transformative clinical benefits to address severe ocular diseases for which there are currently few effective treatment options," said Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya Therapeutics. "The launch of Kriya Ophthalmology, and the appointment of Theresa to lead our specialized division focused exclusively on diseases of the eye, reflects our commitment to delivering better products to patients suffering from a range of rare and prevalent ocular diseases. We believe that Kriya’s advanced technology platforms, combined with Theresa’s leadership and track record in ophthalmology, uniquely position this division to become one of the industry leaders in the development of gene therapies for ocular diseases."

"We are thrilled to welcome Theresa to lead Kriya Ophthalmology," said Ilise Lombardo, M.D., Chief Medical Officer of Kriya Therapeutics. "Her success in developing and launching treatments for ocular diseases will help Kriya play a pivotal role in the advancement of novel gene therapies that address a range of severe ocular conditions."

Dr. Heah is an accomplished executive with more than 20 years of R&D, regulatory strategy and clinical development experience. She previously served as Chief Medical Officer and Executive Vice President of Operations for AsclepiX Therapeutics where she led the company’s Series A financing and advancement of its pipeline products into the clinic. Prior to joining AsclepiX Therapeutics, Dr. Heah served as Chief Medical Officer at Applied Genetic Technologies Corporation (AGTC), where she worked to develop gene therapies in ophthalmology and rare diseases. She has also held several leadership positions with increasing responsibility in early-stage private companies (Fovea Pharmaceuticals), publicly traded companies (Aerie Pharmaceuticals, Allergan) and big pharmaceutical companies (Bayer Healthcare, Sanofi).

Dr. Heah has led multiple successful global regulatory submissions and commercialization of products including Ozurdex, EYLEA, Rhopressa and Rocklatan. While at Bayer, she launched EYLEA successfully into its eventual status as a blockbuster drug. She earned her M.D. from Guy’s, King’s and St. Thomas’ School of Medicine, King’s College, University of London, and her Executive Master’s in Business Administration from the European School of Management & Technology (ESMT), Berlin.

"My career has been dedicated to patients and the development of transformative and meaningful therapies aimed at improving their quality of life," said Dr. Heah. "Many still suffer from serious eye diseases for which there are no viable treatments. There is immense untapped potential in gene therapy, and it will be our mission to uncover and advance novel medicines for the betterment of underserved patient communities."

On October 14, 2021 OnKure, Inc., a clinical-stage biopharmaceutical company discovering and developing the next generation of oncology precision medicines, reported the appointment of Jennifer R. Diamond, M.D., as Chief Medical Officer (Press release, OnKure, OCT 14, 2021, View Source [SID1234591216]). Dr. Diamond, a trained oncologist, brings over 10 years of extensive clinical expertise in early phase trials and translational oncology research.

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"We are very pleased to welcome Jennifer to our growing leadership team," said Tony Piscopio, Ph.D., Co-Founder, President and Chief Executive Officer of OnKure. "Her vast knowledge of conducting early-stage trials for novel oncology therapeutics, as well as her experience exploring novel combination strategies, will be vital as we continue to build and advance our broad pipeline of best-in-class precision medicines."

In addition to serving in her role at OnKure, Dr. Diamond works in the Division of Medical Oncology at the University of Colorado Anschutz Medical Campus as a breast cancer medical oncologist and Associate Professor of Medicine. She is also the Clinical Research Director of the Phase 1 and Expansion/Molecular Studies Program (POEMs) and the founding Co-Director of the Women’s Cancer Developmental Therapeutics Program at the CU Cancer Center. Dr. Diamond has been involved in clinical and translational research in the Phase 1 and Breast Cancer research programs at CU since 2007 and currently runs a research laboratory focused on the development of targeted agents in TNBC, including strategies to overcome treatment resistance. She received her B.A. from the University of Arizona and her M.D. from the University of Arizona College of Medicine. Dr. Diamond completed her fellowship in Medical Oncology at CU under Dr. Gail Eckhart.

"I am thrilled to be joining OnKure at such an important time in the Company’s growth," said Dr. Diamond. "OnKure’s proven structure-based drug design approach has the potential to create optimal, tumor-agnostic treatments for cancer patients with high unmet need. I look forward to leveraging my experiences to lead the Company’s clinical development efforts and advance its promising lead candidate, OKI-179, as well as future oncology programs in the clinic."