On September 23, 2021 Ayala Pharmaceuticals, Inc. (NASDAQ: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, reported the publication of two case studies of adult patients with desmoid tumors treated with AL101 in Current Oncology (Press release, Ayala Pharmaceuticals, SEP 23, 2021, View Source [SID1234594000]). This publication highlights the potential of a gamma secretase inhibitor for the treatment of desmoid tumors.

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The data included in the case study are based on earlier Phase 1 results and compassionate use of AL101 in desmoid tumors. Both patients showcased in these case studies, Case One and Case Two, presented with significant tumor burden and symptomatic and life-threatening disease due to disease bulk and location. Both patients achieved long-lasting partial responses (PR) with AL101 treatment with a maximal decrease in tumor size from baseline of 41% after approximately 1 year (55 weeks) of treatment in Case One, and a maximal decrease in tumor size from baseline of 60% after about 1.6 years (82 weeks) of treatment in Case Two. With continued monitoring, one patient was able to discontinue AL101 after 4.6 years of treatment, while maintaining a PR, and the other patient has maintained a PR at a reduced AL101 dose.

"Both of these patients’ case studies represent additional evidence to support the development of our gamma secretase inhibitor, AL102 for the treatment of desmoid tumors. The body of data conducted by BMS in patients with desmoid tumors implicating the role of gamma secretase inhibition furthers our hypothesis for treating desmoid tumors through AL102’s mechanism of action," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "Desmoid tumors continue to be an area of high unmet medical with a significant impact to patients’ quality of life, and we are pleased to have initiated our pivotal Phase 2/3 RINGISDE trial of AL102 to potentially address this gap in the existing treatment paradigm."

The pivotal Phase 2/3 RINGSIDE trial is designed to evaluate the efficacy, safety and tolerability of AL102 in adult and adolescent patients with desmoid tumors. Part 1 of the study is open label and will enroll up to 36 patients with progressive desmoid tumors in three study arms across three doses of AL102: 1.2 mg daily (QD), 2 mg twice weekly (QIW) and 4mg twice weekly (QIW) with initial follow up of safety, tolerability and tumor volume by MRI after 16 weeks in order to determine the optimal dose. At the end of part 1, all patients will be eligible to enroll into an open label extension study at the selected dose where long-term efficacy and safety will be monitored.

Part 2 of the study will start immediately after dose selection from part 1 and will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized 2:1 between AL102 or placebo. The study’s primary endpoint will be progression free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR) and patient reported Quality of Life (QOL) measures.

About Desmoid Tumors

Desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

On September 23, 2021 Revitope Oncology, Inc. is a privately owned company in Cambridge, MA, reported that focuses on the development of next-generation T cell engager immunotherapies for a variety of solid cancer indications (Press release, Revitope Oncology, SEP 23, 2021, View Source [SID1234591881]). Revitope’s platform is a suite of proprietary and modular bispecific antibodies designed to deliver improved therapeutic efficacy and safety through built-in control mechanisms that enable exquisite tumor-specificity. The company has several cancer programs in preclinical development and expects to have its lead program in the clinic by early 2023.

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On September 23, 2021 Chimeric Therapeutics (ASX:CHM, "Chimeric" or the "Company"), a clinical-stage cell therapy company, reported the European Patent Office’s decision to grant patent EP 3,362,470 B1, which was published in the European Patent Bulletin dated September 22, 2021 (Press release, Chimeric Therapeutics, SEP 23, 2021, View Source [SID1234591514]).

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The granted patent covers certain applications of chimeric antigen receptor (CAR) technology using chlorotoxin (CLTX), including Chimeric’s clinical-stage CAR T asset, CHM 1101, with patent protection expected until 2036.

Chimeric holds the exclusive worldwide license to develop and commercialize EP 3,362,470 B1 and related patent applications filed in other global territories.

Commenting on Chimeric’s intellectual property position, CEO and Managing Director Jennifer Chow said:

"We are pleased to see the continued advancement of the strong intellectual property portfolio underpinning Chimeric’s CLTX CAR T program, on this occasion in a geography that holds significant market potential."

Authorised on behalf of the Chimeric Therapeutics board of directors by Chairman Paul Hopper.

On September 23, 2021  Carevive Systems (Carevive), the leading oncology-focused health technology company centered on understanding and improving the experience of patients with cancer, reported that launched Carevive SMART Data, a dataset containing information from patients about their experience with treatment, combined with demographic and clinical data (Press release, Carevive Systems, SEP 23, 2021, View Source [SID1234590367]).

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As the healthcare industry becomes more saturated with big data, the search continues for data that can inform decisions that positively impact the experience and outcomes of patients with cancer. Big data, defined by large datasets with substantial numbers of patients and a wide array of clinical and claims data, can support certain types of statistical analyses. However, these datasets are limited because their datapoints are discrete, providing only sequential snapshots of a patient’s health status, without a more continuous assessment of the patient’s experience on treatment, which can only originate from the patients themselves.

In response to the need for this type of dataset, Carevive uses its digital oncology platform to collect information from patients about their experience at weekly time points during treatment. These data are combined with demographic and clinical data to create Carevive SMART Data (Symptom Monitoring with Applied Remote Technology).

Carevive SMART Data

Carevive SMART Data are captured as part of Carevive OPT-IN. Carevive OPT-IN, the Oncology Pragmatic Trial Investigator Network, enables patient experience data to be collected from real-world patients who are receiving treatment for cancer. Carevive OPT-IN began in late 2020 to amplify the patient voice in their own care and improve their outcomes. Carevive SMART Data are collected as part of an ongoing, weekly conversation between the patient and their care team, facilitated by Carevive’s cloud-based, digital oncology platform that patients can access with a variety of devices, from any location. Providing patients with remote symptom monitoring enables longitudinal data to be collected describing the holistic patient experience, including symptoms, quality of life, physical function, and healthcare resource utilization.

Licenses to Carevive SMART Data are available for life science companies who would like to analyze patient-centric data combining longitudinal patient-reported outcomes (PRO) with EHR-derived clinical data. By listening to the patient’s voice in cancer care, Carevive can now address many important questions about the patient experience in cancer which have not been answered by big data.

Mordecai Kramer, Carevive’s Vice President of Data Generation & Outcomes, Life Sciences, summarizes the value of this novel dataset as follows: "We are excited about Carevive SMART Data and the holistic view of the patient experience that it provides. Since launching our registries, we are already seeing the value this longitudinal dataset creates for improving patient care and outcomes. We invite life science companies to join the Carevive SMART Data conversation and, together, answer pressing questions about real-world patients with cancer."

On September 23, 2021 Kyowa Kirin, Inc., an affiliate of Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company, reported the launch of two efforts focused on increasing understanding of mycosis fungoides (MF) and Sézary syndrome (SS), two subtypes of cutaneous T-cell lymphoma (CTCL),2 and the role of POTELIGEO (mogamulizumab-kpkc) in treating them.3 Aimed at physicians, the new Treat the Blood (Press release, Kyowa Hakko Kirin, SEP 23, 2021, View Source [SID1234590295]). Treat the Skin. omni-channel campaign seeks to educate about the importance of measuring and monitoring blood involvement to inform treatment and potentially impact patient outcomes in MF and SS. To reach patients, POTELIGEO patient ambassadors will share their CTCL journeys and experiences on treatment through video testimonials and other educational programming. The launch of both initiatives coincides with Blood Cancer Awareness Month.

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"To have a profound impact in a rare disease like CTCL, it’s important to invest in education and programming that helps to advance disease awareness, recognition and management," said Sue Smith, Vice President, North American Oncology Franchise Head, Kyowa Kirin, Inc. "With increasing blood burden linked to poorer prognosis in both mycosis fungoides and Sézary syndrome, it’s critical that the blood is monitored for disease progression and response to treatment. Our physician effort is focused on increasing understanding of the value of routine blood testing, and the proven efficacy of POTELIGEO in treating MF and SS symptoms."3,4-6

CTCL is a rare type of blood cancer that affects the body’s T cells. Because it first appears on the skin, it is often mistaken for common skin conditions like eczema or psoriasis, which can delay diagnosis by years or even decades.7 Most patients have mycosis fungoides, a chronic and typically slow growing cancer that initially manifests in the skin but can spread within the skin or to other areas, including the blood, in 30% of cases.2,5,8,10 Sézary syndrome is a rare (<5%), serious form of CTCL that affects the skin and blood from the start.11 Increasing blood burden in CTCL has been associated with worsening overall survival, disease-specific survival, and an increased risk of disease progression.5,8,12

Encouraging Physicians to Test More
The new Treat the Blood. Treat the Skin. campaign explains the value of understanding patients’ blood burden when determining appropriate treatment selection in MF and SS. Given that skin inflammation is often focused on as the source of discomfort and visible sign of disease, the campaign aims to call attention to the potential risk associated with blood involvement in MF and SS patients.7,13 A recent post-hoc analysis of data from the phase 3 MAVORIC trial published in the Journal of the European Academy of Dermatology and Venereology, evaluated the efficacy and safety of POTELIGEO in MF and SS across all levels of blood classifications compared to vorinostat.4

The campaign will reach healthcare professionals through a targeted mix of personal, print and digital communications supported by a new healthcare professional website (www.poteligeohcp.com) that reviews POTELIGEO’s efficacy and safety in MF and SS patients with varying levels of blood involvement.1,4 Peer-to-peer education featuring experts in the field is planned to further extend the reach of the initiative is planned for early 2022.

Bringing Real Patient Experiences to Life
Additional efforts are being made to educate patients about POTELIGEO as a treatment option.1 Given CTCL is a rare disease, patients are eager to connect with and learn from others who share the same diagnosis. Research indicates they rely heavily on advocacy groups and online forums for information when assessing and evaluating treatment options.2 To address the need for more information about patient experiences on treatment, educational content and programming has been created featuring the stories of real POTELIGEO patients – including their journey to diagnosis, experiences on treatment and what motivates them to never give up.

"I wanted to share my story to encourage others to never give up, to keep pushing for answers," said Jeff, POTELIGEO ambassador living with mycosis fungoides. "It’s easy to feel overwhelmed, to say it’s just not going to work and to give up. By learning how to advocate for myself, I was able to finally find the right specialists, the right diagnosis and ultimately, the right treatment for me."

Jeff’s and other patient stories will be viewable on www.poteligeo.com, and other online channels. Additional events with patient speakers are planned for 2022.

Please see POTELIGEO Indication and Important Safety Information below.

U.S. Indication

POTELIGEO (mogamulizumab-kpkc) injection for intravenous infusion is indicated for the treatment of adult patients with relapsed or refractory mycosis fungoides or Sézary syndrome after at least one prior systemic therapy.

Important Safety Information

Warnings and Precautions:

Dermatologic toxicity: Monitor patients for rash throughout the course of treatment. For patients who experienced dermatologic toxicity in Trial 1, the median time to onset was 15 weeks, with 25% of cases occurring after 31 weeks. Interrupt POTELIGEO for moderate or severe rash (Grades 2 or 3). Permanently discontinue POTELIGEO for life-threatening (Grade 4) rash or for any Stevens-Johnson syndrome (SJS) or toxic epidermal necrolysis (TEN).
Infusion reactions: Most infusion reactions occur during or shortly after the first infusion. Infusion reactions can also occur with subsequent infusions. Monitor patients closely for signs and symptoms of infusion reactions and interrupt the infusion for any grade reaction, and treat promptly. Permanently discontinue POTELIGEO for any life-threatening (Grade 4) infusion reaction.
Infections: Monitor patients for signs and symptoms of infection and treat promptly.
Autoimmune complications: Interrupt or permanently discontinue POTELIGEO as appropriate for suspected immune-mediated adverse reactions. Consider the benefit/risk of POTELIGEO in patients with a history of autoimmune disease.
Complications of allogeneic HSCT after POTELIGEO: Increased risks of transplant complications have been reported in patients who received allogeneic HSCT after POTELIGEO. Follow patients closely for early evidence of transplant-related complications.
Adverse Reactions:

The most common adverse reactions (reported in ≥ 10% of patients) with POTELIGEO in the clinical trial were rash, including drug eruption (35%), infusion reaction (33%), fatigue (31%), diarrhea (28%), drug eruption (24%), upper respiratory tract infection (22%), musculoskeletal pain (22%), skin infection (19%), pyrexia (17%), edema (16%), nausea (16%), headache (14%), thrombocytopenia (14%), constipation (13%), anemia (12%), mucositis (12%), cough (11%), and hypertension (10%).
You are encouraged to report suspected adverse reactions to Kyowa Kirin, Inc. at 1-844-768-3544 or FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch/.
Please click here for full U.S. prescribing information.

About POTELIGEO (mogamulizumab-kpkc)
POTELIGEO is a humanized monoclonal antibody that recruits the body’s own immune cells to kill CCR4+ (CC chemokine receptor 4) malignant T-cells.1,14 CCR4 is overexpressed in Sézary syndrome and in mycosis fungoides at all stages.15,16 POTELIGEO was produced using Kyowa Kirin’s proprietary POTELLIGENT technology platform, which is associated with enhanced antibody-dependent cellular cytotoxicity.16