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TRILLIUM THERAPEUTICS DOSES FIRST PATIENT WITH TTI-622, ITS
SECOND IMMUNE CHECKPOINT INHIBITOR TARGETING CD47

On June 14, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported that it has initiated dosing in its phase 1 clinical trial of TTI-622 (SIRPaFc-IgG4), a checkpoint inhibitor of the innate immune system, in relapsed or refractory lymphoma or myeloma (Press release, Trillium Therapeutics, JUN 14, 2018, View Source [SID1234527309]).

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TTI-622 is a fusion protein that blocks the inhibitory activity of CD47, a molecule that is overexpressed by a wide variety of tumors. CD47 binds to SIRPa on macrophages and delivers a "do not eat" signal that inhibits the ability of macrophages to engulf and destroy cancer cells. Preclinical studies have shown that TTI-622 has anti-tumor activity across a range of hematologic tumors.

TTI-622 is the second SIRPaFc decoy receptor that Trillium has advanced into the clinic. TTI-621 (SIRPaFc-IgG1), which consists of the same CD47-binding domain of human SIRPa as TTI-622 but linked to an IgG1 Fc region, is currently in two multicenter trials and has produced positive signals of activity in mycosis fungoides, Sézary syndrome and diffuse large B-cell lymphoma patients. The different pharmacologic properties of TTI-621 and TTI-622 will allow Trillium to explore the relationships between the level of CD47 blockade, Fc isotype, tolerability and anti-tumor activity in patients.

"This is an exciting time for Trillium as we initiate clinical development with our second CD47 blocking agent," commented Dr. Yaping Shou, Trillium’s Chief Medical Officer. "TTI-622 deepens our presence in the CD47 space, and its minimal binding to human erythrocytes could confer best-in-class status among IgG4-based blocking agents currently in development."

A two-part, multicenter, open-label, phase 1a/1b study of TTI-622 in patients with advanced relapsed or refractory lymphoma or multiple myeloma has been initiated. In the phase 1a dose-escalation part, patients will be enrolled in sequential dose cohorts to receive TTI-622 once weekly to characterize safety, tolerability, pharmacokinetics, and to determine the maximum tolerated dose. In the phase 1b part, patients will be treated with TTI-622 in combination with rituximab, a proteasome inhibitor-containing regimen, or a PD-1 inhibitor. Rituximab and proteasome inhibitors may provide additional "eat" signals that could enhance the efficacy of TTI-622. A PD-1 inhibitor may help amplify any anti-tumor T cell response generated by TTI-622.

Avelas Biosciences to Present at JMP Securities Annual Life Sciences Conference

On June 14, 2018 Avelas Biosciences, Inc., a clinical stage oncology-focused platform technology company that is developing products to advance a new standard-of-care for cancer surgery and therapeutic intervention, reported that Carmine N. Stengone, President and Chief Executive Officer, will present at the JMP Securities Life Sciences Conference on Thursday, June 21, 2018, at 1:30 p.m. EDT (10:30 a.m. PDT) in New York City (Press release, Avelas Biosciences, JUN 14, 2018, View Source [SID1234527308]).

ArQule to Present at The JMP Securities Life Sciences Conference on June 21, 2018

On June 14, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that Paolo Pucci, Chief Executive Officer, and Dr. Brian Schwartz, Chief Medical Officer and Head of Research and Development, will present at The JMP Securities Life Sciences Conferenceon June 21, 2018, at 10:30am EDT at the St. Regis in New York (Press release, ArQule, JUN 14, 2018, View Source [SID1234527307]).

The live webcast of the presentation will be available via the "Investors & Media" section of ArQule’s website, www.arqule.com, under "Events & Presentations." A replay of the webcast will be available shortly after the conclusion of the presentation.

bridgebio pharma launches coa therapeutics to target coenzyme-a for rare genetic disorders

On June 13, 2018 BridgeBio Pharma reported the launch of CoA Therapeutics, a biopharmaceutical company developing novel small-molecules designed to increase Coenzyme-A (CoA) levels in genetic disorders where CoA deficiency is implicated (Press release, BridgeBio, JUN 13, 2018, https://investor.bridgebio.com/news-releases/news-release-details/bridgebio-pharma-launches-coa-therapeutics-target-coenzyme-rare [SID1234576279]). BridgeBio has committed funding to drive the program toward clinical studies. CoA Therapeutics’ lead compound will be entering the clinic in 2019 with a planned initial focus on pantothenate kinase-associated neurodegeneration, or PKAN.

PKAN is an autosomal recessive genetic disorder caused by mutations in the pantothenate kinase 2 (PANK2) gene. The PANK2 enzyme plays a critical role in the synthesis of CoA, which is crucial in energy metabolism and implicated in a large number of developmental disorders. Patients with PKAN either have a complete absence or a significant deficiency of the PANK2 enzyme, which may lead to reduced CoA levels in the brain. There are currently no treatments approved for PKAN.

The CoA Therapeutics’ lead compounds, which were obtained under a license from St. Jude Children’s Research Hospital, were discovered and developed by St. Jude investigators Suzanne Jackowski, Ph.D., Charles Rock, Ph.D., Richard Lee, Ph.D., and Stephen White, Ph.D.

The prevalence of PKAN is estimated to be three in every 1,000,000 people. In the typical form of PKAN, symptoms present before the age of 10, and patients may rapidly experience neuronal degeneration, causing problems with movement, speech and vision.

"PKAN is a progressive, debilitating disease with no current approved therapy, and patients and their families currently rely on supportive treatments, which partially address the symptoms but not the root cause," said Shafique Virani, M.D., CEO of CoA. "We believe that our novel approach, using a highly brain-penetrant compound to directly target enzyme activity in neurons, can safely increase CoA levels, ease patients’ symptoms and make a meaningful difference in their quality of life. Our novel approach also holds promise for other diseases with defects in CoA metabolism, including the organic acidemias."

Joining Dr. Virani is Adam Shaywitz, M.D., Ph.D., who will serve as chief medical officer at CoA as well as CMO-in-residence at BridgeBio. Shaywitz was most recently executive director in clinical sciences at BioMarin Pharmaceuticals where he was involved in the design and planning of a number of clinical studies in rare disease including serving as program lead for the natural history and clinical treatment studies in Sanfilippo Syndrome B (MPS IIIB).

"We are privileged to be working with Drs. Jackowski, Rock, Lee and White, who have been at the forefront of CoA metabolism research in health and disease," said Neil Kumar, Ph.D., CEO of BridgeBio. "Their novel approach, which relies on increasing activity of the PANK enzyme family, is a great example of an allosteric agonist approach to potentially treat patients who have very few options."

Nordic Nanovector Announces Archer-1 Trial of Betalutin® plus Rituximab in 2L Follicular Lymphoma Approved to Start in Norway

On June 13, 2018 Nordic Nanovector ASA (OSE: NANO) reported that it has received approval from the Regional Committees for Medical and Health Research Ethics (REK) in Norway with regard to the Clinical Trial Application (CTA) for the Archer-1 Phase 1b trial with Betalutin (177Lu-satetraxetan-lilotomab) in combination with rituximab in second-line follicular lymphoma patients (2L FL) (Press release, Nordic Nanovector, JUN 13, 2018, View Source [SID1234553498]).

Nordic Nanovector has now received all necessary approvals to begin Archer-1 in Norway and will commence start-up activities immediately. Archer-1 is a Phase 1b clinical trial designed to investigate the safety, tolerability, pharmacokinetic and preliminary efficacy of the Betalutin/rituximab combination in approx. 20 2L FL patients, and the study will initially be conducted in Norway. Further countries are expected to be added later. The first patient is expected to be dosed in the second half of 2018.