Onconova Therapeutics Announces Presentation at BIO Digital 2020

On June 8, 2020 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), reported that Avi Oler, Senior Vice President, Corporate Development, will present at BIO Digital 2020, being held virtually June 8 – 12, 2020 (Press release, Onconova, JUN 8, 2020, View Source [SID1234560892]).

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Mr. Oler will provide an overview of the Company’s corporate development objectives and upcoming milestones for its lead candidate rigosertib and its oncology product candidate pipeline. The presentation will be available here for viewing on-demand.

Mr. Oler will be available for virtual meetings throughout BIO Digital. To arrange a meeting, please visit the BIO One-on-One Partnering webpage.

Nimbus Therapeutics Announces Expansion of its Drug Discovery Pipeline Across Oncology, Immunology and Metabolism

On June 8, 2020 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported the expansion of the company’s pipeline of small molecule candidates across a range of highly prevalent human diseases (Press release, Nimbus Therapeutics, JUN 8, 2020, View Source [SID1234560891]). These preclinical programs — AMPKβ2 (AMP-activated protein kinase, β2 subunit), CTPS1 (CTP synthase 1), Cbl-b (Cbl proto-oncogene B), and WRN (Werner syndrome ATP-dependent helicase) — represent promising targets across oncology, immunology and metabolism, for which Nimbus’ structure-based discovery approaches are uniquely suited.

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"The additional programs we’re unveiling today are a testament to Nimbus’ exceptional talent, the unwavering support of our investors, and the dynamic scientific collaborations we have built over the past decade," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "Our prolific pipeline reflects the breadth of potential we see for our discovery engine going forward, and a new chapter in Nimbus’ leadership of structure-based drug discovery. We look forward to progressing these programs forward to the clinic within our development organization, which advanced our ACC inhibitor to an early proof of mechanism and is currently progressing our Tyk2 inhibitor toward Phase II."

"With the addition of these targets, we’ve built a pipeline of promising therapeutics for the treatment of patients with diseases that have limited or no therapeutic options," said Peter Tummino, Ph.D., Chief Scientific Officer of Nimbus. "Each of these targets represents the ‘sweet spot’ for Nimbus’ approach — they are known to be fundamental drivers of highly prevalent diseases but have proven difficult for the industry to drug. As we have demonstrated with our progress on HPK1, which is being presented at AACR (Free AACR Whitepaper) this month, we believe our structure-based drug discovery engine can generate the potent, selective small molecule therapeutics needed to move the needle on these targets."

A brief overview of our newly disclosed programs follows:

AMPKβ2 for cellular metabolic regulation
AMPK is a kinase that serves as a critical regulator of energy-sensing and metabolic homeostasis in many tissues. Small molecule activation of AMPK has long been recognized as a potential strategy to treat multiple metabolic disorders and other pathologies. Nimbus’ approach leverages new understandings in AMPK subunit structure to identify activators selective for the AMPKβ2 subtype of the protein to improve glucose and lipid homeostasis, while reducing undesired effects.
CTPS1 for controlling immune activation
CTPS1 is a key enzyme in the pyrimidine synthesis pathway in lymphocytes, making it a drug target for autoimmune diseases and cancer. Selective inhibitors of CTPS1 hold promise for attenuating lymphocyte proliferation and providing effective treatments for T and B cell-driven diseases. Nimbus is using structure-based and other computational chemistry approaches to identify small molecules that are highly potent inhibitors of CTPS1 with selectivity over CTPS2.
Cbl-b for enhancing immune sensitivity in cancer
Cbl-b is an E3 ubiquitin ligase that directs the degradation of signaling proteins across a variety of immune cells. Cbl-b is a well-validated immuno-oncology target, given that Cbl-b knockout mice spontaneously reject tumors with enhanced T and NK cell responses, and Cbl-b deficient T cells can be activated in the absence of co-stimulatory signals. Nimbus is pursuing a structure-based approach to designing inhibitors of Cbl-b that can enhance anti-tumor immunity.
WRN as a selective approach to targeting MSI-high tumors
WRN, a helicase required for DNA replication and repair, is a validated target for treating microsatellite-instability high tumors ("MSI-H tumors"). Pharmacological inhibition of helicases has proven difficult in the past, but WRN is now amenable to structural biology approaches, allowing Nimbus to design both active-site and allosteric inhibitors of WRN that should induce synthetic lethality in tumors with microsatellite instability.

Constellation Pharmaceuticals Announces Appointment of Dr. Jeffrey Humphrey as Chief Medical Officer

On June 8, 2020 Constellation Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company using its expertise in epigenetics to discover and develop novel therapeutics, reported that Jeffrey Humphrey, M.D., will join Constellation Pharmaceuticals as Chief Medical Officer (Press release, Constellation Pharmaceuticals, JUN 8, 2020, View Source [SID1234560890]).

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Dr. Humphrey is a medical oncologist with over twenty years’ experience in drug development. Most recently he was Chief Development Officer at Kyowa Kirin Co., where he oversaw the development of over twenty drug candidates including regulatory submissions leading to three drug approvals in the United States and Europe. He previously served in senior management positions for early and late drug development and medical affairs at Pfizer, Bayer, and Bristol Myers Squibb and completed an oncology fellowship at the National Cancer Institute.

"I am delighted that Jeff is joining Constellation," said Jigar Raythatha, president and chief executive officer of Constellation Pharmaceuticals. "His experience in building teams for successful late-stage drug development will be especially valuable as we continue to make progress in our development programs and advance toward our goal of becoming a fully integrated hematology / oncology company with a sustainable product pipeline. I am pleased to have someone of Jeff’s experience and caliber join the team."

Adrian Senderowicz, M.D., Constellation’s current Chief Medical Officer, will transition to the role of Senior Advisor, where he will continue to advise Constellation on initiatives in research and development, translational science and corporate strategy.

The appointment of Dr. Humphrey as Chief Medical Officer and transition of Dr. Senderowicz to a Senior Advisor of Constellation will be effective as of June 22, 2020.

"I thank Adrian for his leadership and his many contributions to Constellation, especially for conceptualizing the development pathways for our lead assets. I am pleased that we will continue to benefit from Adrian’s experience in the future," Mr. Raythatha concluded.

Dr. Humphrey commented, "I am excited to join Constellation and encouraged by the clinical data generated for its portfolio of product candidates. I look forward to working with the Constellation team to advance CPI-0610 through pivotal trials and potential regulatory approval, as well as to expand and drive forward an exciting pipeline of hematology and oncology therapeutics."

Deciphera Announces Publication of the INVICTUS Pivotal Phase 3 Study of QINLOCK™ (ripretinib) in The Lancet Oncology

On June 8, 2020 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported that The Lancet Oncology has published results from the INVICTUS pivotal Phase 3 study of QINLOCK in patients with fourth-line gastrointestinal stromal tumor (GIST) (Press release, Deciphera Pharmaceuticals, JUN 8, 2020, View Source [SID1234560889]). The INVICTUS study met its primary endpoint, demonstrating a statistically significantly improvement in progression free survival (PFS) in patients randomized to QINLOCK compared with patients receiving placebo. The safety profile observed in INVICTUS was consistent with previously published results, and results from the study were previously presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September 2019.

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"Resistance to approved inhibitors of KIT and PDGFRα remains a clinical challenge in advanced GIST," said lead author Jean-Yves Blay, MD, PhD, Centre Léon Bérard, Unicancer, and Université Claude Bernard. "Our findings demonstrate that QINLOCK exhibited a favorable safety profile and significantly improved PFS over placebo in advanced GIST patients who have received three prior therapies. QINLOCK, a TKI whose activity is not restricted to a specific GIST mutation, establishes a new standard of care for the treatment of fourth-line GIST."

"This publication in Lancet Oncology further validates Deciphera’s switch-control TKI approach and demonstrates QINLOCK’s efficacy in treating patients with fourth-line GIST, a patient population who until the recent U.S. FDA approval of QINLOCK, did not have an approved treatment option," said Matthew L. Sherman, MD, Executive Vice President and Chief Medical Officer of Deciphera. "By broadly inhibiting KIT and PDGFRα kinase signaling through a dual mechanism of action that locks the kinase in the inactive state, QINLOCK prevents downstream signaling and cell proliferation."

The article, entitled "Ripretinib in patients with advanced gastrointestinal stromal tumours (INVICTUS): a double-blind, randomised, placebo-controlled, phase 3 trial" is now available online and will be published in a future print issue of The Lancet Oncology. The publication can be accessed at the following link: http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(20)30168-6/fulltext. The journal also published online a companion Comment article, "A new approach to refractory gastrointestinal stromal tumours with diverse acquired mutations," by Toshirou Nishida, Department of Surgery, National Cancer Center Hospital, Tokyo, Japan and Toshihiko Doi, Department of Experimental Therapeutics, National Cancer Center, Hospital East, Chiba, Japan.

INVICTUS Phase 3 Study

INVICTUS is a Phase 3 randomized, double-blind, placebo-controlled, international, multicenter clinical study evaluating the safety, tolerability, and efficacy of QINLOCK compared to placebo in 129 patients with advanced GIST whose previous therapies have included imatinib, sunitinib, and regorafenib. Patients were randomized 2:1 to either 150 mg of QINLOCK or placebo once daily.

Results of the study were as follows:

QINLOCK demonstrated a median PFS of 6.3 months compared to 1.0 month in the placebo arm and significantly reduced the risk of disease progression or death by 85% (hazard ratio of 0.15, p<0.0001).
QINLOCK demonstrated a median overall survival of 15.1 months compared to 6.6 months in the placebo arm and reduced the risk of death by 64% (hazard ratio of 0.36).
The most common adverse reactions (≥20%) were alopecia, fatigue, nausea, abdominal pain, constipation, myalgia, diarrhea, decreased appetite, palmar-plantar erythrodysesthesia syndrome (PPES), and vomiting. Adverse reactions resulting in permanent discontinuation occurred in 8% of patients, dosage interruptions due to an adverse reaction occurred in 24% of patients and dose reductions due to an adverse reaction occurred in 7% of patients who received QINLOCK.
GIST is a cancer affecting the digestive tract or nearby structures within the abdomen, most often presenting in the stomach or small intestine. GIST is the most common sarcoma of the gastrointestinal tract, with approximately 4,000 to 6,000 new GIST cases each year in the United States and a similar incidence rate in the rest of the world. Most cases of GIST are driven by a spectrum of mutations. The most common primary mutations are in KIT kinase, representing approximately 80% of cases, or in PDGFRα kinase, representing approximately 6% of cases. Current therapies are unable to inhibit the full spectrum of primary and secondary mutations that drive resistance and disease progression. Estimates for 5-year survival range from 48% to 90%, depending on the stage of the disease at diagnosis.

About QINLOCK (ripretinib)

QINLOCK is a tyrosine kinase switch control inhibitor that was engineered to broadly inhibit KIT and PDGFRα mutated kinases by using a unique dual mechanism of action that regulates the kinase switch pocket and activation loop. QINLOCK inhibits initiating and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18 involved in GIST, as well as the primary exon 17 D816V mutation involved in SM. QINLOCK also inhibits primary PDGFRα mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, involved in a subset of GIST.

QINLOCK is approved by the U.S. FDA for the treatment of adult patients with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib.

Deciphera Pharmaceuticals is developing QINLOCK for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumor, or GIST, systemic mastocytosis, or SM, and other cancers. Deciphera Pharmaceuticals has an exclusive license agreement with Zai Lab (Shanghai) Co., Ltd. for the development and commercialization of QINLOCK in Greater China (Mainland China, Hong Kong, Macau, and Taiwan). Deciphera Pharmaceuticals retains development and commercial rights for QINLOCK in the rest of the world.

Indications and Usage

QINLOCK (ripretinib) is a kinase inhibitor indicated for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. For more information visit QINLOCK.com.

Important Safety Information

There are no contraindications for QINLOCK.

Palmar-plantar erythrodysesthesia syndrome (PPES): In INVICTUS, Grade 1-2 PPES occurred in 21% of the 85 patients who received QINLOCK. PPES led to dose discontinuation in 1.2% of patients, dose interruption in 2.4% of patients, and dose reduction in 1.2% of patients. Based on severity, withhold QINLOCK and then resume at same or reduced dose.

New Primary Cutaneous Malignancies: In INVICTUS, cutaneous squamous cell carcinoma (cuSCC) occurred in 4.7% of the 85 patients who received QINLOCK with a median time to event of 4.6 months (range 3.8 to 6 months). In the pooled safety population, cuSCC and keratoacanthoma occurred in 7% and 1.9% of 351 patients, respectively. In INVICTUS, melanoma occurred in 2.4% of the 85 patients who received QINLOCK. In the pooled safety population, melanoma occurred in 0.9% of 351 patients. Perform dermatologic evaluations when initiating QINLOCK and routinely during treatment. Manage suspicious skin lesions with excision and dermatopathologic evaluation. Continue QINLOCK at the same dose.

Hypertension: In INVICTUS, Grade 1-3 hypertension occurred in 14% of the 85 patients who received QINLOCK, including Grade 3 hypertension in 7% of patients. Do not initiate QINLOCK in patients with uncontrolled hypertension. Monitor blood pressure as clinically indicated. Based on severity, withhold QINLOCK and then resume at same or reduced dose or permanently discontinue.

Cardiac Dysfunction: In INVICTUS, cardiac failure occurred in 1.2% of the 85 patients who received QINLOCK. In the pooled safety population, cardiac dysfunction (including cardiac failure, acute left ventricular failure, diastolic dysfunction, and ventricular hypertrophy) occurred in 1.7% of 351 patients, including Grade 3 adverse reactions in 1.1% of patients.

In INVICTUS, Grade 3 decreased ejection fraction occurred in 2.6% of the 77 patients who received QINLOCK and who had a baseline and at least one post-baseline echocardiogram. Grade 3 decreased ejection fraction occurred in 3.4% of the 263 patients in the pooled safety population who received QINLOCK and who had a baseline and at least one post-baseline echocardiogram.

In INVICTUS, cardiac dysfunction led to dose discontinuation in 1.2% of the 85 patients who received QINLOCK. The safety of QINLOCK has not been assessed in patients with a baseline ejection fraction below 50%. Assess ejection fraction by echocardiogram or MUGA scan prior to initiating QINLOCK and during treatment, as clinically indicated. Permanently discontinue QINLOCK for Grade 3 or 4 left ventricular systolic dysfunction.

Risk of Impaired Wound Healing: QINLOCK has the potential to adversely affect wound healing. Withhold QINLOCK for at least 1 week prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of QINLOCK after resolution of wound healing complications has not been established.

Embryo-Fetal Toxicity: QINLOCK can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment and for at least 1 week after the final dose. Because of the potential for serious adverse reactions in the breastfed child, advise women not to breastfeed during treatment and for at least 1 week after the final dose. QINLOCK may impair fertility in males of reproductive potential.

Adverse Reactions: The most common adverse reactions (≥20%) were alopecia, fatigue, nausea, abdominal pain, constipation, myalgia, diarrhea, decreased appetite, PPES, and vomiting. The most common Grade 3 or 4 laboratory abnormalities (≥4%) were increased lipase and decreased phosphate.

The safety and effectiveness of QINLOCK in pediatric patients have not been established.

Administer strong CYP3A inhibitors with caution. Monitor patients who are administered strong CYP3A inhibitors more frequently for adverse reactions. Avoid concomitant use with strong CYP3A inducers.

Heat Biologics Announces FDA Clearance of Investigational New Drug (IND) Application for PTX-35

On June 8, 2020 Heat Biologics, Inc. ("Heat") (NASDAQ:HTBX), a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, including multiple oncology product candidates and a novel COVID-19 vaccine, reported that the U.S. Food & Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PTX-35, developed by Heat’s Pelican Therapeutics subsidiary (Press release, Heat Biologics, JUN 8, 2020, View Source [SID1234560888]). Pelican has near-term plans to initiate its Phase 1 clinical trial in patients with solid tumors.

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PTX-35 is a novel, first-in-class agonist antibody targeting TNFRSF25, also known as death receptor 3 (DR3), a receptor that is preferentially expressed by antigen-experienced T cells. TNFRSF25 agonism leads to activation of antigen-experienced memory CD8+ T cells, which are instrumental for tumor destruction. Preclinical studies have demonstrated PTX-35, in combination with antigen-driven immunotherapies, resulted in enhanced anti-tumor properties, including potent proliferation of antigen-specific T cells, production of effector cytokines and augmented effector immune function.

Rahul Jasuja, PhD, CEO of Pelican Therapeutics, commented, "The mechanism of action for PTX-35 is highly differentiated compared to other co-stimulators. Activation of TNFRSF25 by PTX-35 in preclinical models leads to a more pronounced expansion of tumor antigen-specific cytotoxic, or "killer" T cells, resulting in enhanced tumor growth inhibition and prolonged survival. The team has delivered a robust IND package, demonstrating pre-clinical efficacy along with a favorable safety profile in mice and non-human primates."

Jeff Wolf, CEO of Heat, said, "PTX-35 is a potential first-in-class therapy that may provide additional treatment options for cancer patients. We are close to initiating our first-in-human clinical trial of PTX-35 in multiple solid tumors and look forward to providing further updates as we achieve anticipated milestones. We would like to thank Cancer Prevention Research Institute of Texas (CPRIT) for the $15.2 million grant to support the preclinical work, cGMP manufacturing and Phase 1 development of PTX-35."