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."

Oasmia Pharmaceutical AB Signs Phase 1b Trial Agreement with SAKK, The Swiss Group for Clinical Cancer Research for Evaluation of Docetaxel Micellar

On June 8, 2020 Oasmia Pharmaceutical AB reported that partnered with the Swiss Group for Clinical Cancer Research (SAKK) to conduct the first clinical trial of Oasmia’s docetaxel micellar compound in advanced prostate cancer (Press release, Oasmia, JUN 8, 2020, View Source [SID1234560887]). Prostate cancer in men is a significant health problem worldwide and is a leading cause of cancer death in men. Oasmia’s docetaxel micellar formulation uses Oasmia’s proprietary solubility technology platform enabling the intravenous administration of water-insoluble compounds without traditional solubility enhancers. Oasmia’s solubility technology platform has been utilized in another product, Apealea, which has obtained Marketing Authorization in the EU in 2018, in combination with carboplatin for the treatment of adult patients with first relapse of platinum-sensitive ovarian cancer.

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Docetaxel (INN) is a widely approved anticancer drug with proven activity against a wide range of solid malignancies and represents a standard of care option for advanced prostate cancer. The new Oasmia formulation of docetaxel, to be investigated in this clinical trial, docetaxel micellar, is intended to improve the solubility of docetaxel and avoid the mandatory use of steroid pre-medication.

The Executive Board of SAKK was unanimous in making the decision to carry out the clinical trial. In view of the urgent need for new treatment options in this disease stage, affecting many men all over the world, several Swiss qualified hospital centres have confirmed their participation in this initial study in patients suffering from advanced prostate cancer.

Pursuant to the collaboration agreement, SAKK is the legal sponsor of the project and responsible to conduct the phase 1B trial in accordance with the protocol and applicable rules and regulations. Oasmia’s main role in the project is to supply its formulation of docetaxel and carry the cost of the trial, which are deemed not material to Oasmia.

Prof.Dr. med. Markus Jörger MD-PhD, President of the SAKK Project Group "Developmental Therapeutics", commented on the trial: "The investigational drug docetaxel micellar may be a promising alternative to solvent-based docetaxel formulations in advanced prostate cancer, to avoid mandatory use of steroid premedication".

Francois Martelet, M.D., CEO of Oasmia, comments: "We are pleased to enter into this agreement. It provides an cost-efficient opportunity to test our formulation in collaboration with SAKK, an organization with extensive clinical trial experience, and take us further in our goal to develop new drugs based on our proprietary technology platform XR17."

Quantum Leap Healthcare Collaborative Announces the Selection of Sanofi’s SAR439859 as the oral SERD Backbone Agent in the Endocrine Optimization Pilot Study of the I-SPY 2 TRIAL for Clinically High Risk, Molecularly Low Risk Stage 2/3 Breast Cancer

On June 8, 2020 Quantum Leap Healthcare Collaborative (Quantum Leap) reported an evaluation of SAR439859, an oral estrogen receptor degrader (SERD) in a new I-SPY 2 study arm targeting patients with newly diagnosed hormone receptor-positive (HR+) clinical stage 2/3 invasive breast cancer (Press release, QuantumLeap, JUN 8, 2020, View Source [SID1234560884]).

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This study arm, known as the I-SPY-2 Endocrine Optimization Pilot Protocol (EOP), is focused on patients with molecularly low risk (MammaPrint low risk signature), clinically high risk, hormone receptor positive (HR+), and HER2-negative breast cancer. These patients historically have been excluded from the I-SPY2 trial due to the predicted lack of chemotherapy benefit. However, these patients often have late risk of recurrence (after 5 years), and thus also need novel strategies that are more tolerable and more effective than the current standard. The EOP is a sub-study within the main I-SPY-2 clinical trial utilizing an endocrine therapy backbone for patients whose tumors are predicted to be sensitive to endocrine therapy but for whom chemotherapy provides little or no benefit.

The selective estrogen receptor degrader (SERD) class of compounds have demonstrated benefit in HR+ HER2-negative metastatic breast cancer, including tumors with estrogen receptor activating mutations. They appear to be very well tolerated. Next-generation orally-bioavailable SERDs with improved pharmacokinetic properties are promising potential therapies for HR+ breast cancer. The EOP study will use an oral SERD as the endocrine therapy backbone. Sanofi’s SAR439859 has been selected as the oral SERD for this study. SAR439859 is a potent, orally bioavailable, and selective estrogen receptor (ER) inhibitor that belongs to the selective estrogen receptor degrader (SERD) class of compounds. SAR439859 antagonizes the binding of estradiol to ER but also promotes the transition of ER to an inactive conformation that results in up to 98% receptor degradation at nanomolar concentrations in cellular assays. These dual properties of SAR439859 we hope will translate to a deeper inhibition of ER pathways and a more effective antiproliferative activity in ER-dependent breast cancer cell lines driven by mutant or wild-type ER compared to other ER inhibitors. SAR439859 is an investigational compound and its safety and efficacy have not been evaluated by any regulatory authority.

In EOP, Sanofi’s oral SERD will be evaluated alone and in combination with up to three other investigational agents for a total of up to four study arms. The combination partner agents will be selected based on complementary biology utilizing orthogonal mechanisms of action. I-SPY 2 principal investigator, Dr. Laura Esserman of the University of California San Francisco, stated, "The whole I-SPY team is excited to have Sanofi as a partner in this endeavor. This is an important advance for patients with hormone positive high clinical risk breast cancer profiles who are not responsive to chemotherapy and for whom standard hormonal therapies may not be sufficient. As well, we need endocrine therapies that are easier for women to tolerate. We are committed to finding more effective, less toxic treatments and this partnership with Sanofi will help us to achieve this goal. Using the I-SPY model we can accelerate the development of new cancer treatments and target new and innovative treatments to the patients who will benefit most."

Sanofi will supply SAR439859 and provide financial support for this study. Quantum Leap, as sponsor, will provide the clinical sites and clinical expertise.

ArcherDx Targeting $100M in IPO

On June 7, 2020 ArcherDx reported that it has filed a regulatory document with the US Securities and Exchange Commission to go public with plans to raise up to $100 million (Press release, ArcherDX, JUN 7, 2020, View Source [SID1234560915]).

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The Boulder, Colorado-based company has not priced the offering or said how many shares it expects to offer in its Form S-1 filed on Friday. JP Morgan, Bank of America Securities, Stiffel, and Evercore ISI are listed as the proposed offering’s underwriters. ArcherDx plans to list its stock on the Nasdaq under ticker symbol "RCHR".

Founded in 2013, the company has leveraged next-generation sequencing to develop and commercialize more than 325 research-use-only products that it has sold to more than 300 academic and reference laboratories and more than 50 biopharmaceutical firms and contract research organizations in 40 countries, it said in its Form S-1.

Its RUO product lines include DNA-based VariantPlex panels for detecting copy number variations, single-nucleotide variants, and indels; RNA-based FusionPlex panels for generating target-enriched libraries to characterize gene fusions, splice variants, SNVs, indels, and relative expression levels; DNA-based LiquidPlex panels for targeted NGS of ccfDNA, cfDNA, and ctDNA from 28 genes associated with solid tumor type cancers; and RNA-based Immunoverse panels to characterize the human immune repertoire. The company collectively refers to the panels as personalized cancer monitoring (PCM).

ArcherDx also offers Stratafide, a pan-solid tumor diagnostic test designed to identify actionable genomic alterations in tissue or blood samples, and it is developing in vitro diagnostics and provides services for clinical applications.

It intends to submit Stratafide to the US Food and Drug Administration this year and PCM in the future for approval and/or clearance to be marketed as in vitro diagnostics.

The firm said in its SEC document that proceeds from its IPO will go toward R&D and the regulatory submission and commercialization of Stratafide. Proceeds will also be used for working capital and general corporate purposes, including sales and marketing activities, operating expenses, and capital expenditures, as well as possible acquisitions and investments in complementary businesses, technologies, and other assets.

Recent transactions by the company include a collaboration announced in May with AstraZeneca to develop assay to detect minimal residual disease in patients with early-stage non-small cell lung cancer. The month before that, it and Bayer announced a collaboration to develop and commercialize an NGS-based companion diagnostic for Bayer’s Vitrakvi (larotrectinib).

In April it said it reached a two-part deal with Premier to deliver personalized genomic testing to patients, and in January ArcherDx and Illumina inked a partnership to broaden access to NGS-based oncology testing, including companion diagnostics, personalized monitoring, and new recurrence surveillance IVDs.

The company had $50.6 million in total revenues in 2019, up from $28.5 million in 2018. It had a net loss of $50 million in 2019 compared to a net loss of $5.6 million in 2018.

This past December, it raised $55 million in a Series C financing round, and in May 2019 it raised $60 million in a Series B financing.

Through the first three months of 2020, ArcherDx recorded $14.8 million in revenues and a net loss of $19.3 million. It had $36.8 million in cash and cash equivalents as of March 31, 2020.

Jason Myers is the firm’s president and CEO while Josh Stahl is its chief scientific officer and chief operating officer.