On April 16, 2019 Aprea Therapeutics, a privately held, clinical stage biopharmaceutical company developing novel anticancer therapies targeting the tumor suppressor protein p53, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to APR-246 for the treatment of patients with MDS having a TP53 mutation (Press release, Aprea, APR 16, 2019, View Source [SID1234535157]). In addition, FDA has also granted Orphan Drug Designation to APR-246 for treatment of MDS.

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"The granting of Fast Track designation and Orphan Drug Designation by FDA for APR-246 in TP53 mutated MDS underscores the significant unmet medical need in this disease," said Christian S. Schade, President and Chief Executive Officer of Aprea. "With our Phase 3 clinical study in MDS underway, we look forward to continuing our productive dialogue with FDA and bringing APR-246 to patients as soon as possible."

The FDA’s Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to address unmet medical needs. A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug’s development, review and potential approval. In addition, the Fast Track program allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as for Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be submitted for review.

Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the U.S. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, tax credits related to clinical trial expenses, and an exemption from FDA user fees.

About p53 and APR-246

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

APR-246 has been shown to reactivate mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated pre-clinical anti-tumor activity in a wide variety of solid and hematological (blood) tumors, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase I/II clinical program with APR-246 has been completed, demonstrating a favorable safety profile, biological activity and clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) represents a spectrum of hematopoietic stem cell malignancies in which bone marrow fails to produce sufficient healthy blood cells. Approximately 30-40% of MDS patients progress to acute myeloid leukemia (AML) and mutation of the p53 tumor suppressor protein is thought to contribute to disease progression. Mutations in p53 are found in approximately 20% of MDS and AML patients and are associated with poor overall prognosis.

On April 16, 2019 IMV Inc. ("IMV" or the "Corporation") (Nasdaq: IMV; TSX: IMV), a clinical stage immuno-oncology company, reported the following statement regarding recent market activity (Press release, IMV, APR 16, 2019, View Source [SID1234535155]).

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In recent weeks, shares of IMV Inc. have come under unwarranted market pressure and the management team believes it is prudent to provide a mid-quarter update on the health of the business and the Company’s upcoming Q2 clinical milestones.

"From a financial and clinical results standpoint, IMV has recently achieved noteworthy milestones and, based on clinical results observed thus far, our long term outlook remains unchanged and very promising," said Frederic Ors, Chief Executive Officer. "We have strengthened our balance sheet and expanded our shareholder base through a recent equity offering completed with Wells Fargo as lead underwriter. In addition, IMV has also reported promising clinical results from the phase 2 cohort of the DECIDE clinical study, which we believe confirm the potential activity of DPX-Survivac as monotherapy."

Phase 2 Cohort of the DECIDE Clinical Study in Ovarian Cancer

As reported earlier this year, IMV’s latest clinical results update indicated that six patients receiving DPX-Survivac monotherapy with intermittent low-dose cyclophosphamide (mCPA) reached the first CT scan assessment and key related findings were as follows:

83% of the subjects (5 of 6) showed stable disease (SD), including two tumor regressions;
80% (4 of 5) of those with stable disease were in subjects with a lower baseline tumor burden (BTB) of less than 5 centimeters, which also included the two tumor regressions.
In earlier stages of this trial, durable clinical responses occurred after 140 days. As of March 25, 2019, these responses had endured for 20 months or more. Additional data at the 140-day mark of this cohort will be available by the end of the first half of 2019. The amended phase 2 cohort of the DECIDE trial focuses on patients with low tumor burden (less than 5 centimeters targeting the enrollment of at least 16 additional patients at numerous sites in the U.S. and Canada.

Phase 2 Study in Combination with KEYTRUDA in Relapsed/Refractory DLBCL (SPIREL)

As of April 5, 2019, ten patients have been enrolled and treated across four different clinical sites in Canada. Additional patients are being screened and IMV expects to report updated clinical data at about the same time than the bi-annual International Conference on Malignant Lymphoma, which will be held in Lugano Switzerland starting June 18, 2019.

Phase 2 Basket Trial in Combination with KEYTRUDA in Multiple Solid Tumors

Screening and enrollment of patients is ongoing at multiple clinical sites across the U.S. and Canada for five cohorts of patients with bladder, liver (hepatocellular carcinoma), ovarian, or non-small cell lung (NSCLC) cancers as well as tumors shown to be positive for the microsatellite instability high (MSI-H) biomarker.

The first patients have been dosed in the ovarian and lung cancer cohorts and IMV expects to report preliminary clinical results on several of the solid tumor indications before the end of 2019.

The following table indicates IMV expected milestones between now and the first half of 2020 as at the date of this release.

Milestones

Key dates
Initiation of Basket trial in 5 solid tumor indications
September 2018 x

First preliminary Phase 2 clinical results with Merck Keytruda in DLBCL
September 2018 x

Phase 1b/2 clinical results in Ovarian with Incyte
December 2018 x

Meeting with FDA on Ovarian cancer program
December 2018 x

Dosing of first patient in Basket trial
March 2019 x

Phase 2 monotherapy results in Ovarian – ASCO (Free ASCO Whitepaper) June 2019
Phase 1/1b monotherapy long term follow-up – ASCO (Free ASCO Whitepaper) June 2019
Phase 2 clinical results with Merck Keytruda in DLBCL – ICML June 2019
Preliminary clinical results Basket trial in 5 indications H2 2019
Potential registration trial in Ovarian and/or DLBCL for FDA
accelerated/breakthrough designation

H2 2019
Top line clinical results for Basket trial H1 2020
Meeting with FDA on potential accelerated registration trial from Basket trial H1 2020

On April 16, 2019 Turning Point Therapeutics, Inc. (Nasdaq:TPTX), a clinical-stage precision oncology company designing and developing novel drugs to address treatment resistance, reported the pricing of its initial public offering of 9,250,000 shares of its common stock at a price to the public of $18.00 per share (Press release, Turning Point Therapeutics, APR 16, 2019, View Source [SID1234535153]). The gross proceeds to Turning Point Therapeutics from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $166.5 million.

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The shares are expected to begin trading on the Nasdaq Global Market on April 17, 2019 under the symbol "TPTX". The offering is expected to close on April 22, 2019, subject to satisfaction of customary closing conditions. In addition, Turning Point Therapeutics has granted the underwriters a 30-day option to purchase up to an additional 1,387,500 shares of common stock at the initial public offering price less underwriting discounts and commissions.

Goldman Sachs & Co. LLC and SVB Leerink are acting as joint book-running managers for the offering. Wells Fargo Securities is also serving as a joint book-running manager. Canaccord Genuity is acting as lead manager.

The offering is being made only by means of a prospectus. Copies of the final prospectus related to the offering, when available, may be obtained from:

Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, via telephone: 1-866-471-2526 or via email: [email protected];
SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 1-800-808-7525, ext. 6132 or by email at [email protected]; or
Wells Fargo Securities, LLC, Attention: Equity Syndicate Department, 375 Park Avenue, New York, NY 10152, or by telephone at 1-800-326-5897, or by email at [email protected].
Registration statements relating to these securities have been filed with the Securities and Exchange Commission and became effective on April 16, 2019. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On April 16, 2019 Agilent Technologies Inc. (NYSE: A) reported that the U.S. Food and Drug Administration has updated its approval of the company’s PD-L1 IHC 22C3 pharmDx assay (Press release, Agilent, APR 16, 2019, View Source [SID1234535152]).

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The assay can now be used as a companion diagnostic to identify a broader range of patients with stage III or metastatic non-small cell lung cancer (NSCLC) for first-line treatment with KEYTRUDA, a targeted anti-PD-1 therapy manufactured by Merck & Co. (known as MSD outside the United States and Canada).

The FDA previously approved the assay to identify metastatic NSCLC patients whose tumors express PD-L1 Tumor Proportion Score (TPS) ≥ 50% for first-line treatment with KEYTRUDA. Now, patients with stage III NSCLC who are not candidates for surgical resection or definitive chemoradiation, or metastatic NSCLC, and whose tumors express PD-L1 TPS ≥ 1% are eligible for first-line treatment. This expanded indication enables pathologists to identify a larger population of previously untreated patients who are now eligible for treatment with KEYTRUDA.

"Anti-PD-1 therapies are a promising treatment class for many cancer types, and PD-L1 testing provides key information to physicians managing stage III or metastatic NSCLC patients," said Sam Raha, president of Agilent’s Diagnostics and Genomics Group. "The updated FDA approval of PD-L1 IHC 22C3 pharmDx broadens the scope of patients that can be identified for first-line treatment with KEYTRUDA and offers new hope to the many patients diagnosed with stage III or metastatic NSCLC. By expanding the use of PD-L1 IHC 22C3 pharmDx, Agilent strives to continue our legacy of pioneering companion diagnostics to support the launch of landmark therapies."

Lung cancer is the leading cause of cancer-related mortality in the United States, with an estimated incidence of 154,000 deaths in 2018 alone.1 Among these cases, NSCLC accounts for nearly 85% of all diagnoses, and carries a 5-year survival rate of 15%.2 PD-L1 is a critical biomarker for response to anti-PD-1 therapy, and pathology labs play an important role in identifying appropriate patients for these treatments.

Agilent’s PD-L1 IHC 22C3 pharmDx is the first and only companion diagnostic that has been clinically validated and approved to identify NSCLC patients eligible for KEYTRUDA.

KEYTRUDA is a humanized monoclonal antibody that increases the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes, which may affect both tumor cells and healthy cells. KEYTRUDA and other targeted immunotherapies are revolutionizing cancer treatment, and their therapeutic value is being demonstrated across a growing list of cancer types.

Agilent is a worldwide leader in partnering with pharmaceutical companies to develop immunohistochemical-based diagnostics for cancer therapy. Agilent developed PD-L1 IHC 22C3 pharmDx in partnership with Merck & Co. PD-L1 IHC 22C3 pharmDx also helps physicians identify cervical cancer, gastric or GEJ adenocarcinoma, and urothelial carcinoma patients for treatment with KEYTRUDA. PD-L1 expression in NSCLC tissues is interpreted using Tumor Proportion Score (TPS). PD-L1 expression in urothelial carcinoma, cervical cancer, and gastric or GEJ adenocarcinoma tissues is interpreted using Combined Positive Score (CPS).

On April 16, 2019 NeoImmuneTech, Inc. (NIT), a T cell-focused therapeutics company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Hyleukin-7TM, a T cell amplifier, in development for the treatment of Idiopathic CD4+ Lymphocytopenia (ICL) (Press release, NeoImmuneTech, APR 16, 2019, View Source [SID1234535151]). Hyleukin-7 also received ODD from the European Medicines Agency in 2017, and it is the first and only agent that has obtained ODD for ICL.

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ICL was first defined in 1992 by the Centers for Disease Control and Prevention, and is a rare disease in which patients present persistently low CD4+ T lymphocyte counts without human immunodeficiency virus (HIV) infection or any other cause of immunodeficiency.

"Patients with ICL frequently suffer from severe and recurrent opportunistic infections and are at high risk for developing certain types of cancer. Currently, no specific treatment for ICL exists. As such, there is high medical need for therapies that can increase CD4+ T cells in ICL patients," said NgocDiep (Diep) Le, M.D., Ph.D., NIT’s Executive Vice President and Chief Medical Officer. "We are delighted that the FDA recognized the potential of Hyleukin-7 as an innovative and transformative treatment for ICL and look forward to conducting a clinical trial in this patient population."

In the phase 1 trial in healthy subjects and multiple ongoing dose-escalation trials in cancer patients, Hyleukin-7 showed a well-tolerated safety profile and dose-dependent increases of CD4+ and CD8+ T lymphocyte counts. NIT has been also actively conducting and planning multiple proof-of-concept clinical trials to develop Hyleukin-7 as an immune-oncology (IO)-enabling drug in combination with other IO therapeutics.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. Receiving ODD may help to expedite and reduce the cost of development, approval and commercialization of a therapeutic agent.

About Hyleukin-7TM
Hyleukin-7TM (rhIL-7-hyFc, NT-I7), an immuno-oncology agent, is a T cell growth factor composed of a covalently linked homodimer of engineered Interleukin-7 (IL-7) molecule, biologically fused with the proprietary long-acting platform – hyFc. IL-7 is known to be a critical factor for T cells homeostasis, acting to increase both the number and functionality of T cells. Hyleukin-7 amplifies and reinvigorates persistent T cell immunity in the treatment of patients with cancer and lymphopenia, thus providing unique opportunities for immuno-oncology (IO) combination strategies. Hyleukin-7 is being developed as an "IO enabling" therapy to harness T cell immunity in combination with current cancer treatments such as anti-PD-(L)1 agents or chemo/radiotherapy as well as next generation IO therapeutics.