On June 29, 2020 The U.S. Food and Drug Administration approved Keytruda (pembrolizumab) for intravenous injection for the first-line treatment of patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (Press release, US FDA, JUN 29, 2020, View Source [SID1234561533]). This marks the first immunotherapy approved for this patient population as a first-line treatment and which is administered to patients without also giving chemotherapy.

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MSI-H and dMMR tumors contain abnormalities that affect the proper repair of DNA inside the cell. The frequency of MSI-H varies across tumor types and stages, and approximately 5% of patients with metastatic colorectal cancer have MSI-H or dMMR tumors.

"Metastatic colorectal cancer is a serious and life-threatening disease with a poor prognosis. Available current therapy with chemotherapy combinations and other biologics are associated with substantial toxicity," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. "Having a non-chemotherapy option available for selected patients is a noteworthy paradigm shift in treatment."

Keytruda works by targeting the cellular pathway of proteins found on the body’s immune cells and some cancer cells, known as PD-1/PD-L1. By blocking this pathway, Keytruda may help the body’s immune system fight the cancer cells and provide a benefit in patients with MSI-H or dMMR metastatic colorectal cancer. The FDA previously approved Keytruda to treat other types of cancer.

The FDA’s approval for this indication was based on the results of one multicenter, international, open-label, active-controlled, randomized trial that compared Keytruda with chemotherapy treatment in 307 patients with MSI-H or dMMR metastatic colorectal cancer. The study demonstrated a statistically significant improvement in progression-free survival (PFS) as assessed by blinded independent review. Median PFS was 16.5 months in the Keytruda group and 8.2 months in the standard of care group. Longer-term analysis is needed to assess for an effect on survival.

Common side effects of Keytruda include fatigue, musculoskeletal pain, decreased appetite, itchy skin (pruritus), diarrhea, nausea, rash, fever (pyrexia), cough, difficulty breathing (dyspnea), constipation, pain, and abdominal pain. Keytruda can cause serious conditions known as immune-mediated side effects, including inflammation of healthy organs such as the lungs (pneumonitis), colon (colitis), liver (hepatitis), endocrine glands (endocrinopathies) and kidneys (nephritis). Patients who experience severe or life-threatening infusion-related reactions should stop taking Keytruda. Women who are pregnant should be advised that Keytruda may cause harm to a developing fetus. Women who are breastfeeding should not take Keytruda because it may cause harm to a breastfed child.

The FDA granted this application Priority Review, which directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. This review also used the Real-Time Oncology Review, which streamlines data submission prior to the filing of the entire marketing application, the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment of an application, and Summary Level Review, which allows FDA to rely on qualified data summaries to support approval of a supplemental application. The FDA collaborated with international agency counterparts on the review of this application as part of Project Orbis.

The FDA granted this approval of Keytruda to Merck & Co.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On June 29, 2020 Halozyme Therapeutics, Inc. (NASDAQ: HALO) reported that the U.S. Food and Drug Administration (FDA) has approved Roche’s Phesgo (pertuzumab, trastuzumab, and hyaluronidase-zzxf) injection, a fixed-dose combination (FDC) of Perjeta and Herceptin with Halozyme’s ENHANZE technology, administered subcutaneously in combination with intravenous (IV) chemotherapy, for the treatment of eligible patients with early and metastatic HER2-positive breast cancer (Press release, Halozyme, JUN 29, 2020, View Source [SID1234561532]). This is the first time a product has been approved combining two monoclonal antibodies that can be administered by a single subcutaneous injection utilizing Halozyme’s ENHANZE technology.

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"We are excited that HER2-positive breast cancer patients in the U.S. will now have the option to receive this important therapy in a meaningfully shorter period of time," said Dr. Helen Torley, president and chief executive officer. "This subcutaneous delivery was shown to be preferred to IV administration by 85% of patients in the PHranceSCa study due to less time in the clinic and more comfortable treatment administration. Phesgo is the second product to receive FDA approval this year, and the first demonstrating the ability to combine two monoclonal antibodies, utilizing our ENHANZE technology."

Phesgo is available in single-dose vials and can be administered in approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose.(1) This is compared to approximately 150 minutes for a sequential infusion of a loading dose of Perjeta and Herceptin using the standard IV formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines.(2,3) Phesgo can be administered by a healthcare professional in a treatment center or at a patient’s home.

The approval is based on results from the pivotal phase III FeDeriCa study, which met its primary endpoint, with Phesgo showing non-inferior levels of Perjeta in the blood during a given dosing interval (Ctrough), when compared to IV administration of Perjeta. The safety profile of Phesgo with chemotherapy was comparable to IV administration of Perjeta plus Herceptin and chemotherapy, and no new safety signals were identified, including no meaningful difference in cardiac toxicity. The most common adverse events in both arms were alopecia, nausea, diarrhea and anemia.(1,4)

The phase II PHranceSCa study showed that 85% (136/160) of people receiving treatment for HER2-positive breast cancer preferred treatment under the skin to IV administration due to less time in the clinic and more comfortable treatment administration.(1)

About ENHANZE Technology
Halozyme’s proprietary ENHANZE drug-delivery technology is based on its patented recombinant human hyaluronidase enzyme (rHuPH20). rHuPH20 has been shown to remove traditional limitations on the volume of biologics that can be delivered subcutaneously (just under the skin). By using rHuPH20, some biologics and compounds that are administered intravenously may instead be delivered subcutaneously. ENHANZE may also benefit subcutaneous biologics by reducing the need for multiple injections. This delivery has been shown in studies to reduce health care practitioner time required for administration and shorten time for drug administration.

On June 29, 2020 Lantern Pharma (NASDAQ: LTRN), a clinical stage biotechnology company focused on leveraging artificial intelligence ("A.I."), machine learning and genomic data to streamline the drug development process and to identify the patients that will benefit from its targeted oncology therapies, reported that it surpassed the milestone of 450 million curated data points being utilized in its proprietary A.I. and machine learning-powered platform, RADR (Response Algorithm for Drug Positioning and Rescue) (Press release, Lantern Pharma, JUN 29, 2020, View Source [SID1234561531]).

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RADR leverages genomic, transcriptomic, clinical and drug sensitivity data points across more than 145 drugtumor interactions to predict the potential response cancer patients will have to potential drugs, therefore enabling a more personalized approach to therapy that is aimed at better outcomes. Lantern is establishing collaborations and partnerships to expand the functionality of RADR, including algorithms that can operate 200 to 300 percent faster than its existing algorithms, enabling the company to develop robust, gene signatures that can be used to guide patient enrollment in trials and as a companion diagnostic (CDx). During the most recent data enrichment campaign, Lantern focused on significantly increasing the depth and amount of data for: nonsmall cell lung cancer, ovarian cancer, glioblastoma, and gliomas. Reaching this milestone of over 450 million curated data points for oncology drug development will bring greater precision and speed in helping Lantern with its objective of personalizing oncology therapy with reduced risk and cost.

"Our approach in leveraging machine learning to develop biologically relevant, multi-gene signatures in days or weeks allows our team to more efficiently review and test novel insights about the complex mechanisms that can drive patient response to a drug," said Panna Sharma, CEO of Lantern Pharma.

As a pioneer in the application of machine learning to oncology-focused drug development and clinical trial design, Lantern has published gene signatures derived from RADR as posters and presentations at both ASCO (Free ASCO Whitepaper) and AACR (Free AACR Whitepaper). Lantern’s pipeline of compounds includes one candidate in an active Phase 2 clinical trial for metastatic, hormone-refractory prostate cancer using a genomic signature for patient selection; another candidate in preparation for a Phase 2 clinical trial in non-small cell lung cancer in a targeted patient population; and a third candidate in two pre-clinical programs for biomarker-defined solid tumors and glioblastoma.

Mr. Sharma continued, "Because of the increasing availability of large-scale biomarker, genomic and patient data, and rapidly maturing technologies like artificial intelligence and machine learning, oncology is undergoing a monumental shift in the way cancer drugs are discovered, developed, studied, targeted, and commercialized. Lantern is at the forefront of this transformation. For Lantern, exceeding 450 million data points is a significant milestone in our work, and demonstrates our commitment to leveraging paradigm changing technologies that transform oncology drug development with the ultimate objective of cost-effectively personalizing treatment for patients."

Lantern Pharma is ahead of the initial platform development schedule, reaching 400 million data points by the end of 2020, which puts Lantern on track to reach over 1 billion data points earlier than expected. The developmental focus on increasing the number of data points, and improving the performance of the algorithms is expected to yield additional targeted indications for Lantern’s current pipeline of drugs, and also help to uncover additional compounds and therapies that can be in-licensed or acquired and subsequently developed in a more efficient manner that leverages the insights from Lantern’s data-driven, A.I.-enabled approach.

The market opportunity for RADR as a platform for the development of targeted oncology therapies is significant. The highly scalable RADR platform can be leveraged in multiple real-world applications, in addition to drug development, including: identifying potential drug combinations, predicting synergies with immune-oncology agents, developing companion diagnostics (CDx) and evaluating compounds for therapeutic efficacy and optimal positioning. Lantern is a pioneer in the adoption and implementation of data-driven and machine-learning enabled processes for drug development. The intersection of A.I., machine learning and genomics is considered a rapidly growing trend as researchers and investors turn to big data approaches to transform the cost, risk and timeline of oncology drug development.

"Lantern Pharma is using genomics, machine-learning and big data in an effort to develop potentially life-saving cancer drugs, and our ability to fulfill this mission with greater efficiency and speed will be enhanced by focusing on the overall strength and scale of our platform," concluded Mr. Sharma.

On June 29, 2020 Ferring Pharmaceuticals reported the successful launch of its inaugural Swiss Franc Bond offering for a benchmark amount of CHF 270 million (Press release, Ferring, JUN 29, 2020, View Source [SID1234561530]). This inaugural senior unsecured bond transaction issued by Ferring Holding SA has a fixed coupon rate of 1.05% per annum. The bond was issued at a price of 100.097% with a 5-year maturity on 9th July 2025, and will be listed on the SIX Swiss Exchange.

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The offering attracted much interest from high-quality institutional investors and banks, demonstrating recognition of the company’s successful track record and solid cash generation. The company is rated as BBB (Credit Suisse) and Baa- (Fedafin), both with a stable outlook.

Dominic Moorhead, Chief Financial Officer of Ferring Pharmaceuticals, said "We are very happy with the successful outcome of our inaugural bond offering – and for a private company, entering a public capital market is a significant step. This is strong acknowledgement of the company’s achievements, and will enable us to firmly move forward with our next phase of growth."

The net proceeds from the transaction will be used for general corporate purposes including repayment of debt. The issue was lead-managed by Credit Suisse AG and Basler Kantonalbank, with Banque Cantonale Vaudoise acting as co-manager.

On June 29, 2020 Aptose Biosciences Inc. (Nasdaq: APTO; TSX: APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported that the U.S. Food and Drug Administration (FDA) completed its review of the company’s Investigational New Drug (IND) application and has granted IND allowance for the initiation of a Phase 1a/b clinical study of CG-806, the company’s highly potent, oral FLT3/BTK inhibitor, in patients with acute myeloid leukemia (AML) (Press release, Aptose Biosciences, JUN 29, 2020, View Source [SID1234561526]). CG-806 is currently in a Phase 1 dose escalation study in patients with B-cell malignancies, including chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphomas (NHL), who have failed or are intolerant to current therapies.

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"Our strategy was to identify a starting dose of CG-806 that we believe could be therapeutically active in critically ill patients with relapsed or refractory AML. We are pleased that the FDA has allowed us to initiate a clinical trial in these patients at a starting dose of 450mg BID. Despite recent advances in the treatment of AML, many patients relapse or remain refractory to current therapies leading to a poor overall prognosis," said Rafael Bejar, M.D., Ph.D., Senior Vice President and Chief Medical Officer. "Based on strong preclinical evidence of CG-806’s activity against AML – including demonstration of mutation-agnostic and genotype-agnostic potency, particularly compared against other FLT3 inhibitors, and its ability to safely cure AML in murine leukemia models – we believe CG-806 offers hope to the fragile and difficult-to-treat AML patient population. We continue to dose escalate in an ongoing study in patients with CLL and other B cell cancers, and are eager to advance this separate AML protocol through Institutional Review Boards at key clinical sites, recruit appropriate AML patients, and initiate dosing as soon as possible."

Aptose intends to initiate the Phase 1 a/b study in the second half of 2020 in AML patients who have relapsed, are resistant or refractory to current treatment.

About CG-806

CG-806 is an oral, first-in-class FLT3/BTK cluster selective kinase inhibitor and is in Phase 1 clinical studies for the treatment of hematologic malignancies. This small molecule demonstrates potent inhibition of wild type and all mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), cures animals of AML in the absence of toxicity in murine leukemia models, and represents a potential best-in-class therapeutic for patients with AML and other myeloid malignancies. Likewise, CG-806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser (C481S) mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG-806 may be developed for various B cell malignancy patients (including CLL/SLL, FL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent or other non-covalent BTK inhibitors. Because CG-806 targets key kinases/pathways operative in malignancies derived from the bone marrow, it is in development for B-cell cancers and AML.