On December 13, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported the closing of its previously announced underwritten public offering of 3,820,000 shares of its common stock at a public offering price of $22.25 per share (Press release, Rocket Pharmaceuticals, DEC 13, 2019, View Source [SID1234552372]). The gross proceeds to Rocket from the offering are expected to be approximately $84,995,000 million, before deducting the underwriting discounts and commissions and other offering expenses.

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Rocket intends to use the net proceeds from this offering to further fund the development of our pipeline of gene therapies for rare diseases, to support the buildout of in-house manufacturing capabilities, and for general corporate purposes.

J.P. Morgan, Cowen and Evercore ISI acted as joint-bookrunning managers for the offering. LifeSci Capital LLC acted as co-manager.

The public offering was made by Rocket pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC. A final prospectus supplement relating to and describing the terms of this offering was filed with the SEC on December 11, 2019. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or from Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by email at [email protected] or by telephone at (833) 297-2926, or from Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, by telephone at 1-888-474-0200 or by e-mail at [email protected]. You may also obtain these documents free of charge by visiting the SEC’s website at www.sec.gov.

This press release does 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 an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 13, 2019 Susan G. Komen, the world’s leading breast cancer organization, reported the results of a clinical trial, led by Komen Scholar Dr. Bryan Schneider, that moves us closer to predicting recurrence and informing the treatment of triple negative breast cancer (TNBC) (Press release, Susan G Komen, DEC 13, 2019, View Source [SID1234552371]). The results were presented at the 2019 San Antonio Breast Cancer Symposium.

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Schneider and his colleagues conducted a clinical trial at over 25 sites that looked at circulating tumor cells (CTCs) and circulating DNA (ctDNA) from the blood to determine their effectiveness at predicting recurrence, and the prognosis for patients with TNBC. TNBC has limited treatment options and a poor prognosis for patients.

"The results showed that breast cancer recurrence could be predicted for these patients and physicians may be able to use this information in the future to identify patients who are at high risk for recurrence," Schneider said. "Through this approach, researchers can also identify patients who have an incredibly good prognosis leading to the potential for future studies focused on novel de-escalation approaches for some TNBC patients."

A key aspect of this study is that over 25% of the patients enrolled were African-American women – a population that is more likely to be diagnosed with the difficult to treat TNBC. Additionally, more than 25% of the patients enrolled were under the age of 35, which is another population disproportionally impacted by TNBC.

The results of the trial have led to a second clinical trial to refine how liquid biopsy can inform treatment for TNBC patients. "The potential promise of knowing more about your risk and options from a simple blood test is phenomenal," says Schneider.

Victoria Wolodzko, senior vice president of Mission at Susan G. Komen, added, "We have been talking about the potential of liquid biopsy for a long time and we are excited to see this technology working for breast cancer patients. We look forward to the results of the follow-up trial that will lead to better treatment strategies for TNBC patients and give physicians and patients better tools to guide precision medicine."

On December 13, 2019 Dune Medical Devices, (Alpharetta, GA), a medical device company focused on improving the effectiveness and outcome of cancer therapy through real-time tissue characterization, is presenting cutting-edge data and an expanded technology platform during the 2019 San Antonio Breast Cancer Symposium (SABCS) December 10-14 (Press release, Dune Medical Devices, DEC 13, 2019, View Source [SID1234552370]).

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Dune Medical is known primarily for its MarginProbe device for intraoperative margin assessment, shown to reduce re-excisions in breast conserving surgery by over 50%, and has been used in over 20,000 procedures.

The abstract, entitled "Feasibility of incorporating miniaturized, flexible radiofrequency (RF) sensors in a breast biopsy needle for accurate real-time characterization of benign and malignant tissue," reports on data collected at three medical facilities in Israel and will be presented by Avihai Lachman, Vice President Research & Development, Dune Medical.

It has been well established that physiologic differences between benign and malignant tissues are reflected in their electrical properties. "Access to real-time tissue properties during the biopsy procedure has the potential for increasing accuracy by enabling the most suspicious tissue to be sampled, and by providing the tissue characterization to pathology for comparison with histologic findings,” said Dr. Tanir Allweis, Director, Breast Health Center, Kaplan Medical Center, Rehovot, Israel.

The results of this feasibility study demonstrate that breast tissue can be characterized in real-time during the biopsy procedure, providing constant information on the makeup of the tissue with which the needle is in contact. With a sensitivity of 85% and a specificity of 99%, this data affirms that radiofrequency spectroscopy is applicable in a multitude of applications, leading not only to more accurate tissue sampling and diagnosis, but expanded opportunities for delivering targeted therapies in the future.

"The ability to accurately characterize tissue properties in real-time during the biopsy procedure is a game changer. Studies show that there is a 25% discordance rate for breast biopsy diagnostic interpretation, which can affect how a patient is managed. Providing physicians with the ability to sample the most suspicious tissue will ensure that the most relevant biopsy cores are retrieved, enabling the most accurate diagnosis," states Lori Chmura, Dune Medical CEO.

The development of the Smart Biopsy device was made possible through the coveted European Union Horizon 2020 research grant awarded in 2016, and the technology is currently being tested in vivo. In presenting this compelling data at SABCS, Dune Medical reaches a major milestone, marking significant achievement toward its mission to make RF spectroscopy technology available across the oncology spectrum.

The San Antonio Breast Cancer Symposium, a collaboration of the American Society for Radiation Oncology (ASTRO) and the Society of Surgical Oncology (SSO), has brought the international oncology community together to share and collaborate about the latest advancements and research in breast cancer since 1977. Dune Medical will be presenting December 13, from 5-7 pm, during Poster Session 5, Ongoing Trials, Program #OT3-04-01.

On December 13, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended broadening the existing marketing authorisation for Darzalex▼ (daratumumab) to include the use of daratumumab in combination with bortezomib, thalidomide and dexamethasone (VTd) for the treatment of adult patients with newly diagnosed patients with multiple myeloma who are eligible for autologous stem cell transplant (ASCT) (Press release, Janssen Pharmaceutica, DEC 13, 2019, View Source [SID1234552369]).

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The Positive Opinion is supported by data from Part 1 of the Phase 3 CASSIOPEIA (MMY3006) study, published in The Lancet3 in June 2019, and presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting. Additional information about this study can be found at www.ClinicalTrials.gov (NCT02541383).

"Today’s Opinion takes us a step closer to offering the first daratumumab combination regimen to transplant eligible patients, redefining treatment for those people newly diagnosed with multiple myeloma," said Dr Patrick Laroche, Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag. "We are committed to delivering advances in multiple myeloma care, including providing innovative treatment options that meet the evolving needs of people living with this disease."

Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology at Janssen Research & Development, LLC., commented: "Our robust clinical development programme continues to demonstrate that daratumumab provides a foundation for the treatment of patients with multiple myeloma across the treatment continuum."

The CHMP’s Positive Opinion comes after the US Food and Drug Administration’s approval in September 2019. It will now be reviewed by the European Commission, which has the authority to grant marketing authorisation for medicines in the European Economic Area.

#ENDS#

In Europe, daratumumab is indicated:4

in combination with lenalidomide and dexamethasone or with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant
in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy
as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy
About the CASSIOPEIA Trial5

The randomised, open-label, multicentre, Phase 3 study is sponsored by the French Intergroupe Francophone du Myelome in collaboration with the Dutch-Belgian Cooperative Trial Group for Hematology Oncology and Janssen Research & Development, LLC. The study included 1,085 newly diagnosed patients with previously untreated, symptomatic multiple myeloma who were eligible for high-dose chemotherapy and stem cell transplant. In the first part of the study, patients were randomised to receive induction treatment with VTd alone or in combination with daratumumab, high-dose therapy and ASCT, and consolidation therapy with VTd alone or in combination with daratumumab. The primary endpoint in this part of the study is the proportion of patients who achieve an sCR 100 days after transplant. In the second part of the study, which is ongoing, patients who achieved a partial response or better in part one will undergo a second randomisation to receive maintenance treatment with daratumumab 16 mg/kg every eight weeks for up to two years or will be observed with no further treatment. The primary endpoint in this part of the study is progression-free survival (PFS).

About daratumumab

Daratumumab is a first-in-class6 biologic targeting CD38, a surface protein that is highly expressed across multiple myeloma cells, regardless of disease stage.7 Daratumumab is believed to induce tumour cell death through multiple immune-mediated mechanisms of action, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), as well as through apoptosis, in which a series of molecular steps in a cell lead to its death.4 A subset of myeloid derived suppressor cells (CD38+ MDSCs), CD38+ regulatory T cells (Tregs) and CD38+ B cells (Bregs) were decreased by daratumumab.4 Since launch, it is estimated that 100,000 patients have been treated with daratumumab worldwide.2 Daratumumab is being evaluated in a comprehensive clinical development programme across a range of treatment settings in multiple myeloma, such as in frontline and relapsed settings.5,8,9,10,11,12,13,14 Additional studies are ongoing or planned to assess its potential in other malignant and pre-malignant haematologic diseases in which CD38 is expressed, such as smouldering myeloma.15,16 For more information, please see View Source

For further information on daratumumab, please see the Summary of Product Characteristics at View Source

In August 2012, Janssen Biotech, Inc. and Genmab A/S entered a worldwide agreement, which granted Janssen an exclusive licence to develop, manufacture and commercialise daratumumab.17

About Multiple Myeloma

Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.18 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.19 Almost 60 percent of patients with MM do not survive more than five years after diagnosis.20

Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.21 Refractory MM is when a patient’s disease progresses within 60 days of their last therapy.22,23 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.24 While some patients with MM have no symptoms at all, most patients are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.25 Patients who relapse after treatment with standard therapies, including proteasome inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.26

On December 13, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a Positive Opinion recommending approval for expanding the use of Erleada (apalutamide) to include the treatment of adult men with metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy (ADT) (Press release, Janssen Pharmaceutica, DEC 13, 2019, View Source [SID1234552368]).2 The CHMP’s Positive Opinion will now be reviewed by the European Commission (EC), which has the authority to grant approval for the new use of apalutamide.

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The Positive Opinion is based on data from the Phase 3 TITAN study, which assessed the addition of apalutamide to ADT – the current standard of care in mHSPC – in a broad range of patients with mHSPC, regardless of disease volume, prior treatment with docetaxel or staging at initial diagnosis. The dual primary endpoints of the study were overall survival (OS) and radiographic progression-free survival (rPFS). Apalutamide plus ADT significantly improved OS compared to placebo plus ADT with a 33 percent reduction in the risk of death (HR=0.67; 95% CI, 0.51-0.89; p=0.0053).1 In both study arms, median OS was not reached.1 Apalutamide plus ADT also significantly improved rPFS compared to placebo plus ADT with a 52 percent reduction in risk of radiographic progression or death compared to placebo plus ADT (HR=0.48; 95% CI, 0.39-0.60; p<0.0001).1 The median rPFS was 22.1 months for placebo plus ADT and not reached for apalutamide plus ADT.1 The two-year OS rates, after a median follow up of 22.7 months, were 82 percent for apalutamide plus ADT compared to 74 percent for placebo plus ADT.1 These results were presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and simultaneously published online in The New England Journal of Medicine.1,3

The safety profiles for apalutamide plus ADT, versus placebo plus ADT, were similar with 42 percent versus 41 percent of Grade 3/4 adverse events (AEs) observed respectively.1 The most common Grade ≥3 AEs for apalutamide plus ADT versus placebo plus ADT were hypertension (8.4 percent vs. 9.1 percent) and skin rash (6.3 percent vs. 0.6 percent). Treatment discontinuation due to AEs was 8 percent in the apalutamide arm compared to 5 percent in the placebo arm.1

"Today’s Positive Opinion for apalutamide brings us one step closer to providing a much-needed treatment option for a broad population of patients diagnosed with mHSPC," said Joaquín Casariego, M.D., Janssen Therapeutic Area Lead Oncology for Europe, Middle East & Africa, Janssen-Cilag S.A. "At this stage of disease, it is critical to intervene with another treatment that can prolong survival and delay progression to the fatal stage, without compromising the quality of life of patients. We look forward to the EC approval of apalutamide in this setting so we can bring this innovative medicine to patients as soon as possible."

"We are pleased with the CHMP’s Opinion to recommend approval of apalutamide as a treatment for patients with mHSPC," said Craig Tendler, M.D., Vice President, Clinical Development and Global Medical Affairs, Oncology at Janssen Research & Development, LLC. "Results from the TITAN study demonstrated that the addition of apalutamide to ADT improved outcomes for a broad range of patients with mHSPC, compared to ADT alone, highlighting the significance of today’s Opinion. At Janssen, we continue to focus on addressing crucial areas of unmet need in prostate cancer within our clinical trial programme and are committed to further exploring how to improve outcomes for patients across the entire disease continuum."

In Europe, apalutamide is approved for use in adults with non-metastatic castration-resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease.4 In the United States apalutamide is indicated for the treatment of nmCRPC and metastatic castration-sensitive prostate cancer (mCSPC).5

#ENDS#

About the TITAN Study1,3
TITAN is a Phase 3 randomised, placebo-controlled, double-blind study in men with mHSPC regardless of extent of disease or prior docetaxel treatment history. The study included 1,052 patients in intention-to-treat (ITT) population in 23 countries across 260 sites in North America, Latin America, South America, Europe and Asia Pacific. Patients with mHSPC were randomised 1:1 and received either apalutamide (240 mg) plus continuous androgen deprivation therapy (ADT) (n=525), or placebo plus ADT (n=527). The recruitment period for the study spanned from December 2015 to July 2017. The study included mHSPC patients with both low- and high-volume disease, those who were newly diagnosed, or those who had received prior definitive local therapy or prior treatment with up to six cycles of docetaxel or up to six months of ADT for mHSPC. Participants were treated until disease progression or the occurrence of unacceptable treatment-related toxicity. An independent data-monitoring committee was commissioned by the sponsor to monitor safety and efficacy before unblinding and make study conduct recommendations. Dual primary endpoints of the study were OS and rPFS. Secondary endpoints included time to cytotoxic chemotherapy, time to pain progression, time to chronic opioid use and time to skeletal-related event. Exploratory endpoints included time to PSA progression, time to second progression-free survival and time to symptomatic progression. For additional study information, visit ClinicalTrials.gov.

About apalutamide
Apalutamide is an androgen receptor (AR) inhibitor indicated for use in Europe for the treatment of patients with non-metastatic castration-resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease.4 In the U.S. apalutamide is indicated for the treatment of nmCRPC and metastatic hormone-sensitive prostate cancer (mHSPC).5

About Metastatic Hormone-Sensitive Prostate Cancer
Metastatic hormone-sensitive prostate cancer (mHSPC), also referred to as metastatic castration sensitive prostate cancer (mCSPC), refers to prostate cancer that still responds to androgen deprivation therapy (ADT) and has spread to other parts of the body.6 Patients with mHSPC tend to have a poor prognosis, with a median overall survival (OS) of less than five years, underscoring the need for new treatment options.7,8,9