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Janssen Announces U.S. FDA Breakthrough Therapy Designation Granted for Niraparib for the Treatment of Metastatic Castration-Resistant Prostate Cancer

On October 3, 2019 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for niraparib, an orally-administered poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with BRCA1/2 gene-mutated metastatic castration-resistant prostate cancer (mCRPC) who have received prior taxane chemotherapy and androgen receptor (AR)-targeted therapy (Press release, Johnson & Johnson, OCT 3, 2019, View Source [SID1234540049]). A Breakthrough Therapy Designation is granted to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition.1 The criteria for Breakthrough Therapy Designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.1

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BRCA1/2 mutations are the most common DNA-repair gene defects (DRD) in patients with mCRPC.2 Patients with a DRD in BRCA1/2 are at an elevated risk for both prostate cancer occurrence and more aggressive disease.2

"Niraparib is a PARP inhibitor that we believe may help address an important unmet need for patients with metastatic castration-resistant prostate cancer who have mutations in DNA-repair genes," said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. "We are pleased with the FDA’s Breakthrough Therapy Designation as we continue the clinical development of niraparib, and we look forward to working with the agency in our continued focus and commitment to bring new advancements to patients diagnosed with prostate cancer."

The Breakthrough Therapy Designation is based on data from the GALAHAD study, a Phase 2, multicenter, open-label clinical trial evaluating the efficacy and safety of niraparib in the treatment of adult patients with mCRPC and DRD who had received treatment with next-generation androgen-receptor targeting therapies and docetaxel.3 Data from the Phase 2 GALAHAD study were recently presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2019 Annual Congress as a late-breaking abstract.

About Metastatic Castration-Resistant Prostate Cancer
Metastatic castration-resistant prostate cancer is a form of prostate cancer that has spread to other parts of the body and keeps growing even when the amount of testosterone in the body is reduced to very low levels.4,5 The most common metastatic sites are bones, followed by distant lymph nodes, liver and thorax.6 Prostate cancer is the second most common type of cancer in men worldwide.7 More than one million people around the world are diagnosed with prostate cancer each year.7,8

Other Ongoing Studies with Niraparib
Ongoing studies for niraparib include the Phase 3 MAGNITUDE study evaluating niraparib in combination with ZYTIGA (abiraterone acetate) and prednisone in adults with metastatic prostate cancer. The MAGNITUDE study is evaluating niraparib plus ZYTIGA and prednisone in a broader population than GALAHAD in patients with frontline mCRPC disease. In addition, QUEST, a Phase 1b/2 study of niraparib combination therapies for the treatment of mCRPC, is ongoing.

About Niraparib
Niraparib is an orally-administered selective PARP inhibitor that is currently being studied by Janssen for the treatment of patients with prostate cancer. In April 2016, Janssen entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc., for exclusive rights to niraparib in prostate cancer. In the U.S., niraparib is indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.9 Niraparib is currently marketed as ZEJULA by TESARO, an oncology-focused business within GSK, devoted to providing transformative therapies to people facing cancer. Please refer to the full Prescribing Information available at View Source

AMN Healthcare to Host Third Quarter 2019 Earnings Conference Call on Thursday, October 31, 2019

On October 3, 2019 AMN Healthcare Services, Inc. (NYSE: AMN), healthcare’s leader and innovator in workforce solutions and staffing services, reported that it has scheduled a conference call to discuss its third quarter 2019 financial results on Thursday, October 31, 2019 at 5:00 p.m. Eastern Time (Press release, AMN Healthcare Services, OCT 3, 2019, View Source [SID1234540048]). The same day, the Company also expects to issue an earnings news release after market close at approximately 4:15 p.m. Eastern Time.

A live webcast of the call can be accessed through AMN Healthcare’s website at View Source Please log in at least 10 minutes prior to the conference call in order to download the applicable audio software. Interested parties may participate live via telephone by dialing (800) 288-8960 in the U.S. or (612) 234-9960 for international callers. Following the conclusion of the call, a replay of the webcast will be available at the Company’s website. Alternatively, a telephonic replay of the call will be available beginning at 7:30 p.m. Eastern Time on October 31, 2019, and can be accessed until 11:59 p.m. Eastern Time on November 14, 2019 by calling (800) 475-6701 in the U.S. or (320) 365-3844 internationally, with access code 472944.

Rafael Holdings Reports Fourth Quarter and Full Fiscal Year 2019 Results

On October 3, 2019 Rafael Holdings, Inc., (NYSE American: RFL), reported revenue of $4.9 million and a loss per share of $0.35 for the fiscal year ended July 31, 2019 (Press release, Rafael Pharmaceuticals, OCT 3, 2019, View Source [SID1234540047]). Fourth quarter revenue was $1.4 million and the loss per share was $0.16.

Recent Operational Highlights

Rafael Pharmaceuticals, a clinical-stage pharmaceutical company in which the Company holds preferred equity and a warrant to increase ownership to 56% of the fully diluted equity interests, reached an out-licensing agreement with Ono Pharmaceutical Co., Ltd. of Japan. Ono gained exclusive rights to develop and commercialize Rafael Pharma’s lead drug candidate, CPI-613 (devimistat) and related compounds for all indications in certain Asia-Pacific region countries. Rafael Pharma received an upfront payment of $12.9 million, with the right to an additional $150.3 million contingent on attainment of certain developmental and commercial milestones. Rafael Pharma will also receive low-double digit royalties based on net sales.
Rafael Pharma continued to expand its pivotal Phase 3 trial of CPI-613 (devimistat) for patients with relapsed or refractory acute myeloid leukemia (AML) adding clinical trial sites in France, Austria, South Korea and Spain.
Rafael Pharma continued to expand its pivotal Phase 3 trial of CPI-613 (devimistat) in combination with modified FOLFIRINOX as a first-line treatment for patients with metastatic pancreatic cancer adding clinical trial sites in France, Korea and Israel.
Future Oncology, a peer-reviewed medical journal, published two manuscripts about the details of ongoing Phase 2 and Phase 3 studies at Rafael Pharma.
LipoMedix Pharmaceuticals, a clinical-stage company in which we hold a majority interest, was awarded a Horizon 2020 Phase 1 grant for the project: Promitil – a new ‘smart’ nanomedicine for cancer chemo-radiotherapy. Horizon 2020 is a research and innovation program of the European Union.
Remarks by Howard Jonas, Chairman and CEO of Rafael Holdings
"During the fourth quarter, our key pharma holding, Rafael Pharma, continued to advance its clinical development programs, including pivotal, multi-jurisdictional, Phase 3 trials of CPI-613 (devimistat). Rafael Pharma also reached an out-licensing agreement with Ono Pharmaceuticals reflecting the promise of Rafael Pharma’s lead drug candidate.

"In addition, we have established a wholly-owned venture to develop a pipeline of therapeutic compounds including compounds to regulate cancer metabolism. The venture is pursuing collaborative research agreements with scientists from top academic institutions.

"We also continue our efforts to realize the value of our real estate holdings including our 20-story commercial property and associated garage in Newark, New Jersey."

Heron Therapeutics Announces Pricing of Public Offering of Common Stock

On October 3, 2019 Heron Therapeutics, Inc. ("Heron") (NASDAQ: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported the pricing of an underwritten public offering of 8,571,429 shares of its common stock at a price of $17.50 per share (Press release, Heron Therapeutics, OCT 3, 2019, View Source [SID1234540046]). In addition, Heron has granted the underwriters of the offering a 30-day option to purchase up to an additional 1,285,714 shares of its common stock on the same terms and conditions. The offering is expected to close on or about October 8, 2019, subject to customary closing conditions.

The gross proceeds from the offering are expected to be approximately $150 million before deducting customary underwriting discounts and offering expenses. Heron intends to use the proceeds from the proposed sale of its shares of common stock for the commercial launch of HTX-011, if approved by the U.S. Food and Drug Administration, the continued commercialization and marketing of SUSTOL and CINVANTI, Heron’s ongoing and future clinical trials, including further clinical studies for HTX-011, preclinical development work, other product development activities and general corporate purposes.

Jefferies, Cowen and Evercore ISI are acting as joint book-running managers for the offering. Cantor is acting as lead manager for the offering, and JMP Securities, Needham & Company and Northland Capital Markets are acting as co-managers for the offering.

The offering is being made pursuant to a registration statement that was filed with the U.S. Securities and Exchange Commission (the "SEC") and became automatically effective on July 6, 2017. A final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC. The securities described above have not been qualified under any state blue sky laws. This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. Copies of the final prospectus supplement (when available) and accompanying prospectus relating to these securities may also be obtained by sending a request to Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, New York 10022, by telephone at (877) 821-7388, or by email at [email protected], Cowen and Company, LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by email at [email protected] or by telephone at (833) 297-2926; or Evercore Group L.L.C. at Attention Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, by telephone at (888) 474-0200, or by email at [email protected].

Verastem Oncology Receives Orphan Drug Designation from FDA for COPIKTRA for the Treatment of T-Cell Lymphoma

On October 3, 2019 Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported duvelisib (COPIKTRATM) has received orphan drug designation from the U.S. Food and Drug Administration for use in the treatment of T-Cell lymphoma (Press release, Verastem, OCT 3, 2019, View Source [SID1234540044]). The designation was created to encourage the development of drugs that may provide significant benefit to patients suffering from rare diseases.

"Receiving orphan drug designation for T-Cell Lymphoma, in addition to the previously-granted Fast Track status, for Peripheral T-Cell lymphoma, marks another important regulatory milestone to bring COPIKTRA to patients who are faced with this aggressive type of disease with limited therapeutic options," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We look forward to sharing the results of our Phase 2 PRIMO study and efficiently advancing our development program in this indication."

COPIKTRA is approved in the United States for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least 2 prior therapies and accelerated approval in follicular lymphoma (FL) after at least 2 prior systemic therapies. COPIKTRA is not currently approved for the treatment of T-cell lymphoma. The Company’s ongoing Phase 2 PRIMO study will provide guidance on a duvelisib monotherapy dosing regimen in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) and further characterize its efficacy and tolerability in this population.

In the U.S., under the Orphan Drug Act, the FDA’s Office of Orphan Products Development (OOPD) grants orphan drug status to a drug or biologic intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders, which is generally a disease that affects fewer than 200,000 individuals in the U.S. and that are expected to provide a significant therapeutic advantage over existing treatments. Orphan designation qualifies a company for benefits that apply across all stages of drug development, including an accelerated approval process, seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.

About Peripheral T-Cell Lymphoma

Peripheral T-cell lymphoma (PTCL) is a rare, aggressive type of non-Hodgkin lymphoma (NHL) that develops in mature white blood cells called "T cells" and "natural killer (NK) cells"1 which circulate with the lymphatic system.2 PTCL accounts for between 10-15% of all non-Hodgkin lymphomas (NHLs) and generally affects people aged 60 years and older.1 Although there are many different subtypes of peripheral T-cell lymphoma, they often present in a similar way, with widespread, enlarged, painless lymph nodes in the neck, armpit or groin.2 There is currently no established standard of care for patients with relapsed or refractory disease.1

SELECT IMPORTANT SAFETY INFORMATION

This does not include all information needed to use COPIKTRA (duvelisib) safety and effectively. See full Prescribing Information.

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS

See full Prescribing Information for complete boxed warning

Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% (<1% fatal) of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
INDICATIONS AND USAGE

COPIKTRA is a kinase inhibitor indicated for the treatment of adult patients with:

Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies.
Relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Accelerated approval based on overall response rate and continued approval may be contingent upon confirmatory trials
WARNINGS AND PRECAUTIONS

Hepatotoxicity: Monitor hepatic function.
Neutropenia: Monitor blood counts.
Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.
ADVERSE REACTIONS

The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at 1-877-7RXVSTM (1-877-779-8786).

DRUG INTERACTIONS

CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors.
CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates.
USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.3,4,5 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.6 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.