FDA Grants Orphan Drug Designation to Puma Biotechnology’s NERLYNX® for the Treatment of Breast Cancer Patients with Brain Metastases

On September 3, 2019 Puma Biotechnology, Inc. (Nasdaq: PBYI) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to NERLYNX (neratinib) for the treatment of breast cancer patients with brain metastases (Press release, Puma Biotechnology, SEP 3, 2019, View Source [SID1234539244]).

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"Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to developing treatments for patients with HER2-positive breast cancer," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "Despite expanded treatment options for HER2-positive breast cancer, brain metastases in these patients represent a significant clinical challenge, as well as sources of morbidity and mortality for most of these patients. The blood-tumor penetrability of NERLYNX represents a potential treatment option for these underserved patients."

The FDA Orphan Drug Designation program grants orphan designation to investigational drugs designed to treat, prevent, or diagnose rare medical diseases or conditions that affect fewer than 200,000 individuals in the United States. Orphan designation qualifies sponsors for several key benefits and incentives, including opportunities for grant funding towards clinical trial costs, tax credits, user fee waivers, and the potential for a seven-year period of marketing exclusivity upon FDA approval of the investigational drug for the indication for which it has orphan designation.

About HER2-Positive Breast Cancer

Approximately 20 to 25 percent of breast cancer tumors over-express the HER2 protein. HER2-positive breast cancer is often more aggressive than other types of breast cancer, increasing the risk of disease progression and death. Although research has shown that trastuzumab can reduce the risk of early stage HER2-positive breast cancer returning after surgery, up to 25% of patients treated with trastuzumab experience recurrence.

Exelixis to Present at the Morgan Stanley 17th Annual Global Healthcare Conference on September 10, 2019

On September 3, 2019 Exelixis, Inc. (Nasdaq: EXEL) reported that Michael M. Morrissey, Ph.D., the company’s President and Chief Executive Officer, will provide a corporate overview at the Morgan Stanley 17th Annual Global Healthcare Conference, which is being held next week in New York (Press release, Exelixis, SEP 3, 2019, View Source [SID1234539243]). Exelixis’ presentation has been scheduled for Tuesday, September 10, 2019 at 4:40 PM EDT / 1:40 PM PDT.

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To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the presentation to ensure adequate time for any software download that may be required to listen to the webcast. A replay will also be available at the same location for 14 days.

Castle Biosciences Reports Second Quarter 2019 Results

On September 3, 2019 Castle Biosciences, Inc. (Nasdaq: CSTL) reported its financial results for the second quarter and six months ended June 30, 2019 (Press release, Castle Biosciences, SEP 3, 2019, View Source [SID1234539242]).

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"We are pleased with our strong second quarter performance, including our 27% year-over-year growth in DecisionDx-Melanoma test report volume, which we believe was largely driven by our successful first quarter 2019 commercial expansion," said Derek Maetzold, President and CEO of Castle Biosciences. "We are also encouraged by the success of our recently completed initial public offering and the strong level of interest from our investors. We believe the proceeds from our initial public offering, combined with our existing working capital, position us to execute upon our expansion plans and fund research and development activities."

Second Quarter Ended June 30, 2019 Financial Highlights

Revenues of $10.7 million, an increase of $6.8 million from Q2 2018.
Delivered 4,067 DecisionDx-Melanoma and DecisionDx-UM proprietary test reports, an increase of 24% over Q2 2018, with DecisionDx-Melanoma having an increase of 27%.
Gross margin of $8.7 million, or 81%, compared to $2.7 million, or 67% for Q2 2018.
Six Months Ended June 30, 2019 Financial Highlights

Revenues of $19.5 million, an increase of $11.8 million from the first half of 2018.
Delivered 7,659 DecisionDx-Melanoma and DecisionDx-UM proprietary test reports, an increase of 21% over the same period in 2018, with DecisionDx-Melanoma having an increase of 23%.
Gross margin of $15.9 million, or 82%, compared to $5.1 million, or 66% for the first half of 2018.
Supplemental Revenue Information

Affecting the year-over-year comparability of our revenues were (a) the issuance of the Medicare Local Coverage Determination (LCD) for our DecisionDx-Melanoma test, effective December 3, 2018 and (b) confirmation of the Medicare Contractor rate for DecisionDx-Melanoma. As a result of timing of these two elements, all 2018 Medicare claims covered under the LCD were recognized as revenue in the fourth quarter of 2018. Medicare revenues for DecisionDx-Melanoma associated with test reports delivered in the three and six months ended June 30, 2018, but not recorded until the fourth quarter of 2018, were $2.2 million and $3.0 million, respectively. Also, included in revenues for the quarter ended June 30, 2019 and 2018 were positive (negative) revenue adjustments related to tests delivered in prior periods of $3.3 million and $(0.8) million, respectively. For the six months ended June 30, 2019 and 2018, these amounts totaled $2.8 million and $0.9 million, respectively.

Second Quarter Business and Clinical Evidence Updates

An independent, prospective study from investigators at the Saint Louis University Hospital was published during the second quarter in the journal Cancer Medicine. The study demonstrated that DecisionDx-Melanoma accurately identified risk of melanoma recurrence independent of other prognostic factors such as Breslow thickness and sentinel lymph node biopsy. The results are consistent with previously published prospective and retrospective studies demonstrating the high performance of DecisionDx-Melanoma to predict outcomes, supporting its clinical value to inform patient management decisions.
American Skin Association (ASA) honored Castle Biosciences with the 2019 Technology Innovation in Melanoma Award for advancing innovative technology in the field of dermatology.
Poster at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting presented updated results from a prospective, multicenter study demonstrating the accuracy and performance of DecisionDx-Melanoma with median follow-up of over 3 years. Three-year overall survival for patients with T1-T2 melanoma and a DecisionDx-Melanoma Class 1A test result was greater than 99%, providing support for the use of a DecisionDx-Melanoma Class 1A test result to identify patients at low risk for a positive sentinel lymph node biopsy result, allowing patients to avoid the invasive surgical procedure.
Poster at the 2019 Fall Clinical Dermatology Conference for PAs & NPs highlighted accuracy of DecisionDx-Melanoma risk prediction in newly expanded cumulative patient cohort of 901 patients.
Presentation at the 2019 American College of Mohs Surgery (ACMS) Annual Meeting highlighted progress in the development of Castle Biosciences’ gene expression profile (GEP) test candidate for use in cutaneous squamous cell carcinoma (cSCC).
Financing, Liquidity and IPO

As of June 30, 2019, our cash balance was $17.5 million and the outstanding principal balance on our bank term loan was $26.7 million. Subsequently, on July 12, 2019, we issued a $10.0 million convertible promissory note and on July 29, 2019, we completed our IPO, which resulted in net proceeds of approximately $66.0 million, after deducting underwriting discounts and commissions and other offering expenses.

Recent Developments

On August 22, 2019, Palmetto GBA, a Medicare Administrative Contractor, posted a draft LCD that, if finalized, would result in expanded Medicare coverage for our DecisionDx-Melanoma test. We expect that Palmetto GBA will finalize and implement the draft LCD after a public comment period that opens on October 7, 2019 and closes on November 21, 2019.

The nineteenth peer-reviewed publication supporting DecisionDx-Melanoma use was published in the journal Skin in early July. This study was an evidence-based analysis of the clinical performance and utility of DecisionDx-Melanoma using four different datasets ranging in size from 403 to 8,944 patients. The study focused on identifying a tumor thickness that would be appropriate for use of the test to guide follow-up decisions for cutaneous melanoma patients. Results demonstrate that a tumor thickness of 0.3 mm and thicker is an appropriate population in which to use DecisionDx-Melanoma to guide follow-up decisions. Note that previously published validation, performance and utility studies support the two primary clinical uses of the DecisionDx-Melanoma test following the diagnosis of melanoma. The first use is to inform decisions on sentinel lymph node biopsy in most eligible patients with tumor thickness less than or equal to 2.0 mm with no minimum tumor thickness, and the second is to guide follow-up decisions.

Conference Call Details

Castle Biosciences will hold a conference call on September 3, 2019 at 4:30 p.m. Eastern time to discuss its second quarter 2019 results and provide a general business update.

The conference call will be webcast live from the company’s website and will be available via the following link: View Source Please access the webcast at least 10 minutes before the conference call start time.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 2368125.

A replay of the webcast will be available for two weeks following the conclusion of the live broadcast and will be accessible on the Investors section of the company’s website at www.CastleBiosciences.com.

Verastem Oncology to Present at the 21st Annual H.C. Wainwright Global Investment Conference

On September 3, 2019 Verastem, Inc. (Nasdaq: VSTM) (Verastem Oncology or the Company), focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported that the Company will present at the 21st Annual H.C. Wainwright Global Investment Conference on Tuesday, September 10, 2019 at 10:25 a.m. EDT in New York, NY (Press release, Verastem, SEP 3, 2019, View Source [SID1234539241]).

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A live webcast of the presentation will be available on the investors section of the Company’s website at www.verastem.com. An archived presentation will be available for 90 days.

Deciphera Pharmaceuticals Announces Full Exercise of Underwriters’ Option to Purchase Additional Shares in Public Offering of Common Stock

On September 3, 2019 Deciphera Pharmaceuticals, Inc. (Nasdaq:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that the underwriters of its previously announced public offering of common stock have exercised their option to purchase an additional 1,621,621 shares of the Company’s common stock at a price of $37.00 per share before underwriting discounts (Press release, Deciphera Pharmaceuticals, SEP 3, 2019, View Source [SID1234539240]). The option exercise closed on September 3, 2019. On August 19, 2019, Deciphera announced the closing of its registered underwritten public offering in which 10,810,810 shares of the Company’s common stock at a price to the public of $37.00 per share were issued and sold. The gross proceeds to Deciphera from the offering inclusive of the exercise of the underwriters’ option to purchase additional shares, before deducting the underwriting discounts and commissions and other estimated offering expenses, are expected to be approximately $460.0 million.

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J.P. Morgan, Piper Jaffray and Jefferies acted as joint book-running managers for the offering. Guggenheim Securities acted as lead manager for the offering. SunTrust Robinson Humphrey acted as co-manager for the offering.

Deciphera intends to use the net proceeds of the offering to fund: clinical trials for ripretinib, including the expansion stage of its current Phase 1 clinical trial, its ongoing pivotal Phase 3 clinical trials, and additional clinical trials, as well as clinical research outsourcing and manufacturing of clinical trial material, and pre-commercialization manufacturing process development and validation; clinical trials for DCC-3014, including the expansion stage of its current Phase 1 clinical trial, as well as clinical research outsourcing and manufacturing of clinical trial material; clinical trials for rebastinib, including its current Phase 1b/2 clinical trials, as well as clinical research outsourcing and manufacturing of clinical trial material; Investigational New Drug-enabling studies and the potential development of DCC-3116; new and ongoing research activities for future drug candidates using its proprietary kinase switch control inhibitor platform; continued growth of its commercial and medical affairs capabilities to support its transition from a development-stage company toward a commercial-stage company; and working capital purposes, including general operating expenses.

The offering was made only by means of a prospectus supplement and accompanying prospectus forming part of a shelf registration statement on Form S-3 previously filed with the Securities and Exchange Commission (SEC) and declared effective by the SEC on October 12, 2018. The final prospectus supplement and the accompanying prospectus was filed with the SEC and is available on the SEC’s website located at View Source Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from J.P. Morgan Securities LLC c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204, or by email at [email protected]; Piper Jaffray & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota, 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected]; and Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, by telephone at (877) 821-7388 or by email at [email protected].

This press release does not constitute an offer to sell or the 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.