On October 29, 2018 Apexian Pharmaceuticals reported that researchers will continue to explore the impact of Apexian’s target molecule, APX3330, on cancer cachexia with additional grant funding from the National Institutes of Health (NIH) National Cancer Institute(NCI) (Press release, Apexian Pharmaceuticals, OCT 29, 2018, View Source [SID1234530421]). Cancer cachexia is weight loss with chronic inflammation and defective metabolism, which causes roughly one third of all cancer deaths. It is particularly prevalent in pancreatic ductal adenocarcinoma (PDAC), which has a dismal five-year survival rate.

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Dr. Mark R. Kelley, Apexian Chief Scientific Officer and the Betty and Earl Herr Professor of Pediatric Oncology Research at the Indiana University Simon Cancer Center; Dr. Melissa Fishel, Research Associate Professor, Wells Center for Pediatric Research; and Dr. Teresa Zimmers, Associate Professor of Surgery at the Indiana University School of Medicine, have been working to define mechanisms of cachexia stemming from treatment in PDAC, as well as for identifying mechanism-driven, targeted anti-cachexia therapies.

"The goal of this research is to determine the anti-cachexia potential of Ref-1 inhibition, HIF-1a inhibition, or the combination in mouse models of PDAC," said Dr. Kelley. "APX3330 has proven effective at inhibiting Ref-1, and has been safe and well tolerated when taken by patients with advanced cancers in our Phase 1 clinical study."

Previous studies support Ref-1 as a target in PDAC, on-target effects of APX3330, and the use of APX3330 as a clinical agent in cancer. This study will focus on demonstrating improvement in fat/muscle mass and PDAC cachexia symptoms using APX3330. Positive results from this study would lead to immediate clinical trials using APX3330 to prevent or reverse PDAC cachexia.

"Dr. Kelley’s research on APX3330 as a Ref-1 inhibitor continues to offer promise as a treatment for cancer and cancer-related issues like cachexia and cancer chemotherapy-induced neuropathy," said Steve Carchedi, CEO of Apexian Pharmaceuticals. "As we complete our Phase I trial, we continue to aggressively pursue additional therapeutic uses for APX3330 and build on our pipeline of novel, first in class molecules."

The NIH grant of $227,554 pushes Kelley’s grant budget for research on Ref-1 inhibitors to nearly $700,000 just in 2018.

On October 29, 2018 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the third quarter ended September 30, 2018 and provided an update on recent business progress (Press release, Veracyte, OCT 29, 2018, View Source [SID1234530385]). For the third quarter of 2018, revenue was $23.5 million, an increase of 34 percent, compared with $17.5 million for the third quarter of 2017. Genomic test volume was 8,006, an increase of 23 percent compared with the same period in 2017.

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"We delivered another quarter of strong revenue and genomic volume growth for our Afirma and Percepta classifiers, which are changing practice in the diagnosis of thyroid and lung cancer," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "In addition, we are thrilled that we recently received draft Medicare coverage for our third product, the Envisia Genomic Classifier."

Anderson continued, "As a result of our strong third quarter performance and our outlook for the remainder of this year, we are raising our annual revenue guidance and lowering our cash burn guidance for 2018. And, as we look to 2019 and beyond, we believe we are well-positioned to continue making a difference in the lives of patients, providing solutions to physicians to help better inform clinical decisions, and delivering cost savings to the healthcare system."

Third Quarter 2018 Financial Results

For the three-month period ended September 30, 2018 as compared with the third quarter of 2017:

Revenue was $23.5 million, an increase of 34 percent;
Genomic test volume was 8,006, an increase of 23 percent;
Gross margin was 65 percent, an increase of 6 percent;
Operating expenses, excluding cost of revenue, were $19.5 million, an increase of 17 percent;
Net loss and comprehensive loss was ($4.5) million, an improvement of 37 percent;
Basic and diluted net loss per common share was ($0.12), an improvement of 43 percent;
Cash burn1 was $2.4 million, an improvement of 58 percent; and
Cash and cash equivalents was $77.8 million at September 30, 2018.
For the nine-month period ended September 30, 2018, as compared to the prior year period of 2017:

Revenue was $66.3 million, an increase of 27 percent;
Genomic test volume was 22,556, an increase of 20 percent;
Gross margin was 63 percent, an increase of 2 percent;
Operating expenses, excluding cost of revenue were $61.1 million, an increase of 16 percent
Net loss and comprehensive loss was ($19.9) million, an improvement of 12 percent;
Basic and diluted net loss per common share was ($0.56), an improvement of 16 percent; and
Cash burn1 was $13.7 million, an improvement of 28 percent.
1 Refer to the "Non-GAAP Financial Measures" for an explanation of cash burn and the "Reconciliation of Net Cash Used in Operating Activities to Cash Burn" table included in the financial statements in this press release.

Third Quarter 2018 and Recent Business Highlights

Commercial Growth:

Completed national transition from the Afirma Gene Expression Classifier (GEC) to the Genomic Sequencing Classifier (GSC), retiring all GEC testing and accelerating the adoption of the Afirma Xpression Atlas for surgical and treatment decisions in the third quarter of 2018.
Increased the number of physicians ordering Percepta to nearly 200, across 140 institutions, primarily through a continued focus on sales and marketing initiatives, as of September 30, 2018.
Expanded Envisia Early Access Program sites that have submitted samples for genomic testing to 15 – exceeding the company’s expectations.
Reimbursement Expansion:

Received draft Medicare coverage for the Envisia Genomic Classifier through the MolDX program; upon anticipated finalization in early 2019, Veracyte will be positioned to ramp commercial adoption of Envisia and begin to grow associated revenue next year.
Evidence Development:

Positive performance and clinical utility data for the Afirma GSC and Xpression Atlas were presented at the American Thyroid Association annual meeting. Results show that the Afirma GSC is identifying even more benign thyroid nodules in real-world practice than was demonstrated in the company’s clinical validation study, previously published in JAMA Surgery.
Early results of the ongoing Percepta registry clinical utility study were presented at CHEST 2018 and show that the genomic test changed clinical decision-making and reduced invasive procedures at every evaluation time point up to 12 months post-testing.
Clinical validation data for the Envisia classifier were also presented at CHEST 2018 and confirm the test’s ability to improve diagnosis of idiopathic pulmonary fibrosis – without the need for surgery.
Financing:

In July 2018, Veracyte issued and sold 5,750,000 shares of common stock in a registered public offering, including the underwriters’ exercise in full of their option to purchase an additional 750,000 shares, at a price to the public of $10.25 per share. Net proceeds from the offering were approximately $55.0 million.
Updated 2018 Financial Outlook

Veracyte is increasing its 2018 annual revenue guidance to a range of $90.0 million to $91.0 million from its previous guidance range of $87 million and $89 million. In addition, the company is reducing its annual cash burn guidance to a range of $17.0 million to $18.0 million from its previous guidance range of $18 million to $21 million.

Conference Call and Webcast Details

The company will host a conference call and simultaneous webcast today at 4:30 p.m. EDT to discuss its financial results and provide a general business update. The call and webcast may be accessed as follows:

Webcast:
View Source

Dial-in number (U.S.): (855) 541-0980
International Number: (970) 315-0440
Conference ID: 1658434

The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source.

On October 29, 2018 Neuralstem, Inc. (Nasdaq:CUR), a biopharmaceutical company focused on developing novel treatments for nervous system diseases, reported that it has entered into definitive agreements with institutional investors for the purchase of 3,000,000 shares of its common stock, at a purchase price per share of $0.70, in a registered direct offering (Press release, Neuralstem, OCT 29, 2018, View Source [SID1234530381]). Additionally, Neuralstem has also agreed to issue to the investors unregistered warrants to purchase up to 3,000,000 shares of its common stock. The closing of the offering is expected to take place on or about October 29, 2018, subject to the satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants have an exercise price of $0.75 per share of common stock, will be exercisable commencing six months following the issuance date and will expire five and one-half years from the issuance date.

The gross proceeds to Neuralstem, before deducting placement agent fees and other offering expenses, are expected to be $2.1 million. Neuralstem intends to use the net proceeds from this offering to further its clinical and preclinical programs, and for general working capital.

The shares of common stock (but not the warrants or the shares of common stock underlying the warrants) are being offered by Neuralstem pursuant to a "shelf" registration statement on Form S-3 that was filed and declared effective by the Securities and Exchange Commission ("SEC") on June 23, 2017 and the base prospectus contained therein (File No. 333-218608). The offering of the shares of common stock will be made only by means of a prospectus supplement and accompanying base prospectus that form a part of the registration statement.

A final prospectus supplement and accompanying base prospectus relating to the shares of common stock being offered will be filed with the SEC. Copies of the final prospectus supplement and accompanying base prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, New York 10022, by phone at 646-975-6996 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the shares of common stock underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying shares of common stock may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy any of the securities described herein, 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 October 29, 2018 Genmab A/S (Nasdaq Copenhagen: GEN) reported topline results from the Phase III MAIA study (MMY3008) of daratumumab in combination with lenalidomide and dexamethasone (DRd) versus Rd alone as treatment for newly diagnosed patients who are not candidates for high dose chemotherapy and autologous stem cell transplant (ASCT) (Press release, Genmab, OCT 29, 2018, View Source [SID1234530362]). The study met the primary endpoint of improving progression free survival (PFS) at a pre-planned interim analysis (Hazard Ratio (HR) = 0.55 (95% CI 0.43 – 0.72), p < 0.0001) resulting in a 45% reduction in the risk of progression or death in patients treated with DRd. The median PFS for patients treated with daratumumab in combination with Rd has not been reached, compared to an estimated median PFS of 31.9 months for patients who received Rd alone.

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Overall, the safety profile of daratumumab in combination with Rd is consistent with both the known safety profiles of the Rd regimen and daratumumab.

Based on the results at the pre-planned interim analysis conducted by an Independent Data Monitoring Committee (IDMC), it was recommended releasing the interim analysis results. Further analysis of the safety and efficacy data is ongoing and Janssen Biotech, Inc., which licensed daratumumab from Genmab in 2012, will discuss the potential for a regulatory submission for this indication with health authorities, and plans to submit the data to an upcoming medical conference and for publication in a peer-reviewed journal.

"We are highly encouraged by this data as this is the fifth randomized study showing a profound benefit when adding daratumumab to standard of care treatments in multiple myeloma, and the second showing efficacy for patients with newly diagnosed multiple myeloma who are not eligible for ASCT. As such this data increases our hope that daratumumab may one day help even more patients at the outset of treatment of this disease," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Today’s news does not impact Genmab’s 2018 financial guidance.

About the MAIA (MMY3008) study
The Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone was administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is PFS.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 30,770 new patients are expected to be diagnosed with multiple myeloma and approximately 12,770 people are expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 124,225 people would be diagnosed and 87,084 would die from the disease in 2015.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX (daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of 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 patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use 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; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, myelodysplastic syndromes, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On October 29, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company committed to identifying, developing, and commercializing innovative therapies to address significant unmet needs in aesthetic and medical dermatology and immunology, reported that it will report financial results for third quarter 2018 on Tuesday, November 6, 2018 after U.S. financial markets close (Press release, Aclaris Therapeutics, OCT 29, 2018, View Source [SID1234530355]).

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Management will conduct a conference call at 5:00 PM ET that day to discuss the Company’s financial results and provide a general business update. A live webcast of the event can be accessed on the Events and Presentations page on the Investors section of the Aclaris website at View Source A replay of the webcast will be archived on the Aclaris website following the event.

To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 7694929 prior to the start of the call.