On August 9, 2019 Ardelyx, Inc. (NASDAQ: ARDX), a specialized biopharmaceutical company focused on developing first-in-class medicines to improve treatment choices for people with cardiorenal diseases, reported business highlights and financial results for the second quarter ended June 30, 2019 (Press release, Ardelyx, AUG 9, 2019, View Source [SID1234538570]).

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"We are excited for a catalyst-rich second half of 2019 with planned completion of the final stages of development for tenapanor before we seek approval for its use in treating hyperphosphatemia in end-stage renal disease patients on dialysis," said Mike Raab, president and chief executive officer of Ardelyx. "There continues to be a high unmet need for novel hyperphosphatemia treatments to help ESRD patients achieve phosphorus goals. If positive, the results from our ongoing second Phase 3 clinical trial, PHREEDOM, investigating tenapanor as monotherapy, will allow us to file our NDA next year. If approved, tenapanor will provide patients and health care providers with a novel, first-in-class and much more patient-friendly approach to managing phosphorus levels in dialysis patients. We look forward to announcing results for PHREEDOM in the fourth quarter of this year, and announcing results for AMPLIFY, our ongoing Phase 3 clinical trial evaluating tenapanor’s use in combination with phosphate binders in the third quarter of this year."

Remaining Expected 2019 Milestones

Results from the PHREEDOM clinical trial, the company’s second Phase 3 clinical trial evaluating tenapanor as a monotherapy treatment for hyperphosphatemia in patients with end-stage renal disease (ESRD) who are on dialysis, are currently expected to be announced in the fourth quarter of 2019.
Results from the AMPLIFY clinical trial, the company’s Phase 3 clinical trial evaluating tenapanor’s efficacy in combination with phosphate binders, are currently expected to be announced in the third quarter of 2019.
The company’s New Drug Application for U.S. marketing authorization of tenapanor for patients with IBS-C has a target action date under the Prescription Drug User Fee Act (PDUFA) of September 12, 2019.
Second Quarter 2019 Financial Results

Cash Position: As of June 30, 2019, Ardelyx had total capital resources including cash, cash equivalents and short-term investments of $123.9 million compared to total capital resources including cash, cash equivalents and short-term investments of $168.1 million as of December 31, 2018.
R&D Expenses: Research and development expenses were $19.4 million for the three months ended June 30, 2019, an increase of $3.4 million, or 21 percent, compared to $16.0 million for the three months ended June 30, 2018. The increase included a $7.5 million increase in expense primarily related to the Company’s manufacturing of tenapanor, the continued clinical development of tenapanor for the treatment of hyperphosphatemia in ESRD patients on dialysis and the Company’s hyperkalemia program, RDX013, partially offset by an out-of-period adjustment that reduced clinical trial expenses by $4.1 million related to the tenapanor clinical trials.
G&A Expenses: General and administrative expenses were $5.4 million for the three months ended June 30, 2019, a decrease of $0.7 million, or 12 percent, compared to $6.1 million for the three months ended June 30, 2018. The decrease was primarily related to a decrease in professional services and a reduction in stock-based compensation costs partially offset by an increase in headcount and related personnel costs.
Net Loss: Net loss for the three months ended June 30, 2019 was $25.5 million, or $0.41 per share, compared to a net loss of $22.3 million, or $0.42 per share, for the three months ended June 30, 2018.

On August 9, 2019 CytRx Corporation (OTCQB: CYTR), a biopharmaceutical research and development company specializing in oncology and rare diseases, reported financial results for the second quarter ended June 30, 2019, and provided an overview of recent accomplishments (Press release, CytRx, AUG 9, 2019, View Source [SID1234538569]).

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"The first half of 2019 has been marked by significant achievements from our licensees, who continue to make progress in bringing CytRx’s technology closer to patients who are in need of novel and innovative therapies," said Eric Curtis, CytRx’s President and Chief Operating Officer. "As these partners continue to work diligently, CytRx stands to receive milestone payments and royalties, which may strengthen our balance sheet."

Second Quarter 2019 and Recent Highlights

CytRx Corporation

Orphazyme Prepares Regulatory Submission in United States and Europe for Niemann-Pick Disease Type C. In July 2019, CytRx highlighted that arimoclomol licensee Orphazyme announced that following a positive meeting with the U.S. Food and Drug Administration (FDA), the Company remains on track to submit a New Drug Application (NDA) for arimoclomol in NPC in the first half of 2020. Orphazyme also updated the anticipated timing for submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval for arimoclomol in Niemann-Pick disease Type C (NPC). Based on advice received from the EMA’s Scientific Advice Working Group, Orphazyme expects to submit the arimoclomol MAA in the first half of 2020. CytRx is eligible to receive up to $120 million in future milestones, plus royalties, from its arimoclomol licensing agreement with Orphazyme. Specifically, CytRx is eligible to receive $6 million in the U.S. and $4 million in Europe upon approval of arimoclomol in Orphazyme’s first non-ALS indication, plus royalties.
Licensee Orphazyme Completed Enrollment in Phase 3 Trial of Arimoclomol in Amyotrophic Lateral Sclerosis. In July 2019, CytRx highlighted that arimoclomol licensee Orphazyme A/S (ORPH.CO) has completed enrollment in its Phase 3 trial evaluating arimoclomol for the treatment of amyotrophic lateral sclerosis (ALS) ahead of schedule. Orphazyme anticipates announcing top-line results from the Phase 3 trial in the first half of 2021.
New Patent Issued for Aldoxorubicin Formulation. In June 2019, CytRx highlighted that it has been issued a patent from the U.S. Patent and Trademark Office (USPTO) covering the formulation, storage, delivery and administration of aldoxorubicin at room temperature. The new patent, which issued on June 25, 2019 as U.S. Patent No. 10,328,093 and is titled "Anthracycline Formulations," covers a reconstituted formula of aldoxorubicin that stabilizes the compound, solubilizes it in ethanol and water, and eliminates the need for cold handling, allowing it to be administered to patients in all potential disease indications at room temperature.
CytRx Commences Trading on OTCQB Venture Market. In May 2019, CytRx announced that it commenced trading on the OTCQB Venture Market. The Company continues to trade under ticker symbol "CYTR". Investors can find current financial disclosure and real-time Level 2 quotes for the Company on www.otcmarkets.com/stock/CYTR/quote.
Patent Issued for the Use of Aldoxorubicin in the Treatment of Brain Cancer. In May 2019, CytRx announced that it had been issued a patent the USPTO covering the use of aldoxorubicin intravenously, intra-arterially or intramuscularly for the treatment of brain cancer. The new patent issued on May 7, 2019 as U.S. Patent No. 10,278,981, is titled "Cytotoxic Agents for The Treatment of Cancer." This patent was exclusively licensed by CytRx to NantCell in July 2017.
Orphazyme Completed Enrollment in its Phase 2/3 Clinical Trial in Systemic Inclusion Body Myositis. In April 2019, CytRx highlighted that arimoclomol licensee Orphazyme had completed enrollment in its Phase 2/3 clinical trial in Systemic Inclusion Body Myositis (sIBM). The Phase 2/3 trial is a 150-patient, 20-month, randomized, double-blind, placebo-controlled trial in 11 centers in the United States and one in the United Kingdom. Orphazyme expects to conduct an interim analysis in the first half of 2020 and to complete the study by the end of 2020, with results anticipated in the first half of 2021.
Second Quarter 2019 Financial Results

CytRx reported cash, cash equivalents and short-term investments of $19.4 million as of June 30, 2019.

Net loss for the quarter ended June 30, 2019 was $1.3 million, or $(0.04) per share, compared with a net loss of $ 3.0 million, or $(0.10) per share, for the quarter ended June 30, 2018, a reduction of $1.7 million.

General and administrative (G&A) expenses were $1.5 million for the second quarter of 2019, compared with $ 1.7 million for the second quarter of 2018, including non-cash stock-compensation expense of $0.2 million for the second quarter of 2019 as compared to $ 0.4 million for the second quarter of 2018. G&A expenses decreased by approximately $0.2 million, or 9.4%, primarily due to a decrease in legal fees and a reduction in head count.

Based on our currently projected expenditures for the next 13 months, our monthly cash burn rate is estimated at approximately $400,000 per month.

On August 9, 2019 BioSpecifics Technologies Corp. (NASDAQ: BSTC), a biopharmaceutical company that originated and continues to develop collagenase-based therapies with a first in class collagenase-based product marketed as XIAFLEX in the U.S. and Xiapex in Europe, reported its financial results for the second quarter ended June 30, 2019 and provided a corporate update (Press release, BioSpecifics Technologies, AUG 9, 2019, View Source [SID1234538568]).

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"Throughout the second quarter of 2019, the team at BioSpecifics has been focused on continued internal execution and the clinical progression of our CCH development pipeline and the commercial development of our XIAFLEX franchise. Following positive Phase 1 uterine fibroids data, we are working to develop a clinical path forward for this medically necessary indication, and as we further evaluate and analyze this data, we plan to present it in the second half of this year," said Dr. Ron Law, Ph.D., J.D., Principal Executive Officer of BioSpecifics. "We are encouraged by the continued emphasis from our partner, Endo, on the importance of patient awareness and future growth initiatives for both of our commercial indications for XIAFLEX, Peyronie’s Disease and Dupuytren’s Contracture. We also look forward to adding a potential third marketed indication, with an upcoming BLA filing for the treatment of cellulite, which is expected to occur in the second half of 2019."

Second Quarter 2019 Financial Results

BioSpecifics reported net income of $6.4 million for the second quarter ended June 30, 2019, or $0.88 per basic share and $0.87 per share on a fully diluted basis, compared to net income of $4.8 million, or $0.67 per basic share and $0.66 per share on a fully diluted basis, for the same period in 2018.

Total revenue for the second quarter ended June 30, 2019 was $8.9 million, compared to $7.9 million for the same period in 2018. The increase in total revenues for the quarterly period was primarily due to royalties associated with higher net sales of XIAFLEX in Peyronie’s Disease and Dupuytren’s Contracture, and slightly higher mark-up on cost of goods sold revenue.

Research and development expenses for the second quarter ended June 30, 2019 and 2018 were approximately $0.2 million in each period.

General and administrative expenses for the second quarter ended June 30, 2019 were $1.7 million, compared to $2.0 million for the same period in 2018.

Provision for income taxes for the second quarter ended June 30, 2019 and 2018 was $1.1 million in each period.

As of June 30, 2019, BioSpecifics had cash and cash equivalents and investments of $93.5 million, compared to $82.0 million as of December 31, 2018.

As of June 30, 2019, BioSpecifics had 7,331,917 million shares of common stock outstanding.

Commercial & Pipeline Highlights and Anticipated Upcoming Milestones

BioSpecifics’ Royalty Revenues from the XIAFLEX Commercial Franchise Grew by 13% Year-Over-Year for the Second Quarter. The XIAFLEX commercial franchise royalty revenue growth was primarily attributable to increased net sales of XIAFLEX in Peyronie’s Disease and Dupuytren’s Contracture, driven by Endo’s continued successful commercial execution and promotional investment.
Phase 2 Clinical Trial of Uterine Fibroids Future Development Plans Underway: BioSpecifics and its clinical partners continue to analyze the full Phase 1 data to guide the design of a Phase 2 study of collagenase clostridium histolyticum (CCH) for the treatment of uterine fibroids. BioSpecifics will present new data from the Phase 1 clinical trial at an upcoming medical meeting in the second half of 2019. Data from the Phase 1 clinical trial of CCH for the treatment of uterine fibroids were presented at the 66th Annual Meeting of the Society of Reproductive Investigation (SRI) on March 2019 in Paris, France. The reported data showed safety and statistically significant reductions in collagen content compared to control fibroids with a median reduction of 39 percent (p<0.05), as well as a 21 percent average reduction in density of collagen bundles.
New Data Presentations of CCH for the Treatment of Cellulite at Top Aesthetic Medical Meetings:
Endo reported data from the RELEASE-1 and RELEASE-2 Phase 3 studies during the "Premier Global Hot Topics Session" at the Annual Meeting of the American Society for Aesthetic Plastic Surgery on May 17, 2019 in New York, NY. The reported data showed that a greater percentage of women treated with CCH (CCH vs. placebo: RELEASE 1, n=210 vs n=213; RELEASE-2, n=214 vs n=206) met the primary endpoint versus placebo in both the RELEASE-1 (P=0.006) and RELEASE-2 (P=0.002) studies. In addition, statistically significant improvements with CCH vs placebo were observed in eight of eight (RELEASE-1) and seven of eight (RELEASE-2) secondary endpoints. Other patient-centric endpoints were also evaluated, including improvement in the Subject Global Aesthetic Improvement Scale (S-GAIS), a five-point scale rating global aesthetic improvement in appearance, compared to pretreatment, as judged by the subject. Most adverse events observed in CCH-treated patients were mild/moderate and injection-site related.
Endo also reported Phase 2 and Phase 3 data at the Vegas Cosmetic Surgery Meeting in June 2019.
Expansion of Patient Awareness and Future Growth Initiatives for Marketed XIAFLEX Indications: In June 2019, Endo announced that nationally recognized urologist and author, Aaron Spitz, M.D., partnered with Endo and the "Ask About the Curve" campaign to encourage men to get in touch with their anatomy and learn about the signs and symptoms of Peyronie’s Disease. Endo also has continued future growth initiates for Dupuytren’s Contracture with "Facts on Hand" in partnership with Tim Herron, four-time PGA Tour winner and Damon Adamany, M.D. of the CORE Institute.
BLA filing for CCH for Treatment of Cellulite Expected in 2H19: Endo reported that it expects to file its Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for CCH for the treatment of cellulite in the second half of 2019, with an expected commercial launch in the second half of 2020, subject to FDA approval.

On August 9, 2019 Titan Pharmaceuticals, Inc. (NASDAQ:TTNP) reported that it will announce its second quarter 2019 financial results after market close on Wednesday, August 14, 2019 (Press release, Titan Pharmaceuticals, AUG 9, 2019, View Source;conference-call-to-follow-300899215.html [SID1234538567]). Following the release, Titan management will host a conference call at 4:30 p.m. EDT / 1:30 p.m. PDT to review the financial results and discuss business developments in the period.

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The conference call will be hosted by Sunil Bhonsle, President and CEO; Dane Hallberg, Executive Vice President and Chief Commercial Officer; Brian Crowley, Vice President of Finance; and Marc Rubin, M.D., Executive Chairman.

The live conference call may be accessed by dialing 1-888-317-6003 (U.S.) or 1-412-317-6061 (international) and providing passcode 9110840. The call will also be broadcast live and archived on Titan’s website at www.titanpharm.com/news/events.

On August 8, 2019, Stemline Therapeutics, Inc. ("Stemline" or the "Company") reported that it has entered into an underwriting agreement (the "Underwriting Agreement") with J.P. Morgan Securities LLC, as representative of the several underwriters named therein (the "Underwriters") (Filing, 8-K, Stemline Therapeutics, AUG 9, 2019, View Source [SID1234538565]). Pursuant to the Underwriting Agreement, the Company agreed to sell to the Underwriters, in a firm commitment underwritten public offering, 5,000,000 shares (the "Firm Shares") of the Company’s common stock, $.0001 par value per share ("Common Stock"), at a price to the public of $15.25 per share, less underwriting discounts and commissions. In addition, pursuant to the Underwriting Agreement, the Company has granted the Underwriters an option, exercisable for 30 days, to purchase up to an additional 750,000 shares of Common Stock (the "Additional Shares," together with the Firm Shares, the "Shares"). The transactions contemplated by the Underwriting Agreement are expected to close on August 13, 2019, subject to the satisfaction of customary closing conditions. A copy of the Underwriting Agreement is attached hereto as Exhibit 1.1 and is incorporated by reference herein.

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J.P. Morgan Securities LLC and Cowen and Company, LLC are acting as joint book-running managers for the offering.

The gross proceeds to the Company are expected to be approximately $76,250,000, assuming no exercise of the option to purchase Additional Shares and before deducting underwriting discounts and commissions and estimated expenses payable by the Company associated with the offering.

The Underwriting Agreement contains customary representations, warranties and agreements by the Company, customary conditions to closing, indemnification obligations of the Company and the Underwriters, including for liabilities under the Securities Act of 1933, as amended, other obligations of the parties and termination provisions.

Alston & Bird LLP, counsel to the Company, delivered an opinion as to the validity of the Shares, a copy of which is attached hereto as Exhibit 5.1 and is incorporated by reference herein.