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Moleculin Comments on Recent FDA Drug Approvals for Acute Myeloid Leukemia

On August 9, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a preclinical pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the MD Anderson Cancer Center, reported on several recent FDA approvals for new drugs for the treatment of acute myeloid leukemia (AML) (Press release, Moleculin, AUG 9, 2017, View Source [SID1234520119]).

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Walter Klemp, CEO of Moleculin commented: "the recent approvals of three new drugs (Rydapt, Vyxeos and Idhifa) for the treatment of AML are exciting, since they provide additional options for treatments in defined subpopulations, and because they help underscore the magnitude of the potential opportunity for Annamycin, which we will be studying for relapsed or refractory AML. With regard to AML, Rydapt is approved only for patients with a specific gene mutation, and for use in combination with the standard of care chemotherapy. Vyxeos is approved as an option to the standard of care, but only for specific AML patients, namely those with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Jazz Pharmaceuticals purchased this drug in their $1.5 billion acquisition of Celator Pharmaceuticals.

Mr. Klemp continued: "although FDA approval of both of those drugs was based on overall survival comparisons with a standard of care, Idhifa was approved based on an accelerated clinical trial design that showed a 19% response rate in patients with relapsed or refractory AML and IDH2 mutation. What’s interesting is that Idhifa was approved with a single Phase 1/2 clinical trial based on response rate, not overall survival, and a relatively low response rate at that. Also, the patient population for which it is approved represents only 13% of all AML patients. We look forward to working with FDA on a similar approach for Annamycin — reliance on response rate in an accelerated path — but for a larger population of AML patients."

"While these new drugs make valuable incremental improvements in AML therapy," concluded Mr. Klemp, "most AML patients will still fail to respond to (or relapse from) initial therapy; therefore, our initial clinical development plan will attempt to address the significant unmet need of patients who relapse from, or are refractory to, initial therapy. We also believe that, if Annamycin can demonstrate superior efficacy and safety to the current standard of care, the drug may be able to fill major areas for first-line AML treatment. In the meantime, these transactions serve to remind us of the opportunity for our company if Annamycin shows significant activity in our planned clinical trials."

Heron Therapeutics Reports Financial Results for the Three and Six Months Ended June 30, 2017 and Recent Corporate Progress

On August 9, 2017 Heron Therapeutics, Inc. (Nasdaq: HRTX) (the Company or Heron), a commercial-stage biotechnology company focused on developing novel, best-in-class treatments to address some of the most important unmet patient needs, reported financial results for the three and six months ended June 30, 2017 and highlighted recent corporate progress (Press release, Heron Therapeutics, AUG 9, 2017, View Source [SID1234520118]).

Recent Corporate Progress
Pain Franchise
Initiation of Phase 3 Program for HTX-011 in Postoperative Pain Following Successful End-of-Phase 2 Meeting with FDA. Heron reached a general agreement with the U.S. Food and Drug Administration (FDA) on the design and key elements for HTX-011’s Phase 3 program that will be required to support a New Drug Application (NDA). The program includes two pivotal Phase 3 efficacy studies in bunionectomy and hernia repair and an approximately 200-patient Phase 3 safety and pharmacokinetics study to meet the target patient numbers established by the FDA and to provide further evidence of the broad utility of HTX-011 across multiple surgical models. Importantly, the FDA noted that, beyond the agreed-upon Phase 3 studies, no additional clinical work is needed to meet the "Combination Rule" for fixed-dose combination products. The Phase 3 program is designed to achieve a broad indication for the reduction in postoperative pain for 72 hours and reduce the need for opioid analgesic medications following surgery. Heron recently initiated patient enrollment in its Phase 3 program and anticipates completing the Phase 3 program in the first half of 2018. Heron expects to file an NDA in 2018.

Hired Key Talent. Anita Gupta, D.O., Pharm.D. joined Heron as Senior Vice President, Medical Strategy and Government Affairs and will be a key team member providing medical and regulatory input, and working with governmental agencies to support the use of opioid alternatives for postoperative pain. Dr. Gupta has led influential research, advocacy, community and healthcare policy efforts in pain medicine, anesthesiology and opioid prevention. Dr. Gupta is a board-certified anesthesiologist and internationally recognized pain specialist. She recently served as a member of the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and is a fellow at Princeton University at the Woodrow Wilson School.

CINV Franchise
SUSTOL Sales Increase. Net product sales of SUSTOL (granisetron) extended-release injection for the three months ended June 30, 2017 were $8.5 million, compared to $3.6 million for the three months ended March 31, 2017.

SUSTOL Guidance Increased. Based on results for the first half of 2017, Heron has increased its full year 2017 net product sales of SUSTOL guidance to a range of $25 to $30 million.

CINVANTI (HTX-019) FDA Action Date in Q4 2017. The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of November 12, 2017 for a decision on the Company’s NDA for CINVANTI.

"The first half of 2017 has been very exciting for Heron with the submission of the CINVANTI NDA, a successful End-of-Phase 2 meeting for HTX-011, and the excellent growth in net product sales of SUSTOL," said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron. "We have made important progress in the launch of SUSTOL and plan to build upon our success with the anticipated approval of CINVANTI by year-end 2017, which would add a second, complementary commercial product."

Financial Results
Net product sales of SUSTOL for the three months ended June 30, 2017 were $8.5 million, which represents 134% sequential quarter-over-quarter growth, compared to the $3.6 million of net product sales of SUSTOL for the three months ended March 31, 2017. Net product sales of SUSTOL for the six months ended June 30, 2017 were $12.1 million.

Heron’s net loss for the three and six months ended June 30, 2017 was $42.8 million and $93.1 million, or $0.80 per share and $1.79 per share, respectively, compared to a net loss of $43.2 million and $76.7 million, or $1.17 per share and $2.09 per share, respectively, for the same periods in 2016. Net loss for the three and six months ended June 30, 2017, included non-cash, stock-based compensation expense of $8.2 million and $16.2 million, respectively, compared to $5.8 million and $11.2 million, respectively, for the same periods in 2016.

Heron’s net cash used for operating activities for the three months ended June 30, 2017 was $32.0 million, compared to $50.6 million for the three months ended March 31, 2017, a decrease of $18.6 million quarter-over-quarter. Net cash used in operating activities for the six months ended June 30, 2017 was $82.6 million, compared to $59.5 million for the same period in 2016. During the three months ended June 30, 2017, Heron repaid $25.0 million in principal on its outstanding secured promissory note payable.

Heron believes that its cash, cash equivalents and short-term investments of $109.3 million and accounts receivable of $18.6 million at June 30, 2017, along with collections from SUSTOL sales after June 30, 2017, provides the Company with funding sufficient to complete the HTX-011 pivotal Phase 3 efficacy studies in the first half of 2018.

Genocea Biosciences Reports Second Quarter 2017 Financial Results

On August 9, 2017 Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing novel vaccines and immunotherapies targeting T cell antigens, reported financial results for the second quarter of 2017 (Press release, Genocea Biosciences, AUG 9, 2017, View Source [SID1234520117]). Genocea is developing GEN-003, an investigational immunotherapy for the treatment of genital herpes, and is applying its unique and proprietary T cell antigen identification platform, ATLAS, to immuno-oncology and cancer vaccine development.

Highlights of the Second Quarter of 2017 and Recent Events

May 2017 – In its first quarter earnings report, Genocea announced (i) the successful completion of its end-of-Phase 2 meeting for GEN-003 with the U.S. Food & Drug Administration and (ii) data from its prior GEN-003 Phase 2 trial indicating that the initial course of injections sustained clinical and virologic efficacy for at least 24 months.
June 2017 – Genocea announced its addition to the Russell 3000 and Russell 2000 Indices as part of the annual reconstitution of those indexes.
July 2017 – Genocea reported positive top-line 12-month Phase 2b data for GEN-003 including statistically significant data on the expected Phase 3 primary endpoint with the Phase 3 dose, and positive results on multiple secondary clinical endpoints.
Chip Clark, president and chief executive officer of Genocea, commented: "We are delighted with the recent positive GEN-003 12-month Phase 2b data and continue to explore means of securing capital for this program to enable the start of Phase 3. We believe that, if approved, GEN-003 could become the first new therapy to treat genital herpes in more than 20 years and address serious unmet patient needs.

"With respect to our cancer vaccine program, recent scientific publications on neoantigen cancer vaccines suggest that the concept has promise, but we think that there is significant room for improvement. We believe that Genocea’s ATLAS platform can enable better neoantigen selection and that, combined with our vaccinology expertise, positions us strongly in this emerging field. We expect to file an IND by the end of 2017 for our neoantigen vaccine, GEN-009, and we are also continuing our pre-clinical work on common antigen cancer vaccines and an EBV-related cancer vaccine."

Financial Guidance
Genocea expects that its existing cash and cash equivalents are sufficient to support its operating expenses and capital expenditure requirements into 2018. Genocea is currently exploring various avenues to secure capital to advance GEN-003 into Phase 3 trials and does not intend to commence Phase 3 development of GEN-003 until it has secured such capital.

Second-Quarter 2017 Financial Results

Cash Position: Cash and cash equivalents as of June 30, 2017 were $35.2 million compared to cash, cash equivalents and investments of $48.7 million as of March 31, 2017.
Research and Development (R&D) Expenses: R&D expenses for the quarter ended June 30, 2017 increased $4.7 million, to $11.4 million, from the same period in 2016. The increase was primarily driven by higher external manufacturing-related expenses and increases in compensation, consulting and professional services to support both the clinical drug supply and clinical planning activities in support of GEN-003 Phase 3 program readiness. Spending increases on Genocea’s immuno-oncology and cancer vaccine programs were driven primarily by increased manufacturing and compensation, consulting and professional services in anticipation of Genocea’s expected filing of an Investigational New Drug (IND) application for GEN-009 in 2017. Increased spending on these programs was offset by lower costs on deprioritized infectious disease programs.
General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2017 were $3.6 million, compared to $4.0 million for the same period in 2016 reflecting lower depreciation costs and lower consulting and professional services costs.
Net Loss: Net loss was $15.4 million for the quarter ended June 30, 2017, compared to a net loss of $11.0 million for the same period in 2016.

Genmab Announces Financial Results for the First Half of 2017

On August 9, 2017 Genmab reported Interim Report for the First Half of 2017 (Press release, Genmab, AUG 9, 2017, View Source [SID1234520116]).

Highlights
USD 554 million in net sales of DARZALEX (daratumumab); resulting in royalty income of DKK 454 million

DARZALEX approved by U.S. Food and Drug Administration (FDA) in combination with pomalidomide and dexamethasone for relapsed or refractory multiple myeloma

DARZALEX received European regulatory approval in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for relapsed or refractory multiple myeloma

Announced Phase III study combining daratumumab with pomalidomide and dexamethasone in multiple myeloma

Announced plans for new studies of daratumumab in smoldering multiple myeloma and with subcutaneous formulation in amyloidosis and multiple myeloma

Reported preliminary Phase I/II tisotumab vedotin data in cervical cancer

"It’s been another exciting and busy quarter, with highlights including further DARZALEX label expansions allowing for a broader number of multiple myeloma patients to be treated with the drug, as well as the announcement of a number of new pivotal studies with daratumumab. In addition we announced encouraging data from our ongoing Phase I/II tisotumab vedotin trial and have been working hard on progressing projects in our innovative pipeline," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Financial Performance First Half of 2017
Revenue was DKK 1,024 million in the first half of 2017 compared to DKK 524 million in the first half of 2016. The increase of DKK 500 million, or 95%, was mainly driven by higher DARZALEX royalties and milestones.

Operating expenses were DKK 442 million in the first half of 2017 compared to DKK 366 million in the first half of 2016. The increase of DKK 76 million, or 21%, was due to the additional investment in our pipeline of products, including the advancement of tisotumab vedotin, HexaBody-DR5/DR5, DuoBody-CD3xCD20, and other products in our pipeline.

Operating income was DKK 582 million in the first half of 2017 compared to DKK 158 million in the first half of 2016. The increase of DKK 424 million, or 268%, was driven by higher revenue, which was partly offset by increased operating expenses in 2017.

On June 30, 2017, Genmab had a cash position of DKK 5,215 million compared to DKK 3,922 million at December 31, 2016. This represented a net increase of DKK 1,293 million, which was mainly driven by positive working capital adjustments of DKK 630 million related to milestones achieved in the fourth quarter of 2016 that were received in 2017, our operating income of DKK 582 million, and proceeds from the exercise of warrants of DKK 194 million.

Fortress Biotech Reports Second Quarter 2017 Financial Results and Recent Corporate Highlights

On August 9, 2017 Fortress Biotech, Inc. (NASDAQ: FBIO) ("Fortress"), a biopharmaceutical company dedicated to acquiring, developing and commercializing novel pharmaceutical and biotechnology products, reported financial results and recent corporate highlights for the second quarter ended June 30, 2017 (Press release, Fortress Biotech, AUG 9, 2017, View Source [SID1234520115]).

Dr. Lindsay A. Rosenwald, Fortress’ Chairman, President and Chief Executive Officer, said, "Fortress and our Fortress subsidiaries achieved important corporate and clinical milestones during the second quarter of 2017. Avenue Therapeutics completed a $38 million initial public offering, and is on track to initiate a Phase 3 clinical trial in the third quarter of IV tramadol in patients undergoing bunionectomy surgery. If approved, IV tramadol will be the only intravenous Schedule IV opioid for use in the United States. Avenue, along with Checkpoint Therapeutics, began trading their common shares on The NASDAQ Capital Market during the last week of June. In addition, Caelum Biosciences announced the dosing of the final patient in a Phase 1b clinical trial of its lead therapy, CAEL-101, in AL amyloidosis, with a data readout expected in the second half of 2017. Mustang Bio licensed three CAR T cell therapies from its research partner City of Hope, expanding its pipeline to five novel CAR T candidates."

Dr. Rosenwald continued, "Our strong business development engine continues to deliver on our goal of acquiring novel pharmaceutical and biotechnology products for development within Fortress and through our Fortress Companies. We look forward to continuing to execute on our business plan and reaching important milestones in the third quarter to add value for our shareholders."

Financial Results:

· As of June 30, 2017, Fortress’ consolidated cash and cash equivalents totaled $144.3 million, compared to $134.0 million at March 31, 2017, and $88.3 million at December 31, 2016, an increase of $10.3 million for the quarter and $56.0 million year-to-date. These totals as of June 30, 2017 exclude short-term investments of $20.0 million, restricted cash of $15.9 million and cash deposits with clearing organizations of $1.0 million.
· Net revenue totaled $50.7 million for the second quarter of 2017 and $95.4 million for the first six months of 2017, compared to $2.2 million for the second quarter of 2016 and $2.9 million for the first six months of 2016. Net total revenue for the second quarter ended June 30, 2017 includes $4.4 million of Fortress revenue and $46.3 million of revenue from National Holdings Corporation ("National"), which Fortress acquired in September 2016, with no revenue attributable to National prior to the acquisition.
· Research and development expenses were $11.7 million for the second quarter of 2017, of which $10.0 million was related to Fortress Companies, and $18.8 million for the first six months of 2017, of which $15.8 million was related to Fortress Companies. This compares to $6.3 million for the second quarter of 2016, of which $4.0 million was related to Fortress Companies, and $14.1 million for the first six months of 2016, of which $9.0 million was related to Fortress Companies. Non-cash stock-based compensation expense included in research and development for the second quarter of 2017 was $2.4 million, compared to $1.1 million for the second quarter of 2016, and $3.2 million for the first six months of 2017, compared to $2.4 million for the first six months of 2016.
· Research and development expenses from license acquisitions totaled $1.8 million for the second quarter of 2017 and $3.1 million for the first six months of 2017, compared to $2.0 million for the second quarter of 2016 and $2.1 million for the first six months of 2016.

· General and administrative expenses were $11.1 million for the second quarter of 2017, of which $7.0 million was related to Fortress Companies, and $21.4 million for the first six months of 2017, of which $12.8 million was related to Fortress Companies. This compares to $8.6 million for the second quarter of 2016, of which $3.7 million was related to Fortress Companies, and $16.6 million for the first six months of 2016, of which $6.6 million was related to Fortress Companies. Non-cash stock-based compensation expenses included in general and administrative expenses were $2.2 million for the second quarter of 2017, compared to $1.9 million for the second quarter of 2016, and $4.3 million for the first six months of 2017, compared to $3.5 million for the first six months of 2016.
· National’s operating expenses totaled $48.4 million for the second quarter of 2017 and $91.5 million for the first six months of 2017, with no expenses attributable to National prior to Fortress’ acquisition of the company in September 2016.
· Net loss attributable to common stockholders was $17.4 million, or $0.43 per share, for the second quarter of 2017, compared to a net loss attributable to common stockholders of $12.5 million, or $0.31 per share, for the second quarter of 2016. For the first six months of 2017, net loss was $29.3 million or $0.73 per share, compared to $24.7 million or $0.62 per share in the first six months of 2016.

Recent Fortress Biotech and Fortress Company Highlights:

Fortress Biotech, Inc.
· In June 2017, Ms. Robyn Hunter was named Chief Financial Officer of Fortress. Lucy Lu, M.D., Fortress’ former Chief Financial Officer, was appointed President and Chief Executive Officer of Fortress subsidiary Avenue Therapeutics, Inc.
· As of June 30, 2017, Fortress raised a total of $19.0 million in a Subordinated Note Financing; $15.7 million of the $19.0 million was raised in the second quarter of 2017. National Securities Corporation, a subsidiary of National, acted as the Placement Agent.

Avenue Therapeutics, Inc.
· In May 2017, Avenue received a Notice of Allowance from the U.S. Patent and Trademark Office ("USPTO") for a new patent application (U.S. Application No. 15/163,111) titled "Intravenous Administration of Tramadol." The patent application describes and claims a dosing regimen of intravenous ("IV") 50 mg tramadol that provides certain pharmacokinetic parameters that are similar to those of 100 mg tramadol HCl administered orally every six hours at a steady state. Issuance of the patent (U.S. Patent No. 9,693,949) occurred in July 2017. This patent application falls under Avenue’s licensing agreement with Revogenex Ireland Ltd.
· On June 30, 2017, Avenue completed its initial public offering of 6,325,000 shares of common stock, at a public offering price of $6.00 per share, for a total offering size of $37,950,000, before deducting underwriting discounts and offering expenses. The shares sold include 825,000 that were subject to an underwriters’ overallotment option, which was exercised and closed concurrently with the closing of the initial public offering. Avenue’s common stock began trading on The NASDAQ Capital Market under the ticker symbol "ATXI."
· Also in June 2017, Lucy Lu, M.D., was named President and Chief Executive Officer of Avenue, a position she held on an interim basis since the company’s inception.

Caelum Biosciences, Inc.
· In April 2017, the U.S. Department of Health & Human Services confirmed the transfer of two U.S. Food and Drug Administration ("FDA") Orphan Drug Designations for CAEL-101 from Columbia University ("Columbia") to Caelum. The designations cover use as a therapeutic agent for patients with AL amyloidosis and use as a radio-imaging agent in amyloidosis. Caelum in-licensed CAEL-101 from Columbia in January 2017.
· In May 2017, Columbia dosed the final patient in the Phase 1b trial of CAEL-101 in AL amyloidosis. As of July 2017, all patients completed treatment. Preliminary and full Phase 1b data are expected in the second half of 2017.
· Also in May 2017, Caelum entered a biopharmaceutical manufacturing agreement with Patheon N.V. for process development and current good manufacturing practices (cGMP) production to support Phase 2/3 studies of CAEL-101.
· In June 2017, Columbia filed a provisional patent application with the USPTO pertaining to CAEL-101 that, once converted into a U.S. non-provisional utility application, will provide composition of matter protection effective upon a grant of a U.S. patent. The legal protection offered by a granted U.S. patent will exceed any data exclusivity periods associated with Orphan Drug Designation and/or an original, branded-biologic product approved for marketing in the U.S. Just this month, Columbia filed a second provisional patent application with the USPTO to pursue additional method of treatment claims directed to surprising and positive outcomes observed from the Phase 1b trial of CAEL-101. If granted these new claims provide an additional layer of legal protection for the use of Caelum’s lead product candidate.

Checkpoint Therapeutics, Inc.
· In April 2017, Checkpoint presented preclinical data on CK-101, an epidermal growth factor receptor ("EGFR") inhibitor, and CK-301, an anti-programmed cell death ligand-1 ("PD-L1") antibody, in poster sessions at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting.
· In June 2017, Checkpoint’s common stock began trading on The NASDAQ Capital Market under the ticker symbol "CKPT."