On August 3, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its second quarter 2017 financial results and provided an update on the company, including its progress with IPI-549, an oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma) (Press release, Infinity Pharmaceuticals, AUG 3, 2017, View Source [SID1234520032]).

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Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a four-part Phase 1 study in patients with advanced solid tumors. Infinity announced today that it has completed an evaluation of escalating monotherapy doses of IPI-549 ranging from 10 mg once daily (QD) to 60 mg QD and has selected the 60 mg dose for evaluation in the monotherapy expansion component of the study. The selection of the 60 mg QD dose was based on pharmacokinetic and pharmacokinetic analyses, which showed that IPI-549 maintained full suppression of PI3K-gamma at this dose level. Data also showed that the IPI-549 dosed at 60 mg QD was well tolerated, and no dose-limiting toxicities were observed. The monotherapy expansion component of the study is now open for enrollment and will evaluate the safety and activity of IPI-549 dosed at 60 QD in approximately 25 patients.

Dose escalation evaluating IPI-549 in combination with Opdivo is ongoing, and Infinity expects to initiate the combination expansion component of the study in the second half of 2017. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

"We have initiated the monotherapy expansion component of our Phase 1 study and are eager to also move forward with the combination expansion, allowing us to generate additional clinical and translational data from monotherapy treatment as well as from the combination of IPI-549 and Opdivo in specific types of solid tumors," stated Adelene Perkins, Infinity’s chair and chief executive officer. "We also recently undertook an important strategic initiative and amended our license agreement with Takeda Oncology for IPI-549. We issued Takeda an unsecured $6.0 million convertible note in exchange for eliminating future royalty obligations on IPI-549. This amendment reflects our strong belief in the potential of IPI-549 to be a first-in-class, oral immuno-oncology therapy and reduces the total royalty burden on any future net sales of IPI-549 to four percent due to Mundipharma and Purdue from a previous agreement."

The ongoing Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and activity of IPI-549 as a monotherapy and in combination with the approved dose of Opdivo in approximately 175 patients with advanced solid tumors. The study includes four parts: monotherapy dose escalation, combination therapy dose escalation, monotherapy expansion and combination therapy expansion. The combination expansion component of the study will include multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma and squamous cell carcinoma of the head and neck (SCCHN) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint blockade therapy. There is a great need for additional treatment options for the growing number of patients living with these cancers, which account for more than 17 percent of all new cancer cases in the U.S.1,2

Recent Developments

Agreement with Takeda Oncology amended: In July, Infinity amended its license agreement with Takeda Oncology for IPI-549. Under the amended agreement, Infinity will no longer have an obligation to pay Takeda future royalties on worldwide net sales of selective inhibitors of PI3K-gamma, including IPI-549. In exchange for eliminating the royalty obligation, Infinity issued to Takeda an unsecured $6.0 million convertible note that matures on July 26, 2018, and accrues interest at an annual rate of eight percent. The company is obligated to pay the principal amount together with any accrued interest on or before the maturity date in cash or in shares of Infinity common stock, at the election of Takeda. The share payment price would equal the average closing price of Infinity’s common stock for the 20 days prior to the payment date.

Infinity remains obligated to pay development, regulatory and commercial milestones to Takeda for IPI-549. The remaining milestones comprise up to a total of $5 million in development milestones, up to $50 million in success-based regulatory milestones, and up to $115 million in commercial milestones, which are due once certain sales thresholds have been met.

Under a previous agreement, Infinity is obligated to pay Mundipharma International Corporation Limited and Purdue Pharmaceutical Products L.P. a four percent royalty in the aggregate on worldwide net sales of IPI-549, which steps down to one percent in the U.S. after a certain sales threshold is met.
Recommended IPI-549 monotherapy dose determined and monotherapy expansion initiating: Infinity reported today that it has completed patient enrollment in the monotherapy dose-escalation component of the Phase 1 study and has selected IPI-549 dosed at 60 mg QD as the recommended dose for evaluation in the monotherapy expansion. Infinity selected the 60 mg QD dose based on pharmacokinetic and pharmacodynamic analyses, which showed that IPI-549 maintained full suppression of PI3K-gamma at this dose level. Data also showed that IPI-549 dosed at 60 mg QD was well tolerated, and no dose-limiting toxicities were observed. The monotherapy expansion is now open for patient enrollment.
IPI-549 combination dose escalation ongoing: Infinity also announced today that it is continuing to evaluate data from the dose-escalation component of the Phase 1 study assessing IPI-549 30 mg QD in combination with the approved dose of Opdivo. The company expects to complete combination dose escalation and initiate the combination expansion component of the study in the second half of 2017.
Presentation of preclinical rationale for IPI-549 at Precision: Lung Cancer World R&D Summit: In July, Jeffery Kutok, M.D., Ph.D., Infinity’s chief scientific officer, gave an oral presentation entitled "Reprogramming Tumor-Associated Macrophages by Targeting PI3K-Gamma with IPI-549" at the 2017 Precision: Lung Cancer World R&D Summit held in Boston, MA. In his presentation, Dr. Kutok discussed the preclinical rationale for targeting PI3K-gamma previously reported in two publications in Nature.3,4. Preclinical research in solid tumor models has demonstrated that PI3K-gamma blockade in tumor-associated macrophages by IPI-549 results in a transcriptional reprogramming of the M2, or pro-tumor macrophage phenotype, to the M1, or anti-tumor phenotype. In this setting, IPI-549 was active as a monotherapy and showed even greater activity in combination with checkpoint inhibitor therapies. Importantly, in specific models resistant to checkpoint inhibitor therapy, IPI-549 in combination with checkpoint inhibitors was able to overcome this resistance. Taken together, these preclinical data demonstrate that PI3K-gamma plays a key role in the immuno-suppressive tumor microenvironment and provide a strong rationale for the ongoing Phase 1 study.

Dr. Kutok also summarized the Phase 1 clinical data previously reported at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017. These data demonstrated that IPI-549 was well tolerated both as a monotherapy and in combination with Opdivo.5 Additionally, data from the monotherapy component of the study showed IPI-549 has a favorable pharmacokinetic and pharmacodynamic profile that supports once daily dosing.5
Exited lease for Infinity’s office facility at 784 Memorial Drive: In June, Infinity exited its lease agreement for its office facility, allowing the lease to end effective August 31, 2017. Infinity made a termination payment of $4.5 million in June 2017 and will make a final payment of $0.5 million in August 2017. The original lease agreement was to expire in March 2025. Future payment obligations on the lease, excluding operating costs and taxes, as of June 30, 2017, decreased from $16.9 million to $0.2 million. Infinity will be moving into office space with a significantly smaller lease obligation.
Second Quarter 2017 Financial Results

At June 30, 2017, Infinity had total cash and cash equivalents of $66.2 million, compared to $75.4 million at March 31, 2017. Cash used for operating activities during the second quarter of 2017 included payments of $0.8 million related to the company’s 2016 restructuring activities and $4.5 million related to exiting the company’s lease for 784 Memorial Drive.
Infinity did not record any revenue during the second quarter of 2017. Revenue for the second quarter of 2016 was $9.5 million, all of which related to Infinity’s previous collaboration agreement with AbbVie Inc.
Research and development (R&D) expense for the second quarter of 2017 was $3.9 million, compared to $52.9 million for the same period in 2016. The decrease in R&D expense was primarily due to the decrease in clinical development expenses following Infinity’s out-license of duvelisib in addition to the company’s 2016 restructuring activities.
General and administrative (G&A) expense was $6.2 million for the second quarter of 2017 compared to $15.7 million for the same period in 2016. The decrease in G&A expense was primarily due to the company’s 2016 restructuring activities.
Other expense of $6.9 million for the second quarter of 2017 related to expense recognized to exit the company’s facility lease agreement.
Net loss for the second quarter of 2017 was $17.0 million, or a basic and diluted loss per common share of $0.34, compared to net income of $53.0 million, or a basic and diluted earnings per common share of $1.05, for the second quarter of 2016. During the second quarter of 2016, Infinity recorded a non-recurring gain on AbbVie opt-out of the duvelisib collaboration of $112.2 million.
Cash and Investments Outlook
Infinity’s 2017 financial outlook remains as follows:

Net loss: Infinity expects net loss for 2017 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2017 with a cash, cash equivalents and available-for-sale securities balance ranging from $40 million to $50 million.
Based on its current operational plans, Infinity expects that its existing cash and cash equivalents at June 30, 2017, will be adequate to satisfy the company’s capital needs into the first quarter of 2019. The company’s financial outlook excludes additional funding or business development activities.

Conference Call Information
Infinity will host a conference call today, August 3, 2017, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 40376609. An archived version of the webcast will be available on Infinity’s website for 60 days.

About the IPI-549 and the Ongoing Phase 1 Study
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor to an anti-tumor phenotype and is able to overcome resistance to checkpoint inhibition.3,4 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

A Phase 1 study of IPI-549 in patients with advanced solid tumors is ongoing to explore the activity, safety, tolerability, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in patients with advanced solid tumors.6 The four-part study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The expansion component of the study evaluating IPI-549 plus Opdivo will include patients with non-small cell lung cancer (NSCLC), melanoma and squamous cell carcinoma of the head and neck (SCCHN). Patients enrolled in these combination expansion cohorts represent a difficult-to-treat population, as they must have demonstrated initial resistance or subsequently develop resistance to a PD-1 or PD-L1 therapy immediately prior to enrolling in the study. Overall, the study is expected to enroll approximately 175 patients.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On August 3, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported progress on its clinical development programs and its financial results for the second quarter ended June 30, 2017 (Filing, Q2, GlycoMimetics, 2017, AUG 3, 2017, View Source [SID1234520031]).

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"In the second quarter of 2017, GlycoMimetics achieved multiple milestones for its GMI-1271 program, a highlight of which was the FDA’s granting of Breakthrough Therapy designation to GMI-1271 for the treatment of relapsed/refractory AML patients. This milestone was coupled with the presentation of updated data from our ongoing Phase 1/2 clinical trial at both the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) meetings and the completion of a public offering of our common stock, from which we raised gross proceeds of $92.6 million to significantly extend our cash runway. We believe that these milestones provide evidence of the opportunity residing in our GMI-1271 program and our drug development platform more generally. The achievements during the second quarter have importantly underscored the differentiated potential of GMI-1271," noted Rachel King, Chief Executive Officer.

"The recent spotlight on our fast-progressing program in AML, however, should not obscure the potential of our platform and broad pipeline opportunities. In particular, GMI-1359, is a compound with potential in multiple cancer indications and is now in Phase 1 trials in healthy volunteers. During 2018, we plan to identify the initial patient population in which we will evaluate this compound. In addition, according to our collaborator Pfizer, our most advanced clinical development program is on track to complete enrollment of the Phase 3 pivotal trial in the second half of 2018. This is to evaluate GlycoMimetics’ drug candidate, rivipansel, for the treatment of vaso-occlusive crisis of sickle cell disease," she added.

Key Operational Highlights for the Second Quarter of 2017:
·

The company presented new data from its Phase 1/2 AML trial of GMI-1271 at the June 2017 annual meetings of ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper). In the relapsed or refractory disease arm of the trial, 66 patients had been enrolled. Of the 54 relapsed/refractory AML patients for whom data were available, the CR/CRi rate was 41%. The mortality rate among this group at 60 days was 7%. We believe these results compare favorably to what would be expected in this population, based on published historical controls in similar patients. Researchers also observed a median E-selectin ligand expression of 35% at baseline, with higher rates among those patients in this cohort who achieved remission. In the newly-diagnosed, treatment-naïve, elderly patient arm of the trial, 25 patients had been enrolled. Among these 25 patients, the CR/CRi rate was 68%, with a 73% rate for patients with de novo disease and 64% for patients with secondary AML.

·
In May 2017, the company completed enrollment of 91 patients in the Phase 1/2 trial of GMI-1271. The Phase 2 portion of the trial included one cohort of 25 patients over 60 years of age with newly diagnosed AML and a second cohort of 44 patients with relapsed or refractory AML. Unlike in the Phase 1 portion, some of the patients in the Phase 2 portion may be treated with multiple cycles of GMI-1271. We plan to provide additional updates from this clinical trial in the second half of 2017 and in 2018. We also intend to discuss with the FDA the design of a potential pivotal trial that could support an application for marketing approval for GMI-1271 for the treatment of AML.

·
In May 2017, GMI-1271 received Breakthrough Therapy designation from the FDA for the treatment of adults with relapsed/refractory AML. The FDA grants Breakthrough Therapy designation to companies to help accelerate development and review of drug candidates when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. The designation is designed to expedite the development and review of designated therapies, without changing FDA standards for new drug approval. In addition, in May 2017, the European Commission, based on a favorable recommendation from the European Medicines Agency Committee for Orphan Medicinal Products, granted orphan product designation for GMI-1271 for



the treatment of AML.
Second Quarter 2017 Financial Results:

·

Cash position: As of June 30, 2017, GlycoMimetics had cash and cash equivalents of $119.1 million as compared to $40.0 million as of December 31, 2016. The Company raised $86.8 million in net proceeds from the public offering of common stock completed in May 2017 and an additional $7.4 million in net proceeds under an at-the-market equity facility that was terminated in May 2017 in connection with the public offering.

·

R&D Expenses: The company’s research and development expenses decreased to $5.7 million for the quarter ended June 30, 2017 as compared to $5.8 million for the second quarter of 2016. The decrease was primarily caused by a decrease in expenses related to non-clinical toxicology studies and manufacturing and process development for its earlier-state drug candidate, GMI-1359, partially offset caused by the on-going costs associated with the ongoing clinical trials of GMI-1271 for the treatment of AML and multiple myeloma (MM).

·

G&A Expenses: The company’s general and administrative expenses increased to $2.5 million for the quarter ended June 30, 2017 as compared to $2.3 million for the second quarter of 2016. These increases were primarily attributable to annual salary adjustments and additional stock-based compensation expense caused by 2017 equity awards to employees and directors.

·

Shares Outstanding: Shares of common stock outstanding as of June 30, 2017 were 32,716,357.

About GMI-1271
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 2 clinical trial that has now completed enrollment, GMI-1271 is being evaluated in both elderly and relapsed/refractory patients with AML. In both populations, patients treated with GMI-1271 together with standard chemotherapy have continued to achieve higher than expected remission rates based on historical controls, as well as lower than expected induction-related mortality rates. Importantly, treatment in this patient population has been well tolerated with minimal adverse effects.






About GMI-1359
GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and MM or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 is currently in Phase 1 testing in healthy volunteers.

On August 3, 2017 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the quarter ended June 30, 2017, and provided an overview of recent accomplishments and plans (Press release, CytRx, AUG 3, 2017, View Source [SID1234520029]).

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"We are very proud of the tireless work undertaken over the past several months to secure a high-value, strategic partnership with NantCell Inc. which charts the best path forward for aldoxorubicin, through both expansion into multiple tumor types beyond soft tissue sarcomas (STS) and innovative combinations with immuno-oncology and cell-based therapies," said Steven A. Kriegsman, CytRx’s Chairman and CEO. "We believe NantCell has the vision and expertise to maximize aldoxorubicin’s potential and bring it to the patients and physicians who need it. Looking ahead for CytRx, we are rapidly expanding our pipeline of ultra-high potency oncology candidates at our laboratory in Freiburg, Germany, using our LADR (Linker Activated Drug Release) technology platform, a discovery engine designed to leverage CytRx’s expertise in albumin biology and linker technology for the development of a new class of potential breakthrough anti-cancer therapies."

Second Quarter 2017 and Recent Highlights
Entered an Exclusive Global Licensing Agreement and Strategic Investment with NantCell for Aldoxorubicin. Last week, CytRx and NantCell, Inc. (a private subsidiary of NantWorks, LLC) executed a global strategic licensing agreement giving exclusive rights to NantCell to develop and commercialize aldoxorubicin for all indications. The NantCell team is led by Dr. Patrick Soon-Shiong, who developed, gained regulatory approval under a 505(b)(2) pathway, and commercialized Abraxane, an albumin-mediated cytotoxic agent which currently grosses approximately $1 billion in annual sales. Under the terms of the agreement, NantCell purchased $13 million of CytRx common stock at a per share price of $1.10 representing approximately a 92% premium to the current market price. CytRx is eligible to receive up to an additional $343 million in regulatory and commercial milestones, plus increasing double-digit royalties on sales in aldoxorubicin’s lead indication of soft tissue sarcomas, and mid to high single-digit royalties for any additional indications. CytRx also issued NantCell a warrant to purchase up to 3 million shares of common stock at $1.10 over the next 18 months.

Amended Long-Term Loan Facility. In conjunction with the NantCell transaction, CytRx amended its long-term loan facility and made a payment of $5 million to the lender upon the closing of the exclusive global license and strategic investment for aldoxorubicin.

Appointed Dr. Shanta Chawla Senior Vice President of Drug Development. In June 2017, CytRx announced the appointment of Shanta Chawla, M.D., as the Senior Vice President of Drug Development. Dr. Chawla has served as Vice President of Clinical Development for three years and will assume the leadership of CytRx’s clinical and regulatory functions.

Presented Aldoxorubicin Clinical Trial Data in Patients with Soft Tissue Sarcomas (STS) at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper). In June 2017, CytRx presented results from its global Phase 3 clinical trial evaluating aldoxorubicin versus investigators’ choice in patients with relapsed and refractory STS (n=246) at ASCO (Free ASCO Whitepaper) 2017. An oral presentation given by Sant Chawla, M.D., F.R.A.C.P., Director of the Sarcoma Oncology Center in Santa Monica and principal investigator, described how aldoxorubicin demonstrated median progression-free survival (PFS) of 5.32 months, compared to a median PFS of 2.96 months for investigator’s choice therapy, a statistically significant improvement of 2.36 months (p=0.007; hazard ratio (HR)=0.62, 95% CI 0.44-0.88), representing a 38% reduction in the risk of tumor progression. In patients treated in North America plus Australia (n=312), aldoxorubicin demonstrated a median PFS of 4.21 months, compared to a median PFS of 2.96 months for investigator’s choice therapy, a statistically significant improvement of 1.25 months (p=0.023, HR=0.71, 95% CI 0.53-0.96). In the overall intent to treat (ITT) trial population (n=433), aldoxorubicin performed better than investigator’s choice demonstrating a median PFS of 4.11 months, compared to a median PFS of 2.96 months for investigator’s choice therapy, narrowly missing statistical significance (p=0.087; HR=0.81, 95% CI 0.64-1.03). All responses in this study were determined by an independent, blinded central lab assessment of scans.

Presented Aldoxorubicin and Ifosfamide/Mesna Combination Trial Data in Patients with Metastatic or Locally Advanced Sarcomas at ASCO (Free ASCO Whitepaper). In June 2017, CytRx presented updated data from its ongoing Phase 1/2 clinical trial combining aldoxorubicin with ifosfamide/mesna in patients with first-line soft tissue sarcomas at ASCO (Free ASCO Whitepaper) 2017. Of the 44 evaluable patients as of May 10, 2017, 16 patients (36%) achieved a partial response (PR), 25 patients (57%) achieved SD, with 20 patients (45%) achieving SD for ≥4 months, for an overall disease control rate (DCR) of 82% (PR+SD≥4). Twenty-two of 44 (50%) patients received at least 6 cycles of aldoxorubicin (>1,300 mg/m2 cumulative doxorubicin equivalent). As of the data cutoff date, the median PFS had not been reached.

Completed $15 Million Financing. In May 2017, CytRx closed the sale of 30 million shares of common stock in a public offering at a price of $0.50 per share, resulting in net proceeds of approximately $14.0 million after deducting placement agent’s fees and other estimated offering expenses. Additionally, the Company received approximately $2.8 million from the exercise of warrants resulting in a total raise of $16.8 million in the second quarter. Subsequent to the end of the second quarter, 6.3 million warrants with a strike price of $0.70 expired on July 19, 2017.

Second Quarter 2017 Financial Results
CytRx reported cash, cash equivalents and short-term investments of $55.0 million as of June 30, 2017. Subsequent to the close of the quarter, CytRx entered into a global strategic licensing agreement for aldoxorubicin with NantCell Inc., and received a strategic investment of $13.0 million. Concurrent with the closing of the aldoxorubicin license agreement, CytRx amended its existing long-term loan and made a payment of $5 million to the lender.

On May 2, 2017, CytRx completed a public offering of 30 million shares of its common stock at a price of $0.50 per share. The net proceeds to CytRx from the offering, after deducting placement agent’s fees and other estimated offering expenses, were approximately $14.0 million. In addition, CytRx received $2.8 million in proceeds from the exercise of warrants in the second quarter of 2017.

Net loss for the quarter ended June 30, 2017, was $14.4 million, or $(0.10) per share, compared with a net loss of $18.3 million, or $(0.27) per share, for the quarter ended June 30, 2016, a reduction of $3.9 million. During the second quarter of 2017, the Company recognized a non-cash loss of $4.3 million on the fair value adjustment of warrant derivative liability related to warrants issued in 2016, compared to a non-cash gain of $0.9 million during the second quarter of 2016 related to now expired warrants.

Research and development (R&D) expenses were $6.2 million for the second quarter of 2017, and included development expenses of $3.7 million for aldoxorubicin, approximately $0.9 million for pre-clinical development of new albumin-binding, ultra-high potency cancer drugs (German lab), and approximately $1.6 million for non-cash expenses and general operation of our clinical programs. This is a reduction of approximately 50 percent compared to R&D expenses of $12.5 million for the second quarter of 2016.

General and administrative (G&A) expenses were $3.1 million for the second quarter of 2017, compared with $6.1 million for the second quarter of 2016, a reduction of $3 million.

On August 3, 2017 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the U.S. Food and Drug Administration (FDA) has approved Vyxeos (daunorubicin and cytarabine) liposome for injection for the treatment of adults with two types of Acute Myeloid Leukemia (AML), a rapidly progressing and life-threatening blood cancer (Press release, Jazz Pharmaceuticals, AUG 3, 2017, View Source [SID1234520026]). Vyxeos is indicated for the treatment of adults with newly-diagnosed t-AML or AML-MRC.

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"Vyxeos is the first new chemotherapy advance in more than 40 years for adults with newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The FDA approval of Vyxeos reflects our commitment to addressing unmet needs within the hematology oncology community."

Designed with Jazz’s CombiPlex proprietary technology, Vyxeos is a unique liposomal formulation that delivers a fixed-ratio of daunorubicin and cytarabine to the bone marrow that has been shown to have synergistic effects at killing leukemia cells in vitro and in animal models. Vyxeos is the first product developed with the company’s proprietary CombiPlex platform, which enables the design and rapid evaluation of various combinations of therapies.

"Vyxeos is the first chemotherapy to demonstrate an overall survival advantage over the standard of care in a Phase 3 randomized study of older adults with newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes," said Jeffrey E. Lancet, MD, Chair of the Department of Malignant Hematology at Moffitt Cancer Center. "The prognosis for these patients is poor, so the FDA approval of this new drug provides a welcome therapeutic advance."

Vyxeos will be commercially available within a week. For more information about Vyxeos in the United States, please visit View Source

Vyxeos has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute Vyxeos for other daunorubicin- and/or cytarabine- containing products.

The FDA approval is based on data from a pivotal Phase 3 clinical trial that evaluated the efficacy and safety of Vyxeos compared to cytarabine and daunorubicin (7+3) in 309 patients 60 to 75 years of age with newly diagnosed t-AML or AML-MRC. In the Vyxeos arm, patients received 44mg/100mg per m2 (daunorubicin and cytarabine) liposome intravenously via a 90 minute infusion on days 1, 3 and 5 of induction (days 1 and 3 if a second induction was needed) and 29mg/65mg per m2 (daunorubicin and cytarabine) liposome on days 1 and 3 for consolidation. Patients in the 7+3 arm received induction with cytarabine 100mg/m2/day on days 1-7 by continuous infusion and daunorubicin 60mg/m2/day on days 1-3. For consolidation, cytarabine was dosed on days 1-5 and daunorubicin on days 1-2. For the primary endpoint of overall survival, Vyxeos demonstrated an improvement that was superior to the 7+3 treatment regimen. The median overall survival for the Vyxeos treatment group was 9.6 months compared with 5.9 months for the 7+3 treatment group (p = 0.005; HR = 0.69 [0.52, 0.90]). Vyxeos also demonstrated a statistically significant improvement in complete response rate of 38 percent versus 26 percent; p=0.036. The overall, all-cause 30-day mortality was 6 percent in the Vyxeos arm and 11 percent in the control arm. Six percent of patients in both the Vyxeos and control arm had a fatal adverse reaction on treatment or within 30 days of therapy that was not in the setting of progressive disease. During the first 60 days of the study, 14 percent of patients died in the Vyxeos arm versus 21 percent of patients in the 7+3 arm. In addition, the overall rate of hematopoietic stem cell transplant (HSCT) was 34 percent in the Vyxeos arm and 25 percent in the 7+3 arm. In the Phase 3 study, the most common adverse reactions (incidence ≥ 25%) were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders and vomiting.

About VyxeosTM

Vyxeos (daunorubicin and cytarabine) liposome for injection 44mg/100mg is a liposome formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion.1 Vyxeos is indicated for the treatment of adults with newly-diagnosed t-AML or AML-MRC. The FDA granted the Vyxeos application Priority Review status, designated Vyxeos as a Breakthrough Therapy and also granted Fast Track Designation. Vyxeos received Orphan Drug Designation by the FDA and the European Commission for the treatment of AML.

Important Safety Information

Vyxeos has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute Vyxeos for other daunorubicin- and/or cytarabine- containing products.

Vyxeos should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine or any of its ingredients.

Vyxeos can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with Vyxeos. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

Vyxeos can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles or legs
unusual tiredness
Vyxeos may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
Vyxeos contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

Vyxeos can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

Vyxeos can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving Vyxeos. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of Vyxeos.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Please see full Prescribing Information for Vyxeos before prescribing: View Source

About AML

Acute myeloid leukemia (AML) is a blood cancer that begins in the bone marrow, which produces most of the body’s new blood cells.2 AML cells crowd out healthy cells and move aggressively into the bloodstream to spread cancer to other parts of the body.3 AML is a relatively rare disease representing 1.3 percent of all new cancer cases.4 It is estimated that more than 21,000 people will be diagnosed with AML in the United States this year with the potential for nearly 11,000 people to die from the disease.5 The median age at diagnosis is 68 years old,4 with rising age associated with a progressively worsening prognosis.6 There is also a reduced tolerance for intensive chemotherapy as patients age.7 AML has the lowest survival rate of any other form of leukemia.4 Patients with newly diagnosed t-AML or AML-MRC may have a particularly poor prognosis.8-10 A hematopoietic stem cell transplant (HSCT) may be a curative treatment option for patients.11

On August 3, 2017 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective drug candidates for the treatment of human cancers, reported its financial results for the second quarter ended June 30, 2017 (Press release, Curis, AUG 3, 2017, View Source [SID1234520025]).

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Curis also reported today data from the interim analysis of the Phase 2 trial of CUDC-907 in patients with MYC-altered DLBCL. Of the 36 evaluable patients with MYC-altered DLBCL in the interim analysis, 7 patients experienced confirmed durable objective responses (19.4% ORR), including 3 with complete responses. In addition, and consistent with the original hypothesis, all 7 responders in the Phase 2 study had MYC-altered disease, while no objective responses were observed in 12 patients with MYC-negative disease status.

"The ability of CUDC-907 treatment to result in durable complete responses, and the continued correlation of this benefit selectively in patients with MYC-altered disease, is encouraging for the continued development of CUDC-907," said Ali Fattaey, President and CEO. "While we believe the results from this Phase 2 trial are insufficient to serve as the basis of a request for accelerated approval of CUDC-907, we are evaluating alternative designs for a separate registration-enabling trial to demonstrate CUDC-907’s benefit for patients with MYC-altered DLBCL."

"The reported response rate is impressive, especially in this population of patients with relapsed/refractory MYC-altered lymphomas who rarely respond to salvage therapies," said Dr. Daniel J. Landsburg, Assistant Professor of Clinical Medicine at the Abramson Cancer Center at the Hospital of the University of Pennsylvania. "I believe this agent warrants continued investigation."

Second Quarter 2017 Financial Results

Curis reported a net loss of $14.1 million, or $0.10 per share, on both a basic and diluted basis for the second quarter of 2017, as compared to a net loss of $11.3 million, or $0.09 per share, on both a basic and diluted basis for the same period in 2016. Curis reported a net loss of $29.8 million, or $0.21 per share, on both a basic and diluted basis for the six months ended June 30, 2017, as compared to a net loss of $20.7 million, or $0.16 per share on both a basic and diluted basis for the same period in 2016.

Revenues for the second quarter of 2017 were $2.1 million, as compared to $1.7 million for the same period in 2016. Revenues for the six months ended June 30, 2017 were $4.2 million, as compared to $3.4 million for the same period in 2016. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge.

Operating expenses were $15.2 million for the second quarter of 2017, as compared to $12.4 million for the same period in 2016. Operating expenses for the six months ended June 30, 2017 were $32.4 million, as compared to $22.9 million for the same period in 2016, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.1 million for both the second quarter of 2017 and 2016. Cost of royalty revenues for the six months ended June 30, 2017 and 2016 were $0.2 million for both periods.

Research and Development Expenses. Research and development expenses were $11.3 million for the second quarter of 2017, as compared to $8.8 million for the same period in 2016. The increase was primarily due to increased direct spending related to clinical activities of CUDC-907 and CA-170 and increased employee-related expenses primarily due to additional headcount to support the multiple programs. Research and development expenses were $24.8 million for the six months ended June 30, 2017 as compared to $15.7 million for the same period in 2016.

General and Administrative Expenses. General and administrative expenses were $3.8 million for the second quarter of 2017 as compared to $3.4 million for the same period in 2016. The increase in general and administrative expenses was driven primarily by higher personnel costs and stock-based compensation expense due to increased headcount. General and administrative expenses were $7.4 million for the six months ended June 30, 2017, as compared to $7.1 million for the same period in prior 2016.

Other expense, net was $1.0 million for the second quarter of 2017, as compared to $0.6 million for the same period in 2016. Other expense, net primarily consisted of interest expense related to Curis Royalty’s (a wholly owned subsidiary of Curis) debt obligations. Other expense, net was $1.7 million and $1.2 million for the six months ended June 30, 2017 and 2016, respectively.

As of June 30, 2017, Curis’s cash, cash equivalents, marketable securities and investments totaled $51.0 million and there were approximately 143.9 million shares of common stock outstanding. On a fully-diluted basis, which includes 18.6 million options, there were 162.5 million shares outstanding.

Recent Operational Highlights

Precision oncology (CUDC-907: HDAC / PI3K inhibitor program):

The following summarizes the CUDC-907 Phase 1 trial results and Phase 2 interim analysis:

View Source

Immuno-oncology (CA-170: PD-L1 / VISTA antagonist program; Aurigene collaboration):

In May 2017, Curis presented the CA-170 Phase 1 trials-in-progress poster at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Upcoming Activities

Curis expects that it will make presentations at the following conferences through September 2017:

CA-170 poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Conference (Sept. 8-12, 2017) in Madrid.