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OncoSec Enters Research Collaboration Agreement with UCLA and Roger S. Lo, M.D., Ph.D.

On August 6, 2018 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, reported that it has entered into a research collaboration agreement with the University of California, Los Angeles (UCLA) on behalf of Roger S. Lo, M.D., Ph.D. and his research team (Press release, OncoSec Medical, AUG 6, 2018, View Source [SID1234528629]). Dr. Roger S. Lo, Professor of Medicine and Molecular and Medical Pharmacology in the UCLA David Geffen School of Medicine, Associate Chief of Dermatology and a member of the UCLA Jonsson Comprehensive Cancer Center, is a preeminent physician-scientist widely recognized for his work in understanding treatment-resistant melanoma.

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Under the research collaboration, Dr. Lo and his research team will perform genetic, transcriptomic and methylomic analyses of patients in OncoSec’s PISCES/KEYNOTE-695 Phase 2b clinical trial, which is evaluating TAVO (tavokinogene telseplasmid) in combination with KEYTRUDA (pembrolizumab) for the treatment of metastatic melanoma in patients that have progress after receiving all available treatments including anti PD-L1 checkpoint immunotherapy.

"UCLA and Dr. Lo represent an ideal collaborator for OncoSec to augment our ongoing PISCES/KEYNOTE-695 Phase 2b clinical program with specialized research designed to pinpoint the genetic and epigenetic mechanisms that correspond to specific clinical outcomes in the treatment of metastatic melanoma with TAVO in combination with pembrolizumab," said Christopher G. Twitty, Chief Scientific Officer of OncoSec. "The PISCES/KEYNOTE-695 Phase 2b study continues to progress as planned with enrollment in Stage 1 expected to be complete in the third quarter 2018 and topline data anticipated prior to year-end. Our collaboration with Lo Lab has the potential to not only offer important insights into the mechanism of action of TAVO in combination with pembrolizumab for the treatment of metastatic melanoma, but could be applicable across our clinical pipeline, including the ongoing KEYNOTE-890 Phase 2b trial of TAVO in combination with pembrolizumab in triple negative breast cancer."

Under Dr. Lo’s direction, his research team focuses on genomic, epigenomic and immunologic factors that shape the cancer’s evolution on molecularly targeted therapies and/or immune checkpoint inhibitors. Dr. Lo’s research has received funding from the National Cancer Institute, American Skin Association, Melanoma Research Alliance, The Melanoma Research Foundation, Ressler Family Foundation, and Steven C. Gordon Family Foundation.

Kura Oncology Reports Second Quarter 2018 Financial Results and Provides Corporate Update

On August 6, 2018 Kura Oncology, Inc., (Nasdaq: KURA) a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported second quarter 2018 financial results and provided a corporate update (Press release, Kura Oncology, AUG 6, 2018, View Source [SID1234528626]).

"I am very pleased with the continued progress we made over the past quarter, with a particular focus on operational execution," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We have enhanced our leadership team, improved our ability to screen and identify patients with HRAS mutations, positioned our pipeline to create additional opportunities and strengthened our balance sheet through a successful public offering. We believe the proceeds from this offering along with our existing funds give us the resources to advance our pipeline through a series of potential milestones, including data from a registration-directed trial of our lead drug candidate tipifarnib in HRAS mutant HNSCC."

Corporate Update

Updated data from RUN-HN at ESMO (Free ESMO Whitepaper) – An abstract relating to Kura’s ongoing Phase 2 trial of tipifarnib in HRAS mutant HNSCC has been accepted for oral presentation at the upcoming European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in Munich in October. The update will also include patients from an exploratory cohort in other HRAS mutant squamous cell carcinomas. Approximately 25 clinical sites are now open for enrollment worldwide and RUN-HN is currently enrolling at a rate of approximately two patients per month.

Screening collaboration to support enrollment – The company has signed an agreement with OncoDNA, an oncology-focused healthcare technology company based in Belgium, to support patient enrollment for Kura’s ongoing Phase 2 trial of tipifarnib in patients with HRAS mutant HNSCC. OncoDNA provides physicians internationally with next-generation sequencing for HNSCC oncogenic mutations, including HRAS, for patients who may be eligible to enroll in RUN-HN.

Registration-directed trial anticipated to initiate by year end – Kura has initiated startup activities for its upcoming registration-directed trial of tipifarnib in at least 59 recurrent or metastatic patients with HRAS mutant HNSCC. The company anticipates that the trial will be conducted at approximately 100 clinical sites worldwide and will take approximately two years to enroll. Based on feedback from the U.S. Food and Drug Administration, Kura believes that the single-arm trial, if positive, could support an application for accelerated approval.

HRAS mutant lung squamous cell carcinoma (LSCC) – An investigator-sponsored trial of tipifarnib in HRAS mutant LSCC has been initiated and is now open for enrollment. The proof-of-concept trial is being conducted in collaboration with the Spanish Lung Cancer Group, a cooperative group consisting of more than 150 public and private oncology centers in Spain.

Biomarker-enriched data in hematologic malignances – Kura is prospectively investigating the CXCL12 pathway and bone marrow homing as potential biomarkers of activity for tipifarnib in its three ongoing Phase 2 trials in hematologic malignancies. The PTCL trial was the first of the three to begin and is actively enrolling into two cohorts: 1) Patients with angioimmunoblastic T-cell lymphoma (AITL) and 2) patients with PTCL who have the absence of a single nucleotide variation in the 3’ untranslated region of the CXCL12 gene. The company expects to have biomarker-enriched data from PTCL and potentially other indications by the end of 2018.

Strengthened management team – Kura expanded its leadership team with the additions of Marc Grasso, M.D., and John Farnam. Dr. Grasso will join the company as Chief Financial Officer and Chief Business Officer on August 21, 2018, after 20 years in healthcare investment banking. Mr. Farnam joined Kura in the newly created position of Chief Operating Officer on July 1, 2018, from Celgene Receptos. The company has also added industry veterans Bridget Martell, M.D., and Blake Tomkinson, Ph.D., as Vice Presidents of Clinical Development.

Public offering of common stock – On July 2, 2018, Kura completed a public offering in which the company sold an aggregate of 4,600,000 shares of common stock at a price of $16.75 per share. Net proceeds from the public offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $74.5 million.

Upcoming Milestones

Update from ongoingPhase 2 trial of tipifarnib in HRAS mutant HNSCC at ESMO (Free ESMO Whitepaper) in October 2018

Initiation of registration-directed trial of tipifarnib in HRAS mutant HNSCC by the end of 2018

Dosing of first patient in investigator-sponsored study of tipifarnib in HRAS mutant LSCC

Biomarker-enriched data from Phase 2 trial of tipifarnib in PTCL by the end of 2018

Submission of an investigational new drug application for KO-539, a potent and selective menin-MLL inhibitor, in late 2018 or early 2019

Financial Results

Research and development expenses for the second quarter of 2018 were $11.5 million, compared to $5.7 million for the second quarter of 2017.

General and administrative expenses for the second quarter of 2018 were $3.8 million, compared to $2.3 million for the second quarter of 2017.

Net loss for the second quarter of 2018 was $14.7 million, or $0.45 per share, compared to $7.8 million, or $0.40 per share, for the second quarter of 2017.

Cash, cash equivalents and short-term investments totaled $125.9 million as of June 30, 2018, compared with $93.1 million as of December 31, 2017.

As adjusted for the $74.5 million in net proceeds resulting from the company’s public offering of common stock that closed on July 2, 2018, Kura had, on a pro forma basis, $200.4 million in cash, cash equivalents and short-term investments at June 30, 2018.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations through 2021.

Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, August 6, 2018, to discuss the financial results for the second quarter of 2018 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers

and entering the conference code: 1588245. A live webcast of the call will be available from the Investors and Media section of the company website at www.kuraoncology.com, and will be archived there for 30 days.

Vericel Reports Record Second Quarter Revenues of $19.0 Million and Raises Full Year 2018 Revenue Guidance

On August 6, 2018 Vericel Corporation (NASDAQ:VCEL), a leader in advanced cell therapies for the sports medicine and severe burn care markets, reported financial results and business highlights for the second quarter ended June 30, 2018 (Press release, Vericel, AUG 6, 2018, View Source [SID1234528625]).

Second Quarter 2018 Financial Highlights

Total net revenues of $19.0 million compared to $17.0 million in the second quarter of 2017; second quarter 2017 revenues included a favorable $1.4 million reversal of a revenue reserve for Carticel and MACI related to a contractual dispute between one of the Company’s pharmacy providers and a third-party payer;

Gross margins of 59% compared to gross margins of 55% on a GAAP basis and 51% on a non-GAAP basis excluding the impact of the revenue reserve reversal in the second quarter of 2017;

Net loss of $4.7 million, or $0.12 loss per share, compared to net loss of $2.4 million, or $0.07 per share on a GAAP basis and $3.7 million, or $0.11 per share, on a non-GAAP basis excluding the impact of the revenue reserve reversal, in the second quarter of 2017;

Non-GAAP adjusted EBITDA loss of $1.4 million compared to a loss of $2.7 million in the second quarter of 2017;

As of June 30, 2018, the company had $95.0 million in cash compared to $26.9 million in cash at December 31, 2017; and

Full year 2018 revenue guidance raised to $80 to $83 million compared to previous full year revenue guidance of $73 to $78 million.
Recent Business Highlights
During and since the second quarter of 2018, the company:

Reported record second quarter revenues marking the fifth consecutive quarter with record revenues for the reported quarter;

Deployed the expanded MACI sales force, which increased from 28 to 40 sales representatives;

Completed an expansion of MACI manufacturing capacity to support expected growth in MACI demand;
Implemented an expanded pharmacy distribution network to continue expansion of MACI payer access;

Closed a $74.8 million public offering; and

Joined the Russell 3000 Index.
"We continued our strong start to 2018 with solid revenue growth and expanding margins in the second quarter, and we believe that key performance indicators point to continued robust growth for MACI in the second half of the year," said Nick Colangelo, president and CEO of Vericel. "Moreover, based on our strengthened financial position, we are well positioned to execute on our business and strategic plans."

Second Quarter 2018 Results
Total net revenues for the quarter ended June 30, 2018 were $19.0 million, which included $14.1 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue and $4.9 million of Epicel (cultured epidermal autografts) net revenue, compared to $12.9 million of Carticel (autologous cultured chondrocytes) and MACI net revenue and $4.0 million of Epicel net revenue, respectively, in the second quarter of 2017. Total net revenues for the quarter ended June 30, 2017 included a favorable $1.4 million reversal of a revenue reserve for Carticel and MACI related to a contractual dispute between one of the Company’s pharmacy providers and a third-party payer.
Gross profit for the quarter ended June 30, 2018 was $11.3 million, or 59% of net revenues, compared to $9.3 million, or 55% of net revenues on a GAAP basis and 51% on a non-GAAP basis excluding the impact of the revenue reserve reversal, for the second quarter of 2017. See table reconciling non-GAAP measures for more details.
Total operating expenses for the quarter ended June 30, 2018 were $15.5 million compared to $11.8 million for the same period in 2017. The increase in operating expenses was due primarily to a $1.7 million increase in stock-based compensation expense, a $1.6 million increase in MACI related sales and marketing activities, and $0.7 million increase in R&D expense related to the preparations for a MACI pediatric clinical study in the U.S.
Loss from operations for the quarter ended June 30, 2018 was $4.2 million, compared to a loss of $2.5 million on a GAAP basis and $3.9 million on a non-GAAP basis excluding the impact of the revenue reserve reversal for the second quarter of 2017. Material non-cash items impacting the operating loss for the quarter in the current year included $2.5 million of stock-based compensation expense and $0.4 million in depreciation expense, compared to $0.8 million of

stock-based compensation expense and $0.4 million in depreciation expense in the second quarter of 2017.
Other expense for the quarter ended June 30, 2018 was $0.4 million compared to other income of $0.1 million for the second quarter of 2017.
Non-GAAP adjusted EBITDA loss was $1.4 million for the quarter ended June 30, 2018 compared to a loss of $2.7 million in the second quarter of 2017.
Vericel’s net loss for the quarter ended June 30, 2018 was $4.7 million, or $0.12 per share, compared to a net loss of $2.4 million, or $0.07 per share on a GAAP basis and $3.7 million, or $0.11 per share on a non-GAAP basis excluding the impact of the revenue reserve reversal, for the second quarter of 2017.
As of June 30, 2018, the company had $95.0 million in cash compared to $26.9 million in cash at December 31, 2017.
Full Year 2018 Financial Guidance
The company now expects total net product revenues for the full year 2018 to be in the range of $80 to $83 million, compared to the previous full year revenue guidance of $73 to $78 million.
Conference Call Information
Today’s conference call will be available live at 4:30pm Eastern time in the Investor Relations section of the Vericel website at View Source." target="_blank" title="View Source." rel="nofollow">View Source Please access the site at least 15 minutes prior to the scheduled start time in order to download the required audio software if necessary. To participate in the live call by telephone, please call (877) 312-5881 and reference Vericel Corporation’s second-quarter 2018 investor conference call. If calling from outside the U.S., please use the international phone number (253) 237-1173.
If you are unable to participate in the live call, the webcast will be available at View Source until August 6, 2019. A replay of the call will also be available until 7:30pm (EDT) on August 11, 2018 by calling (855) 859-2056, or from outside the U.S. (404) 537-3406. The conference ID is 9699288.

Jazz Pharmaceuticals and MD Anderson Cancer Center Collaborate to Evaluate Potential Treatment Options for Hematologic Malignancies

On August 6, 2018 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and The University of Texas MD Anderson Cancer Center reported a five-year collaboration agreement with a goal of evaluating therapies for multiple hematologic malignancies, including acute myeloid leukemia (AML) and myelodysplastic syndromes (Press release, Jazz Pharmaceuticals, AUG 6, 2018, View Source;p=RssLanding&cat=news&id=2362297 [SID1234528511]).

The joint effort brings together MD Anderson’s translational medicine and clinical research capabilities with Jazz’s hematology/oncology portfolio, including its FDA-approved medicines as well as current and potential future investigational therapies.

"This collaboration represents a significant opportunity to efficiently develop innovative therapies and therapeutic combinations," said Tapan Kadia, M.D., associate professor of Leukemia at MD Anderson. "Our aim is to always provide leading-edge care for our leukemia patients, and it is our hope that this joint effort will result in new treatment solutions."

Jazz and MD Anderson will pursue research opportunities in areas of high unmet need. The initial focus of the collaboration is to evaluate and generate additional data for Vyxeos (daunorubicin and cytarabine) liposome for injection, in new patient populations and in combination with other therapies.

"Jazz is committed to providing meaningful medicines for people with hematologic cancers, particularly those with serious unmet clinical needs," said Allen Yang, M.D., Ph.D., vice president and acting chief medical officer of Jazz Pharmaceuticals. "We look forward to collaborating with MD Anderson to help advance treatment options for patients as part of our growing hematology oncology therapeutic area."

Vyxeos received FDA approval in August 2017 for the treatment of adults with newly-diagnosed therapy-related (t-AML) or AML with myelodysplasia-related changes (AML-MRC), which represents part of high-risk or secondary AML populations. AML-MRC is more common in older patients who often do not respond well to intensive chemotherapy; while t-AML can occur as a result of previous chemotherapy or radiation therapy.

About Vyxeos
Vyxeos is a liposome formulation of a fixed combination of daunorubicin and cytarabine for intravenous infusion. Vyxeos is indicated for the treatment of adults with newly-diagnosed t-AML or AML-MRC. For more information about Vyxeos in the United States, please visit View Source

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 including BOXED Warning at View Source

Neon Therapeutics Reports Second Quarter 2018 Financial Results and Recent Business Highlights

On August 6, 2018 Neon Therapeutics, Inc. (Nasdaq: NTGN), a clinical-stage immuno-oncology company developing neoantigen-targeted therapies, reported financial results and provided a business update for the second quarter of 2018 (Press release, Neon Therapeutics, AUG 6, 2018, View Source [SID1234528483]).

"The first half of 2018 was transformative for Neon, highlighted by the successful completion of our initial public offering. The capital raised will enable the continued execution of our strategy to develop neoantigen-targeted therapies that have the potential to transform patient lives," said Hugh O’Dowd, President and Chief Executive Officer of Neon. "We are pleased by the continued progress across our entire product portfolio of vaccine and T-cell programs, including completion of enrollment of our first Phase 1b clinical trial for NEO-PV-01. In addition, updated data from this trial will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October 2018."

Second Quarter Business Highlights

Neon achieved several key operational milestones during the second quarter of 2018, including the following:

· In June 2018, Neon completed its initial public offering of common stock, raising $100 million in gross proceeds.

· In May 2018, Neon announced that the first patient was dosed in NT-002, its Phase 1b clinical trial evaluating the company’s personal neoantigen vaccine, NEO-PV-01, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), along with chemotherapy.

· In April 2018, Neon presented updated data from NT-001, its Phase 1b clinical trial of NEO-PV-01 in the metastatic setting, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

Pipeline Overview and Upcoming Milestones

NEO-PV-01

· NT-001: Phase 1b Clinical Trial of NEO-PV-01 in the Metastatic Setting

· Updated data expected in October 2018 at ESMO (Free ESMO Whitepaper)

· 52-week data expected in the first half of 2019

· NT-002: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Non-Small Cell Lung Cancer

· 52-week data expected in the second half of 2019

· NT-003: Phase 1b Clinical Trial of NEO-PV-01 in Metastatic Melanoma Combinations

· Trial initiation expected in the second half of 2018

· NT-004: Phase 1b Clinical Trial of NEO-PV-01 in earlier disease setting

· Planning ongoing

NEO-PTC-01

· Phase 1 Clinical Trial in Solid Tumor Setting: Neon intends to submit a European Clinical Trial Application (CTA) for NEO-PTC-01, a personal neoantigen T-cell therapy, in the first half of 2019.

NEO-SV-01

· Phase 1 Clinical Trial in Subset of ER+ Breast Cancer: Following the completion of target validation and preclinical product development work, Neon expects to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in the first half of 2019.

Second Quarter 2018 Financial Results

· Cash Position: As of June 30, 2018, cash, cash equivalents and marketable securities were $138.6 million, as compared to cash, cash equivalents and marketable securities of $79.7 million as of December 31, 2017.

· R&D Expenses: R&D expenses were $14.8 million for the quarter ended June 30, 2018, as compared to $7.3 million for the same quarter last year. The increase of $7.5 million was due primarily to increased costs related to the advancement of NEO-PV-01, including increased external manufacturing and other external costs to support our ongoing and planned clinical trials, as well as increased personnel-related costs due to additional headcount to support the growth of our research and development organization.

· G&A Expenses: G&A expenses were $4.3 million for the quarter ended June 30, 2018, compared to $2.4 million for the same quarter last year. The increase of $1.9 million was driven by increased personnel-related costs due to additional headcount, as well as increased consulting and professional fees.

· Net Loss Attributable to Common Stockholders: Net loss attributable to common stockholders was $22.1 million for the quarter ended June 30, 2018, or $7.84 per basic and diluted share, as compared to a net loss attributable to common stockholders of $12.1 million for the same quarter last year, or $7.55 per basic and diluted share.

Financial Guidance: Based on its current operating plan, Neon expects that its cash, cash equivalents and marketable securities as of June 30, 2018, including the proceeds from its initial public offering, will enable it to fund its operating expenses and capital expenditure requirements into at least the first quarter of 2020.