On August 8, 2018 INSYS Therapeutics, Inc. (NASDAQ: INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids (CBD) and spray technology, reported financial results for its second quarter ended June 30, 2018 (Press release, Insys Therapeutics, AUG 8, 2018, View Source;p=RssLanding&cat=news&id=2362910 [SID1234528760]).

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OVERALL HIGHLIGHTS

Reached agreement in principle to settle Department of Justice investigation with financial terms that are consistent with previous public statements and disclosures.
Achieved gross revenue of $38.0 million and net revenue of $23.5 million in the second quarter.
Continued to advance prioritized R&D programs with $16.5 million investment in the second quarter.
Reported encouraging results from pharmacokinetics (PK) study of epinephrine nasal spray as potential product candidate for treatment of anaphylaxis.
Commenced enrolling Phase 2 clinical trial of cannabidiol (CBD) oral solution as potential treatment for Prader-Willi syndrome.
Initiated Phase 3 clinical trial of CBD oral solution as potential treatment for infantile spasms.
Continued enrolling Phase 2 clinical trial of CBD oral solution as potential treatment for childhood absence epilepsy.
Established collaboration partnership with University of California San Diego’s Center for Medicinal Cannabis Research to study CBD oral solution in various disease states, starting with autism.
Received Complete Response Letter from FDA regarding buprenorphine New Drug Application (NDA).
Announced exclusive licensing partnership with Lunatus to commercialize SUBSYS in the Middle East.
"Our continuing commitment to the potential of CBD and our nasal spray technology to significantly improve the lives of patients was highlighted by several important milestones in the second quarter of 2018, as we continue to make progress against our strategic plan," said Saeed Motahari, president and chief executive officer of INSYS Therapeutics. "We received encouraging results from our pharmacokinetics study of epinephrine nasal spray and initiated a Phase 2 study of CBD for Prader-Willi Syndrome. Furthermore, we believe we remain on track to submit an NDA for naloxone nasal spray by the end of 2018. These critical milestones are in keeping with our long-term goal to submit one NDA per year through 2021."

Motahari continued, "Prescriptions for our primary commercial product, SUBSYS, declined at a slower rate than the overall TIRF market in the second quarter. Our commercial efforts are showing signs of traction, as we gained prescription share in the TIRF market sequentially for the first time in seven quarters. These efforts include new managed care wins, patient education, upgrading the salesforce talent and optimizing territory alignment."

Motahari added, "Albeit off a small base, prescriptions of SYNDROS experienced a solid improvement in the second quarter as net revenue improved 56 percent sequentially, driven by the initial success of our patient access and educational programs. We remain resolute in our commitment to patients who rely on SYNDROS and SUBSYS and believe our product pipeline has the potential to significantly improve the lives of patients with unmet medical needs—particularly our two life-saving drug candidates, epinephrine and naloxone nasal sprays."

Financial & Operating Highlights

Gross revenue for the second quarter of 2018 of $38.0 million, compared to $58.2 million for the second quarter of 2017 driven primarily by declines in the TIRF market, but slightly offset by market share gains in the second quarter of 2018.
Net revenue for the second quarter of 2018 was $23.5 million, compared to $42.6 million for the second quarter of 2017, as a result of lower gross revenue and returns of expired product.
Gross margin was 84.7 percent for the second quarter of 2018, compared to 90.8 percent in the same period of 2017.
Sales and marketing investment was $9.1 million for the second quarter of 2018, compared to $13.3 million for the second quarter of 2017.
Research and development investment increased to $16.5 million for the second quarter of 2018, compared to $14.1 million for the second quarter of 2017.
General and administrative expense of $10.9 million for the second quarter of 2018, compared to $10.6 million in the second quarter of 2017.
Legal expense increased to $11.1 million for the second quarter of 2018, compared to $6.5 million in the second quarter of 2017.
Income tax expense was $0.1 million for the second quarter of 2018 compared to a benefit of $1.7 million during the second quarter of 2017.
Net loss for the second quarter of 2018 was ($27.4 million), or ($0.37) per basic and diluted share, compared to a net loss of ($8.2 million), or ($0.11) per basic and diluted share, for the second quarter of 2017. Adjusted net loss for the quarter was ($0.33) per basic and diluted share.
Adjusted EBITDA loss for the second quarter of 2018 was ($22.5 million), compared to Adjusted EBITDA of $0.3 million in the prior-year quarter. The reconciliation of net income to Adjusted EBITDA is included at the end of this news release.
The Company had $123.5 million in cash, cash equivalents and short-term and long-term investments with no debt as of June 30, 2018.
Webcast Information

A conference call is scheduled for 5:00 p.m. Eastern Standard Time on Aug. 8, 2018, to discuss the financial and operational results for the second quarter 2018. Interested parties can listen to the call live as it occurs via the company’s website, View Source, on the Investors section’s Presentations & Events page; or by dialing 844-263-8304 (from inside the U.S.) or 213-358-0958 (from outside the U.S.), and using the Conference ID 2070039. A webcasted replay of the call will be available on the site a few hours after the event.

On August 8, 2018 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), a clinical-stage biopharmaceutical firm engaged in the development of immunotherapies for cancer and stem cell therapies for degenerative diseases, reported financial results and business highlights for the second quarter and six months ended June 30, 2018 (Press release, Cellular Biomedicine Group, AUG 8, 2018, View Source [SID1234528976]).

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"The acceptance of CBMG’s IND application for "C-CAR011" anti-CD19 chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with B-cell Non-Hodgkin’s lymphoma (NHL) and acute lymphoblastic leukemia (ALL) reinforces the strength of our immuno-oncology platform. We look forward to working with the CFDA to obtain approval to move to the next phase of development," commented Tony (Bizuo) Liu, Chief Executive Officer of CBMG. "We continue to deploy our working capital to pursue and develop a robust non-CD19 pipeline targeting other liquid and solid tumors. We are also advancing our quality systems and automated manufacturing capabilities by utilizing digital technologies with the goal of becoming a premier international biopharma company and a preferred collaborator for cell therapy development in China. With the expansion and relocation of our U.S. R&D center to Gaithersburg, Maryland, we are committed to leverage our talented team to develop the latest technology in cancer cell therapy. Being in the heart of this renowned research hub presents us with opportunities to collaborate with leading experts in this ecosystem to bridge new therapies developed in the U.S. into our clinical development in China, ultimately leading to serve the China market."

Second Quarter and First Half 2018 Financial Performance

G&A Expenses: General and administrative expenses remain relatively flat for the six months ended June 30, 2018 compared to the same period in 2017 due to the efficient management and utilization of resources. General and administrative expenses for the quarter and six months ended June 30, 2018 were $3.1 million and $6.3 million, respectively, compared to $3.3 million and $6.4 million for the same periods in 2017.
R&D Expenses: Research and development expenses grew substantially for the six months ended June 30, 2018 compared to the same period in 2017 due to the expanded commitment to research and development, process improvement and anticipated clinical activities. Research and development expenses for the quarter and six months ended June 30, 2018 were $6.2 million and $11.4 million respectively, compared to $3.3 million and $6.4 million for the same periods in 2017.
Net Loss: Net loss allocable to common stock holders for the quarter and six months ended June 30, 2018 was $9.2 million and $17.7 million respectively, compared to $6.2 million and $12.4 million for the same periods in 2017.
Current Immuno-Oncology Pipeline of Targeted Indications and Potential Therapies

io pipeline july 2018

Business & Technology Highlights First Half 2018

MOVED TO NEW R&D CENTER IN GAITHERSBURG: In May 2018, the Company moved its Maryland lab to a larger research and development center in Gaithersburg to accelerate the Company’s robust oncology research pipeline, to attract new recruits and to work closely with potential collaborating partners;
SUBMITTED IND APPLICATIONS TO CFDA: In April 2018, the CFDA accepted the IND applications for anti-CD19 CAR-T therapy "C-CAR011" targeting NHL and ALL and the Company is working with the CFDA for approval to move to the next phase of development;
PUBLISHED KOA DATA: In March 2018, the Company presented its allogeneic adipose-derived mesenchymal progenitor cell off-the-shelf therapy AlloJoinTM for Knee Osteoarthritis (KOA) 48-week clinical data from the Phase I clinical trial in China, which demonstrated good safety and early efficacy for the prevention of cartilage deterioration;
OBTAINED OPTION TO LICENSE PATENT ON AFP TCR-T for HEPATOCELLULAR CARCINOMA: In February 2018, CBMG’s wholly-owned subsidiary entered into an agreement with Augusta University to take a three-year option to license technology for Alpha fetoprotein (AFP) T Cell Receptor (TCR), targeting Hepatocellular Carcinoma (HCC) (patent pending);
COMPLETED PRIVATE EQUITY FINANCING: In February 2018, Sailing Capital invested $30.6 million in CBMG. Sailing Capital is a global private equity firm focused on disruptive technologies from innovative global companies in the healthcare, technology and consumer sectors.

On August 8, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the six months ended June 30, 2018 (Press release, Trillium Therapeutics, AUG 8, 2018, View Source [SID1234528539]).

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"We are continuing to build upon the observed single-agent activity of TTI-621, our lead CD47 blocking agent, in T cell lymphoma patients, and are also now dosing patients with TTI-622, our second SIRPaFc decoy receptor," said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "With two unique CD47-directed therapies in clinical testing, and strong support and enthusiasm from our clinical collaborators, we continue our role as a leader in the field of CD47 research."

2018 Second Quarter Highlights:

Based on clinical observations in the TTI-621 intravenous trial, the company has refined its monotherapy efforts to focus on T cell malignancies, specifically T cell lymphomas. Combination cohorts with rituximab and nivolumab continue to enroll. Patients with cutaneous T cell lymphoma and Sezary syndrome are being enrolled in the expansion phase of the intratumoral trial, receiving continued weekly injections. In both trials, new clinical investigators and sites with experience in the treatment of T cell lymphoma are being added.
The company initiated dosing in its two-part, multicenter, open-label, phase 1a/1b clinical trial of TTI-622 (SIRPaFc-IgG4), a checkpoint inhibitor of the innate immune system, in relapsed or refractory lymphoma or myeloma. In the phase 1a dose-escalation part, patients will be enrolled in sequential dose cohorts to receive TTI-622 once weekly to characterize safety, tolerability, pharmacokinetics, and to determine the maximum tolerated dose. In the phase 1b part, patients will be treated with TTI-622 in combination with rituximab, a proteasome inhibitor-containing regimen, or a PD-1 inhibitor.
Yaping Shou MD, PhD, joined Trillium as Chief Medical Officer. Dr. Shou has more than 18 years of industry experience spanning clinical development and translational medicine, with a strong focus in oncology. She most recently served as Executive Medical Director at Takeda Pharmaceuticals, where she also held several other clinical leadership positions over the past seven years.
Trillium entered into a Second Amended and Restated License Agreement with the licensors of the SIRPaFc technology and removed the sublicense revenue sharing provisions in return for a payment to the Licensors of $3.0 million in the form of 369,621 common shares. Trillium believes the amendments to the agreement provide the company with greater financial potential and flexibility in any future partnership discussions.
Second Quarter 2018 Financial Results:

As of June 30, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital of $64.7 million and $53.4 million, respectively, compared to $81.8 million and $68.9 million, respectively at December 31, 2017. The decrease in cash and cash equivalents and marketable securities was due mainly to cash used in operations of $20.0 million, net of an unrealized foreign exchange gain of $3.0 million. The decrease in working capital was due mainly to cash used in operations, an increase to prepaid expenses, and a decrease to accounts payable and accrued liabilities due to clinical trial payments.

Net loss for the six months ended June 30, 2018 of $20.9 million was lower than the loss of $23.1 million for the six months ended June 30, 2017. The net loss was lower due mainly to a net foreign currency gain of $3.0 million for the six months ended June 30, 2018, compared to a net foreign currency loss of $2.6 million in the prior year period, and lower manufacturing costs, partially offset by higher clinical trial expenses and the expense relating to the amendment of the SIRPaFc license agreement.

On August 8, 2018 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of bone marrow transplant to more patients, reported that the Company is scheduled to present at the 2018 Wedbush PacGrow Healthcare Conference on Tuesday, August 14th, at 9:10 a.m. ET (Press release, Magenta Therapeutics, AUG 8, 2018, View Source [SID1234528623]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors and Media section of the company’s website at www.magentatx.com. A replay of the webcast will be archived on the Magenta website for 30 days following the presentation.

On August 8, 2018 La Jolla Pharmaceutical Company (Nasdaq: LJPC), a leader in the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported financial results for the three and six months ended June 30, 2018 and highlighted recent corporate progress (Press release, La Jolla Pharmaceutical, AUG 8, 2018, View Source [SID1234528761]).

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Recent Corporate Progress

In August 2018, La Jolla announced that the Centers for Medicare & Medicaid Services (CMS) had granted a New Technology Add-on Payment (NTAP) for GIAPREZATM (angiotensin II) Injection for Intravenous Infusion. The NTAP program provides additional reimbursement to hospitals beyond the Medicare Severity Diagnosis-Related Group (MS-DRG) reimbursement for specific products that meet strict criteria for the treatment of Medicare patients. The amount of the NTAP is equal to 50% of the amount by which the covered costs exceed the MS-DRG reimbursement, or 50% of the cost of the drug, whichever is less. The NTAP for GIAPREZA is effective for the CMS 2019 fiscal year, which begins on October 1, 2018, and is expected to continue for a period of up to two or three years, after which the MS-DRG payments will be adjusted based on hospital-reported costs and utilization. The NTAP program is only available to new drugs that represent an advance in medical technology that substantially improves, relative to technologies previously available, the treatment of Medicare patients.

In June 2018, La Jolla announced that the Marketing Authorization Application (MAA) for GIAPREZA was validated by the European Medicines Agency (EMA). Validation of the MAA confirms that the submission is complete and starts the EMA’s centralized review process. La Jolla submitted the GIAPREZA MAA for the treatment of hypotension in adults with distributive or vasodilatory shock who remain hypotensive despite fluid and vasopressor therapy. The MAA is based on data from the ATHOS-3 Phase 3 study, which establishes the safety and efficacy of GIAPREZA in the proposed indication. If approved, GIAPREZA could be available for marketing in the European Union in the second half of 2019.

In June 2018, two presentations on LJPC-401 (synthetic human hepcidin) were given at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). The first was an oral presentation, entitled "A Phase 1, Open-Label Study to Determine the Safety, Tolerability, and Pharmacokinetics of Escalating Doses of LJPC-401 (Synthetic Human Hepcidin) in Patients with Iron Overload." The second was a poster presentation, entitled "A Phase 1, Placebo-Controlled Study to Determine the Safety, Tolerability, and Pharmacokinetics of Escalating Subcutaneous Doses of LJPC-401 (Synthetic Human Hepcidin) in Healthy Adults."

"Since GIAPREZA’s launch in March, more than 200 hospitals have approved GIAPREZA for inclusion on their formularies," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "This is an important first step in GIAPREZA’s commercial roll-out, as it allows physicians and pharmacists to begin integrating GIAPREZA into their hospital systems and lays the groundwork for potential routine use in their practice."

Results of Operations

For the three and six months ended June 30, 2018, La Jolla recognized GIAPREZA net product sales of $1.6 million and $2.4 million, respectively. La Jolla launched GIAPREZA in March 2018. La Jolla’s net loss for the three and six months ended June 30, 2018 was $52.8 million and $103.3 million, or $2.02 per share and $4.22 per share, respectively, compared to $26.7 million and $50.0 million, or $1.21 per share and $2.46 per share, respectively, for the same periods in 2017.

As of June 30, 2018, La Jolla had $241.4 million in cash and cash equivalents, compared to $90.9 million as of December 31, 2017. Cash used for operating activities for the six months ended June 30, 2018 was $83.4 million, compared to $41.2 million for the same period in 2017.

Conference Call Details

The Company will host a conference call and webcast today, August 8, 2018, at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time). The conference call can be accessed by dialing 877-359-9508 for domestic callers and 224-357-2393 for international callers. Please provide the operator with the conference ID number 2692098 to join the conference call or click here for the webcast. An archive of the conference call and webcast will be available on La Jolla’s website for 30 days following the call.

About Shock and Septic or Other Distributive Shock

Over 1 million Americans are affected by shock on an annual basis, with 1 in 3 patients being treated for shock in the intensive care unit. Distributive shock is the most common type of shock in the inpatient setting with approximately 800,000 distributive shock cases in the United States per year. Of these cases, an estimated 90% are septic shock patients. Approximately 300,000 do not achieve adequate blood pressure response with standard of care vasopressor therapy (catecholamines and vasopressin). The inability to achieve or maintain adequate blood pressure results in inadequate blood flow to the body’s organs and tissue and is associated with a mortality rate exceeding most acute conditions requiring hospitalization. In the European Union, the annual incidence of sepsis in adults is estimated to be more than 500,000, with more than 170,000 progressing to septic shock.

About GIAPREZA

In December 2017, GIAPREZA (angiotensin II) was approved by the U.S. Food and Drug Administration (FDA) as a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock. GIAPREZA mimics the body’s endogenous regulatory peptide that is central to the renin-angiotensin-aldosterone system to increase blood pressure. Prescribing information for GIAPREZA is available at www.giapreza.com. GIAPREZA is marketed by La Jolla Pharmaceutical Company on behalf of La Jolla Pharma, LLC, its wholly owned subsidiary.

IMPORTANT SAFETY INFORMATION

Contraindications

None

Warnings and Precautions

There is a potential for venous and arterial thrombotic and thromboembolic events in patients who receive GIAPREZA. Use concurrent venous thromboembolism (VTE) prophylaxis.

Adverse Reactions

The most common adverse reactions that were reported in greater than 10% of GIAPREZA-treated patients were thromboembolic events.

Drug Interactions

Angiotensin converting enzyme (ACE) inhibitors may increase response to GIAPREZA. Angiotensin II receptor blockers (ARB) may reduce response to GIAPREZA.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.