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.

On August 8, 2018 FLX Bio, Inc., a clinical-stage biopharmaceutical company focused on the discovery and development of oral small-molecule drugs to activate the immune system, reported that Brian Wong, M.D., Ph.D., CEO will present at the 2018 Wedbush PacGrow Healthcare Conference on August 15, 2018 at 12:45 p.m. ET in New York (Press release, FLX Bio, AUG 8, 2018, View Source [SID1234528786]).

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A live webcast and audio archive of the presentation may be accessed here or on the FLX Bio website at View Source Please connect to the website 10 minutes prior to the presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast.

On August 8, 2018 Kyowa Hakko Kirin Co., Ltd., (Kyowa Kirin) reported that the U.S. Food and Drug Administration (FDA) has granted approval for Poteligeo (mogamulizumab-kpkc) for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy (Press release, Kyowa Hakko Kirin, AUG 8, 2018, View Source [SID1234529006]). FDA granted Priority Review and Breakthrough Therapy Designation in late 2017.

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Poteligeo is a humanized monoclonal antibody (mAb) directed against CC chemokine receptor 4 (CCR4), which is frequently expressed on leukemic cells of certain blood cancers including CTCL. Using the proprietary POTELLIGENT technology, the amount of fucose in the sugar chain structure of Poteligeo is reduced, which enhances the antibody dependent cellular cytotoxicity (ADCC).

"I believe the approval is very good news for patients who have been suffering from mycosis fungoides (MF) or Sézary syndrome (SS) in the US," said Mitsuo Satoh Ph.D., Executive Officer, Vice President Head of R&D Division of Kyowa Hakko Kirin. "Since this antibody was discovered through our cutting-edge R&D activity, it is also another important achievement for Kyowa Hakko Kirin in leaping forward to become a global specialty pharmaceutical company."

"Mycosis fungoides (MF) and Sézary syndrome (SS) can be disfiguring, and debilitating. MAVORIC, the largest study of systemic therapy ever conducted in MF and SS, showed that mogamulizumab prolonged progression-free survival compared to vorinostat in patients with relapsed or refractory MF or SS," said Jeffrey S. Humphrey, MD, President of Kyowa Kirin Pharmaceutical Development, Inc.. "We look forward to the publication of MAVORIC’s primary results and to ongoing scientific exchange within the medical and academic communities."

Because CTCL manifests itself in skin lesions, it is often mistaken for other non-critical skin conditions, which can delay conclusive diagnosis and treatment options. MF and SS are the two most common subtypes of CTCL. MF is the most common subtype, accounting for 50-70% of cases. It is a slow progressing form of lymphoma that can involve the skin, blood, lymph nodes and organs, and may be associated with severe infections. SS accounts for approximately 3% of CTCL cases and is a more aggressive, leukemic form of CTCL.

The FDA approval of Poteligeo is supported by the MAVORIC (Mogamulizumab anti-CCR4 Antibody Versus ComparatOR In CTCL) study, which is the largest randomized trial in MF and SS and the first pivotal trial in CTCL to use PFS as a primary endpoint. MAVORIC was a Phase 3 open-label, multi-center, randomized study of mogamulizumab versus vorinostat in patients with MF and SS who have failed at least one prior systemic treatment. The study was conducted in the U.S., Europe, Japan and Australia, and randomized a total of 372 patients to mogamulizumab or vorinostat. The results showed that mogamulizumab demonstrated significantly superior PFS at a median of 7.6 months [95% CI: 5.6, 10.2] compared to 3.1 months with vorinostat [95% CI: 2.8, 4.0], [hazard ratio 0.53: 95% CI: 0.41, 0.69; p<0.001]. The confirmed overall response rate for mogamulizumab and vorinostat was 28% and 5%, respectively (p<0.001).

FDA granted Poteligeo Breakthrough Therapy Designation for the treatment of MF and SS in adult patients, and evaluated Poteligeo with Priority Review, which is reserved for drugs that treat a serious condition and, if approved, would provide a significant improvement in treatment safety or effectiveness.

Kyowa Kirin International PLC, a Kyowa Hakko Kirin Group company, will be responsible for commercializing Poteligeo in the U.S. and this is planned to commence in the fourth quarter of 2018. A Marketing Authorization application for mogamulizumab is currently under review by the European Medicines Agency.

The Kyowa Hakko Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

Please see Poteligeo indication and Important Safety Information below.

INDICATION
POTELIGEO (mogamulizumab-kpkc) injection for intravenous infusion is indicated for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy.
Important Safety Information
Warnings and Precautions:

Dermatologic toxicity: Monitor patients for rash throughout the course of treatment. For patients who experienced dermatologic toxicity in the pivotal trial the median time to onset was 15 weeks, with 25% of cases occurring after 31 weeks. Interrupt POTELIGEO for moderate or severe rash (Grades 2 or 3). Permanently discontinue POTELIGEO for life-threatening (Grade 4) rash or for any Stevens-Johnson syndrome (SJS) or toxic epidermal necrolysis (TEN).
Infusion reactions: Most infusion reactions occur during or shortly after the first infusion. Infusion reactions can also occur with subsequent infusions. Monitor patients closely for signs and symptoms of infusion reactions and interrupt the infusion for any grade reaction and treat promptly. Permanently discontinue POTELIGEO for any life-threatening (Grade 4) infusion reaction.
Infections: Monitor patients for signs and symptoms of infection and treat promptly.
Autoimmune complications: Interrupt or permanently discontinue POTELIGEO as appropriate for suspected immune-mediated adverse reactions. Consider the benefit/risk of POTELIGEO in patients with a history of autoimmune disease.
Complications of allogeneic HSCT after POTELIGEO: Increased risks of transplant complications have been reported in patients who received allogeneic HSCT after POTELIGEO. Follow patients closely for early evidence of transplant-related complications.
Adverse Reactions:

The most common adverse reactions (reported in ≥ 10% of patients) with POTELIGEO in the clinical trial were rash, including drug eruption (35%), infusion reaction (33%), fatigue (31%), diarrhea (28%), drug eruption (24%), upper respiratory tract infection (22%), musculoskeletal pain (22%), skin infection (19%), pyrexia (17%), edema (16%), nausea (16%), headache (14%), thrombocytopenia (14%), constipation (13%), anemia (12%), mucositis (12%), cough (11%), and hypertension (10%).
You are encouraged to report suspected adverse reactions to Kyowa Kirin, Inc. at 1-844-768-3544 or FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch/.