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Kyowa Kirin Announces FDA Approval of Poteligeo® (mogamulizumab-kpkc) for the Treatment of Mycosis Fungoides and Sézary Syndrome

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/.

Second Quarter and First Half 2018 Financial Results and Business Highlights

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]).

"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.

Bio-Path Holdings to Announce Second Quarter 2018 Financial Results on August 15, 2018

On August 8, 2018 Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that it will host a live conference call and audio webcast on Wednesday, August 15, 2018 at 8:30 a.m. ET to report financial results for the second quarter ended June 30, 2018 and to provide a business overview (Press release, Bio-Path Holdings, AUG 8, 2018, View Source [SID1234528868]).

To access the live conference call, please call (844) 815-4963 (domestic) or (210) 229- 8838 (international) at least five minutes prior to the start time and refer to conference ID 5174289. A live audio webcast of the call will also be available on the Events section of the Company’s website, www.biopathholdings.com. An archived webcast will be available on the Bio-Path website approximately two hours after the event.

Syros to Present at 2018 Wedbush PacGrow Healthcare Conference

On August 8, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the development of medicines that control the expression of genes, reported that its Chief Financial Officer Joseph J. Ferra will present a corporate overview at the 2018 Wedbush PacGrow Healthcare Conference (Press release, Syros Pharmaceuticals, AUG 8, 2018, View Source [SID1234528843]). Details are as follows:

2018 Wedbush PacGrow Healthcare Conference
Date: Wednesday, August 15
Presentation Time: 10:20 a.m. ET
Location: Parker New York 119 West 56th St., New York, NY

A live webcast of the presentation will be available on the Investors & Media section of the Syros website at www.syros.com. An archived replay will be available for approximately 30 days following the presentation.

Scholar Rock Reports Second Quarter 2018 Financial Results and Updates on Business Progress

On August 8, 2018 Scholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the second quarter ended June 30, 2018 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 8, 2018, View Source [SID1234528841]).

"Scholar Rock made transformative progress in the second quarter with the successful completion of our IPO and the initiation of the Company’s first clinical trial," said Nagesh Mahanthappa, Ph.D, President and CEO of Scholar Rock. "We are well-positioned to continue to progress SRK-015 and build our pipeline of future product candidates focused on addressing neuromuscular disorders, cancer, fibrosis, and anemia. We plan to initiate a Phase 2 proof-of-concept study of SRK-015 to improve muscle function in patients with later-onset spinal muscular atrophy (SMA) in the first quarter of 2019."

Key Business and Clinical Highlights

Successfully Completed IPO. In May 2018, Scholar Rock successfully completed an initial public offering (IPO) of 6,164,000 shares of common stock, inclusive of the full exercise of the over-allotment option by the underwriters, raising gross proceeds of approximately $86.3 million.

Initiated Enrollment and Dosing in Multiple-Ascending Dose Portion of Phase 1 Clinical Trial for SRK-015. SRK-015 is a selective inhibitor of the activation of myostatin and was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of SMA in March 2018. A placebo-controlled, double-blind Phase 1 clinical trial was initiated in May 2018 to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of intravenous SRK-015 in healthy adult volunteers. Dosing has completed in the single-ascending dose portion of the study and has advanced to the multiple-ascending dose portion of the study.
Upcoming Milestones

On Track to Initiate Phase 2 Proof-of-Concept Study for SRK-015 in SMA in the First Quarter of 2019. Pending supporting safety data from the Phase 1 clinical trial, Scholar Rock plans to initiate a Phase 2 proof-of-concept study in the first quarter of 2019 to evaluate the safety and efficacy of SRK-015 in patients with later-onset SMA (Type 2 and Type 3) as a monotherapy or in conjunction with an approved survival motor neuron (SMN) upregulator therapy as background standard of care.

Identify Second Indication for SRK-015 in the First Half of 2019. Scholar Rock is actively assessing numerous preclinical models in which the selective inhibition of the activation of myostatin may offer therapeutic benefit. Scholar Rock intends to identify a second indication for SRK-015 in the first half of 2019.

Nominate Product Candidate for TGFβ1 Program by the End of the First Half of 2019. Scholar Rock’s second antibody program is focused on the discovery and development of highly specific inhibitors of the activation of TGFβ1. Scholar Rock is progressing its evaluation of a number of selective inhibitors in multiple disease models and intends to nominate a product candidate and first indication in oncology, immuno-oncology or fibrosis by the end of the first half of 2019.
Second Quarter 2018 Financial Results

Net loss for the quarter ended June 30, 2018 was $14.7 million or $1.39 per share compared to a net loss of $5.9 million or $3.67 per unit for the same quarter last year.

Research and development expense was $11.4 million for the quarter ended June 30, 2018, compared to $4.7 million in the same quarter in 2017. The $6.7 million increase year-over-year was primarily attributable to development and manufacturing costs associated with our lead product candidate, SRK-015, as well as employee costs related to increased headcount.

General and administrative expense was $3.5 million for the quarter ended June 30, 2018, compared to $1.2 million in the same quarter in 2017. The $2.3 million increase year-over-year was mainly due to increased headcount and higher professional and consulting fees associated with ongoing business activities and operating as a public company.
As of June 30, 2018, Scholar Rock had cash, cash equivalents, and marketable securities of $115.1 million, compared to $58.0 million at the end of 2017. The cash balance is inclusive of the approximately $77.8 million in net proceeds from its IPO in May 2018. Scholar Rock believes the cash balance will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2020.