On October 23, 2017 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to MOR208, in combination with lenalidomide, for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation (Press release, MorphoSys, OCT 23, 2017, View Source [SID1234521105]). MOR208 is an investigational Fc-engineered monoclonal antibody directed against CD19 which is currently in clinical development in blood cancer indications.

FDA Breakthrough Therapy designation is intended to expedite development and review of drug candidates, alone or in combination with other drugs. It is granted if preliminary clinical evidence indicates that the drug candidate may demonstrate substantial improvement over existing therapies in the treatment of a serious or life-threatening disease.

DLBCL is the most frequent type of malignant lymphoma worldwide and accounts for approximately 30% of all non-Hodgkin lymphomas. Between 30% and 40% of all patients with DLBCL either fail to respond to or show a relapse to initial therapy.

“DLBCL is a very aggressive lymphoma. In particular, those patients who fail standard treatments are in need of more therapeutic options. We look forward to working closely with the FDA and to develop MOR208 as a potential new treatment option for these patients as quickly as possible,” said Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

FDA’s Breakthrough Therapy designation is based on preliminary data from the ongoing phase 2 L-MIND study (NCT02399085), which is evaluating the safety and efficacy of MOR208 in combination with lenalidomide in patients with R/R DLBCL who are ineligible for high-dose chemotherapy and autologous stem cell transplantation. Preliminary data based on 34 eligible patients presented at ASCO (Free ASCO Whitepaper) 2017, showed an objective response rate (ORR) of 56% and a complete response rate of 32%.

“For MorphoSys, relapsed/refractory DLBCL is a key development focus. We expect to report further data from our ongoing phase 2 L-MIND trial with MOR208 plus lenalidomide at this year’s American Society of Hematology (ASH) (Free ASH Whitepaper) conference in December. In addition, we are currently evaluating MOR208 in combination with bendamustine in our phase 3 B-MIND trial. MorphoSys intends to speed up and potentially broaden the development of MOR208 in other indications of unmet need,” Dr. Peters continued.

About CD19 and MOR208
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 has been reported to enhance B cell receptor (BCR) signaling, which is assumed important for B cell survival, making CD19 a potential target in B cell malignancies.
MOR208 (previously Xmab(R)5574) is an investigational Fc-enhanced monoclonal antibody directed against CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. Furthermore, MOR208 has been observed in preclinical models to induce direct apoptosis by binding to CD19, which is assumed to be a crucial component for B cell receptor (BCR) signaling.
MorphoSys AG is clinically investigating MOR208 as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) was started in March 2016 and is designed to investigate the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed/refractory DLBCL. The phase 2/3 B-MIND study was started in August 2016 and transitioned into its phase 3 pivotal part in June 2017 following a recommendation of the IDMC based on the available data from the phase 2 initial safety evaluation. The B-MIND study is designed to investigate MOR208 in combination with the chemotherapeutic agent bendamustine in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) in comparison to the combination of the anti-CD20 antibody rituximab plus bendamustine. Furthermore, MOR208 is currently being clinically investigated in patients with relapsed/refractory CLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On October 23, 2017 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported that its Board of Directors has selected Aron Knickerbocker to succeed Lewis T. “Rusty” Williams, M.D., Ph.D., as President and CEO of Five Prime effective January 1, 2018. Mr. Knickerbocker is currently Five Prime’s Chief Operating Officer (COO), and will maintain that position until the end of 2017 (Press release, Five Prime Therapeutics, OCT 23, 2017, View Source [SID1234521104]). He has been a member of the Board of Directors since 2013 and will continue to serve his term once CEO. As announced earlier this year, Dr. Williams intends to transition from the position of President, CEO and Chairman of the Board to the role of Executive Chairman of the Board at the beginning of 2018.

Mr. Knickerbocker joined Five Prime in 2009 to lead the company’s business development efforts, eventually serving as Executive Vice President and Chief Business Officer and later COO. He helped the company establish an important cabiralizumab license and collaboration agreement with Bristol-Myers Squibb Company (BMS), a research collaboration with BMS for two immune checkpoint pathways, additional collaborations with GlaxoSmithKline, UCB and Human Genome Sciences, as well as multiple technology in-licensing agreements. During his tenure, Mr. Knickerbocker also has led the company’s Portfolio Management Group, has managed the research teams involved in Five Prime’s collaborations with GSK and UCB, and has been responsible for overseeing investor relations.

“I couldn’t be more pleased to see Aron become the next CEO of Five Prime,” said Dr. Williams. “Aron’s broad executive abilities have been evident since he joined the company and business development is only one of his many strengths. Aron also brings a deep working knowledge of oncology and cancer immunotherapy, which is essential in this role. He has been instrumental in the design and execution of our corporate strategy–whether it be clinical, operational or financial—which contributed to his promotion to COO. Importantly, through our IPO and beyond, he has remained fully committed to communicating and delivering value for our shareholders through effective pipeline growth and the lucrative collaborations we have established with leading pharmaceutical companies. I am confident in the strength of our programs and in the ability of Aron and the rest of our seasoned management team to successfully execute our clinical and corporate strategy.”

Mark D. McDade, Lead Independent Director of the Five Prime Board, commented: “Since Rusty informed us of his intention to transition from the CEO position, the Board has been conducting a comprehensive search for the right CEO candidate to continue advancing and expanding the pipeline and moving Five Prime toward being a commercial stage company. After months of extensive search efforts that considered numerous highly qualified candidates including Aron, it became clear that he stood out due to his oncology experience, strategic thinking and leadership skills, which will be critical as the company grows and advances its pipeline. The Board and I would like to reiterate our immense gratitude to Rusty for his tireless dedication to making the Five Prime vision a reality, with a protein therapeutics platform of unparalleled scope and scale and a robust and growing clinical pipeline. The company is in a very strong position as the torch is passed, and we are fortunate that Aron’s first-hand knowledge and appreciation for Five Prime’s technology, team and shareholders should ensure a smooth leadership transition.”

Prior to Five Prime, Mr. Knickerbocker served at Genentech for eight years in positions of increasing responsibility, including leading the oncology business development team as Senior Director, Business Development. Previously, Mr. Knickerbocker served as Director of Commercial Development at ALZA Corporation (which was acquired by Johnson & Johnson), and in oncology sales, marketing and corporate development roles at Amgen, and as a research scientist at BMS. Mr. Knickerbocker received an A.B. in biology from Washington University in St. Louis and an M.B.A. from the University of Michigan.

“I am honored to be chosen to lead Five Prime as we look to the future and our next stages of growth,” said Mr. Knickerbocker. “I am excited to work with the Board, the management team, and all of our employees as we endeavor to bring promising new protein therapies to cancer patients. The power of our platform and the caliber of our people offer us extraordinary potential in this industry. I will strive to ensure that we build on the momentum of our discovery and development programs and preserve the culture and spirit of innovation that has inspired and motivated us all over the years.”

On October 23, 2017 Amgen (NASDAQ: AMGN) reported top-line results of the Phase 3 A.R.R.O.W. trial, which showed KYPROLIS (carfilzomib) administered once-weekly at the 70 mg/m2 dose with dexamethasone allowed relapsed and refractory multiple myeloma patients to live 3.6 months longer without their disease worsening than KYPROLIS administered twice-weekly at the 27 mg/m2 dose with dexamethasone (Press release, Amgen, OCT 23, 2017, View Source [SID1234521103]). The overall safety profile of the once-weekly KYPROLIS regimen was comparable to that of the twice-weekly regimen.

The study included 478 patients with relapsed and refractory multiple myeloma who received two or three prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent (IMiD). Patients in the trial treated with the once-weekly KYPROLIS regimen achieved a statistically significant superior progression-free survival (PFS) with a median of 11.2 months compared to 7.6 months for those treated with the twice-weekly KYPROLIS regimen (HR = 0.69, 95 percent CI, 0.54 – 0.88).

“KYPROLIS has been demonstrated to be the most effective proteosome inhibitor available to patients with multiple myeloma,” said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. “We are encouraged by the efficacy and safety profile of KYPROLIS and dexamethasone administered once-weekly in the A.R.R.O.W. study.”

The most frequently reported treatment-emergent adverse events (greater than or equal to 20 percent) in either treatment arm were anemia, diarrhea, fatigue, hypertension, insomnia and pyrexia.

On October 23, 2017 Vical Incorporated (Nasdaq:VICL) reported financial results for the three months ended September 30, 2017 (Press release, Vical, OCT 23, 2017, View Source [SID1234521088]). Net loss for the third quarter of 2017 was $3.1 million, or $0.27 per share, compared with a net loss of $2.5 million, or $0.24 per share, for the third quarter of 2016. Revenues for the third quarter of 2017 were $3.2 million, compared with revenues of $2.6 million for the third quarter of 2016, reflecting revenues from Astellas Pharma Inc. for services performed under ASP0113 collaborative agreements.

Vical had cash and investments of $35.2 million at September 30, 2017. The Company’s cash burn for the first nine months of 2017 was $6.9 million, which was consistent with the Company’s full year guidance of between $8 million and $11 million.

Program updates include:

ASP0113 CMV Therapeutic Vaccine

The multinational Phase 3 registration trial in HCT recipients completed enrollment in September 2016 with a total of 515 patients. As recently announced, the last patient completed their final assessment in the one year follow-up period in September 2017. The primary endpoint of the trial is a composite of overall mortality and CMV end organ disease which will be assessed one year after transplantation. Astellas expects top-line data to be available in the first quarter of 2018. Vical and Astellas continue to make progress towards a potential Biologics License Application filing with the U.S. Food and Drug Administration (FDA). Assuming a successful trial outcome, Astellas would seek to commercialize ASP0113 in North America, Europe, and Asia.
VCL-HB01 HSV-2 Therapeutic Vaccine

Vical is developing the HSV-2 therapeutic vaccine, VCL-HB01, to treat patients with symptomatic genital herpes infection. The vaccine is currently being evaluated in a Phase 2 study in healthy adult subjects, 18 to 50 years of age who are randomized 2:1 to receive either vaccine or placebo. Recruitment of 261 subjects at 15 U.S. clinical sites was completed in April 2017 and 4-dose vaccination series was completed in July 2017. Following the 4th vaccination, each subject entered a 12-month surveillance period during which each new lesion recurrence is assessed in the clinic by the investigator. Once all subjects have completed a minimum of 9-months of surveillance, the primary endpoint of annualized recurrence rate will be calculated based on those recurrences that are both clinically- and virologically-confirmed. This endpoint provides important information on the number of recurrences over time in this chronic disease setting and is clinically meaningful for both patients and treating physicians. Vical remains on target to deliver top-line results during the second quarter of 2018.
VL-2397 Antifungal

Vical is developing its novel antifungal, VL-2397, for the treatment of patients with invasive fungal infections. The FDA has advised that VL‑2397 would be eligible for a Limited Use Indication (LUI) approval for the treatment of invasive aspergillosis, assuming a successful outcome of a single Phase 2 trial carried out in accordance with a protocol and statistical analysis plan consistent with the Agency’s advice. The final determination whether the drug is approvable will be made by FDA after review of all relevant data. The Company intends to initiate a Phase 2 trial of VL-2397 for the treatment of invasive aspergillosis in the fourth quarter of 2017. In addition, the FDA has granted Vical Qualified Infectious Disease Product, Orphan Drug and Fast Track designations to VL-2397 for the treatment of invasive aspergillosis.

Vical will conduct a conference call and webcast today, October 23, at noon Eastern Time, to discuss the Company’s financial results and program updates with invited participants. The call and webcast are open on a listen-only basis to any interested parties. To listen to the conference call, dial in approximately ten minutes before the scheduled call to (719)457-2619 (preferred), or (888)349-9582 (toll-free), and reference confirmation code 3889356. A replay of the call will be available for 48 hours beginning about two hours after the call. To listen to the replay, dial (719)457-0820 (preferred) or (888)203-1112 (toll-free) and enter replay passcode 3889356. The webcast will also be available live and archived through the events page at www.vical.com. For further information, contact Vical’s Investor Relations department by phone at (858)646-1127 or by e-mail at [email protected].

On October 23, 2017 Atossa Genetics Inc. (NASDAQ:ATOS), a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods for breast cancer and other breast conditions, reported that it will host a conference call on October 25, 2017 at 10 am EDT to discuss preliminary results from its Phase 1 dose escalation study of its proprietary oral Endoxifen (Press release, Atossa Genetics, OCT 23, 2017, http://ir.atossagenetics.com/news/detail/826/atossa-genetics-to-host-conference-call-to-announce-preliminary-results-from-phase-1-study-of-oral-endoxifen-wednesday-october-25-2017-at-10-am-edt [SID1234521084]). Endoxifen is an active metabolite of the FDA-approved drug tamoxifen, which is currently used to treat breast cancer and for breast cancer prevention in high risk patients.

The objectives of this double-blinded, placebo-controlled, Phase 1 study of 48 healthy female subjects were to assess the pharmacokinetics of proprietary formulations of both oral and topical Endoxifen dosage forms as single (oral) and repeat (oral and topical) doses, as well as to assess safety and tolerability. The study was conducted in two parts based on route of administration. Preliminary results from the topical arm of the study were announced on September 14, 2017.

To listen to the call by phone, interested parties within the U.S. should call 1-844-824-3830 and International callers should call 1-412-317-5140. All callers should ask for the Atossa Genetics conference call. The conference call will also be available through a live webcast at www.atossagenetics.com. Details for the webcast may be found on the Company’s IR events page at View Source

A replay of the call will be available approximately one hour after the end of the call through November 24, 2017. The replay can be accessed via Atossa’s website or by dialing 877-344-7529 (domestic) or 412-317-0088 (international) or Canada Toll Free at 855-669-9658.