On November 6, 2019 NewLink Genetics Corporation (NASDAQ:NLNK) reported financial results for the third quarter ended September 30, 2019 and provided an update on corporate activities (Press release, NewLink Genetics, NOV 6, 2019, View Source [SID1234550399]).

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"We are excited about the proposed merger with Lumos Pharma that we announced at the close of the third quarter and continue to advance the process required to close the transaction," commented Brad Powers, General Counsel and member of NewLink’s Office of the CEO. "We believe this merger positions the company to improve therapeutic options for patients with pediatric growth hormone deficiency and other rare and neglected diseases, and to bring greater value to our shareholders."

Eugene Kennedy, MD, Chief Medical Officer and member of NewLink’s Office of the CEO added, "We are also pleased by the FDA’s recent acceptance of the biologic licensing application for priority review of our partnered investigational Ebola vaccine, which we licensed to Merck in 2014. We are encouraged, as well, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending a conditional marketing authorization for V920 Ebola Zaire vaccine. We believe these actions demonstrate the urgency with which regulatory bodies are addressing this deadly disease in order to help the affected individuals and communities."

Proposed Merger and Related Milestones

On September 30, 2019, NewLink announced its intent to merge with Lumos Pharma, a private clinical-stage biopharmaceutical company targeting rare and neglected diseases. Under the terms of the merger agreement, Lumos and NewLink stockholders will each own approximately 50% of the combined company which will be renamed "Lumos Pharma" at the close of the transaction. Rick Hawkins, current CEO of Lumos Pharma, is expected to become CEO of the combined company. The proposed merger has been approved by the Boards of both companies and the major stockholders of Lumos Pharma and NewLink’s largest stockholder has entered into a support agreement with NewLink to vote in favor of various proposals relating to the Merger.

The combined company would focus initially on the development of Lumos Pharma’s lead product candidate, LUM-201 (ibutamoren), an oral growth hormone (GH) secretagogue targeting pediatric growth hormone deficiency (PGHD) and other rare endocrine disorders. If approved, LUM-201 has the potential to be the first orally administered growth hormone stimulating therapy for PGHD, a well-established and sizable market where daily recombinant human growth hormone injections represent current standard-of-care therapy.

The initiation of a Phase 2b trial for LUM-201 in a subset of PGHD patients meeting certain predictive enrichment markers (PEMs) is anticipated in mid-2020. The combined company is expected to have resources sufficient to support clinical development through the readout of this planned Phase 2b trial. Additional target indications are being evaluated for LUM-201 clinical development, including Turner Syndrome and children born Small for Gestational Age (SGA).

NewLink will file a proxy statement with the Securities and Exchange Commission (SEC) providing additional information about the proposed merger. The transaction is expected to close in the first quarter of 2020, subject to the satisfaction of customary closing conditions, including approval of certain proposals by the stockholders of NewLink.

Additional Updates

The combined company may receive additional, non-dilutive financing should the U.S. Food and Drug Administration (FDA) approve NewLink’s Ebola vaccine V920 partnered with Merck Sharp & Dohme Corp. (Merck). On September 17, 2019, Merck announced that the FDA has accepted its biologics license application (BLA) and granted priority review for the investigational Ebola vaccine V920 and the Prescription Drug User Fee Act (PDUFA), or target action date, is set for March 14, 2020. If the vaccine receives approval by the FDA, a priority review voucher will be issued, in which the Company owns a substantial interest and which the Company plans to monetize.

On October 18, 2019, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use(CHMP) adopted a positive opinion recommending a conditional marketing authorization for investigational V920 Ebola Zaire vaccine (rVSV∆G-ZEBOV-GP), as confirmed by our partner, Merck (NYSE:MRK). This Committee’s recommendation will now be reviewed by the European Commission (EC) for a centralized marketing authorization of the vaccine (brand name ERVEBO) across EU member countries.

Updated Phase 1b results for indoximod for the cohort of pediatric patients with newly diagnosed treatment-naïve diffuse intrinsic pontine glioma (DIPG) has been accepted for presentation at the upcoming ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress 2019, 11-14 December 2019, Geneva, Switzerland. NewLink expects to continue to evaluate its oncology portfolio to determine value creation opportunities.

Financial Results for the Three-Month Period Ended September 30, 2019

Restructuring: On September 30, 2019, NewLink Genetics announced it had adopted a restructuring plan to reduce its headcount by approximately 60% to align with future priorities and to conserve resources. In conjunction with the restructuring and the departures of our former CEO and President, the company recorded restructuring and severance charges of $4.5 million during the third quarter.

Cash Position: NewLink Genetics ended the quarter on September 30, 2019, with cash and cash equivalents totaling $98.5 million compared to $120.7 millionDecember 31, 2018.

R&D Expenses: Research and development expenses for the third quarter of 2019 were $7.0 million, a decrease of $546,000 from $7.6 million for the same period in 2018. The decrease was primarily due to reductions of $1.1 million in personnel-related and stock compensation expense, $255,000 in contract research and manufacturing spend, and $333,000 in legal and consulting and supplies expense, offset by increases of $1.1 million in restructuring costs and $87,000 in clinical trial and licensing expense.

G&A Expenses: General and administrative expenses in the third quarter of 2019 were $8.3 million, an increase of $691,000 from $7.6 million for the same period in 2018. The increase was due primarily to increases of $2.0 million in restructuring and severance expense, and $1.2 million in legal and consulting expense, offset by decreases of $1.7 million in stock compensation expense, $431,000 in personnel-related expense, and $353,000 in supplies.

Net Loss: NewLink Genetics reported a net loss of $14.5 million or ($0.39) per diluted share for the third quarter of 2019 compared to a net loss of $7.4 million or ($0.20) per diluted share for the third quarter of 2018.

NewLink Genetics ended Q3 2019 with 37,314,076 shares outstanding.

Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss its financial results and to give an update on clinical and business development activities.

Access to the live conference call is available five minutes prior to the start of the call by dialing (855) 469-0612 (U.S.) or (484) 756-4268 (international). The conference call will be webcast live and a link to the webcast can be accessed through the NewLink Genetics website at www.NewLinkGenetics.com in the "Investors & Media" section under "Events and Presentations" or through the link View Source To ensure a timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and using the passcode 3794399. The replay will be available for two weeks from the date of the call.

On November 6, 2019 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH; Nasdaq: IPHA) reported that it will provide an update on its preclinical pipeline at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2019 Congress in National Harbor, Maryland, November 6-10, 2019 (Press release, Innate Pharma, NOV 6, 2019, View Source [SID1234550398]). Highlights will include:

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In vivo data on anti-CD39 IPH5201 supporting the rationale for assessing combination with PD-1/PD-L1 checkpoint inhibitors
Preclinical results of anti-CD73 IPH5301 targeting the adenosine immunosuppressive pathway
New generation trifunctional NK cell engagers (NKCEs), supporting its clinical development in immunotherapy
Innate will also present in vitro data on IPH5401, demonstrating that the molecule has the potential to block activation and migration of human neutrophils, a major mechanism to allow for the reactivation of effector cells. The Company believes this data supports its ongoing multi-center, open label, dose-escalation and dose expansion Phase I clinical trial (STELLAR-001), evaluating the safety and efficacy of IPH5401 in combination with durvalumab (anti-PD-L1) in patients with advanced solid tumors.

Eric Vivier, Chief Scientific Officer of Innate Pharma, will also give a presentation on harnessing innate imnunity in cancer, as part of the faculty session dedicated to NK cells.

"We are pleased to present data at SITC (Free SITC Whitepaper) 2019 on several of our preclinical and clinical assets, notably three antibodies targeting the tumor microenvironment, as well as on our new NKCEs technology," commented Eric Vivier, Chief Scientific Officer of Innate Pharma. "These data support the science and innovative technologies our scientists continue to develop, helping to further advance our multi-target strategy in immuno-oncology."

Oral presentation:

– Concurrent Session 312: NK Cells: From Basic Science to Clinical Application "Basic Science" presented by Eric Vivier, DVM, PhD, Innate Pharma
Saturday, November 9 | 5:15 pm

Poster presentations:

– IPH5301, a CD73 blocking antibody targeting the adenosine immunosuppressive pathway for cancer immunotherapy, Poster P323
Friday November 8 | 12:30 – 2 pm & 6:30 – 8 pm

– IPH5201, a blocking antibody targeting the CD39 immunosuppressive pathway, unleashes immune responses in combination with cancer therapies, Poster P488
Saturday November 9 | 12:35 – 2:05 pm & 7 – 8:30 pm

– IPH5401 anti-human C5aR antibody targets suppressive myeloid cells in the TME, Poster P268
Saturday November 9 | 12:35 – 2:05 pm & 7 – 8:30 pm

– Multifunctional natural killer cell engagers targeting NKp46 trigger protective tumor immunity, Poster P776
Saturday November 9 | 12:35 – 2:05 pm & 7 – 8:30 pm

On November 05, 2019 Allogene Therapeutics and Notch Therapeutics reported an exclusive worldwide collaboration and license agreement to research and develop induced pluripotent stem cell (iPSC) AlloCAR therapy products for initial application in non-Hodgkin lymphoma, leukemia and multiple myeloma (Press release, Allogene, NOV 5, 2019, View Source [SID1234618291]). Under the partnership, Allogene and Notch will create allogeneic cell therapy candidates from T cells or natural killer (NK) cells using Notch’s Engineered Thymic Niche (ETN) platform.

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Notch was established in 2018 by Juan Carlos Zúñiga-Pflücker, Ph.D. and Peter Zandstra, Ph.D., recognized pioneers in iPSC and T cell differentiation technology. Notch is developing a next-generation approach to differentiating mature immune cells from iPSCs. The Notch ETN technology platform offers potential flexibility and scalability for the production of stem cell-derived immune cell therapies. iPSCs may provide renewable starting material for AlloCAR T therapies that could allow for improved efficiency of gene editing, greater scalability of supply, product homogeneity and more streamlined manufacturing.

"This collaboration exemplifies Allogene’s long-term commitment to advancing the field of cancer treatment as we continue to expand and progress our innovative pipeline of off-the-shelf AlloCAR candidates," said David Chang, M.D., Ph.D., President, CEO and Co-Founder of Allogene Therapeutics. "The scientific founders of Notch Therapeutics are among the most respected experts in the field of stem cell biology and its applications to generating T cells and other functional immune cells. We are confident that their technology and expertise, combined with Allogene’s leadership in AlloCAR therapies, has the potential to unlock future generations of cell therapy treatments for patients."

"Renewable-source, off-the-shelf cell therapies that may produce cells with greater consistency and at industrial scale have long been the dream for people working in this field," said Ulrik Nielsen, Ph.D., Executive Chairman of Notch. "We are delighted to spring into the research collaboration for iPSC-based AlloCAR therapies with Allogene, a leader in the allogeneic CAR T field, with the goal of expanding options for patients."

Under the terms of the agreement, Notch will be responsible for preclinical research of next-generation iPSC AlloCAR T cells. Allogene will clinically develop the product candidates and holds exclusive worldwide rights to commercialize resulting products. Allogene will provide to Notch an upfront payment of $10 million. Notch will be eligible to receive up to $7.25 million upon achieving certain agreed research milestones, up to $4.0 million per exclusive target upon achieving certain pre-clinical development milestones, and up to $283 million per exclusive target and cell type upon achieving certain clinical, regulatory and commercial milestones as well as tiered royalties on net sales in the mid to high single digits. In addition to this collaboration and license agreement, Allogene has acquired a 25 percent equity position in Notch and will assume a seat on Notch’s Board of Directors.

"Master cell banks of genetically modified, induced pluripotent stem cells could provide an inexhaustible source of cell therapies that may improve outcomes and expand applicability to new areas," said Notch Co-Founder Juan Carlos Zúñiga-Pflücker, Ph.D., a senior scientist at Sunnybrook Research Institute and a Professor and Chair of the Department of Immunology at the University of Toronto.

"This work with Allogene may also pave the way for additional off-the-shelf cell therapeutics that are custom-designed to treat other immunity-related diseases such as infectious diseases, autoimmune diseases and aging," said Notch Co-Founder and Chief Scientific Officer Peter Zandstra, Ph.D., a Professor at the University of British Columbia and University of Toronto.

On November 5, 2019 eFFECTOR Therapeutics, Inc., a leader in the development of selective translation regulators (STRs) for the treatment of cancer, reported the dosing of the first subject in the phase 1/2 study of zotatifin (eFT226) being developed for advanced solid tumor malignancies (Press release, eFFECTOR Therapeutics, NOV 5, 2019, View Source [SID1234553137]). Zotatifin is a novel, potent and selective small molecule inhibitor of eukaryotic initiation factor 4A (eIF4A).

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The study will enroll patients with activating mutations, amplifications or fusions in HER2, ERBB3, FGFR1, or FGFR2 receptor tyrosine kinases, or any KRAS mutation subtype. It will also include pancreatic adenocarcinoma with no molecular typing since the large majority of those patients harbor a KRAS mutation.

"There is an immediate need for more effective treatment options in patients with advanced cancers unable to respond to alternative therapies," said Steve Worland, Ph.D., president and chief executive officer of eFFECTOR. "Tumors treated with agents targeting specific RTKs frequently become resistant, and there are no available therapies targeting multiple KRAS mutation subtypes. The antitumor response observed in preclinical studies demonstrates the potential for zotatifin in the treatment of solid tumors with genetic modifications associated with aggressive disease, including RTK alterations such as FGFR1/2 and HER2 and KRAS, and provides direction for patient selection in our clinical trial."

In the open label, dose escalation and cohort expansion study, subjects will be assigned sequentially to increasing zotatifin doses until the maximum tolerated dose is reached. Zotatifin will be administered as a monotherapy in weekly intravenous infusions in subjects with advanced solid tumor malignancies. Treatment and study subject evaluations will be performed in 21-day cycles.

The primary endpoints of the study include safety and tolerability of zotatifin as monotherapy. Secondary endpoints include antitumor activity and survival, as well as pharmacokinetics of the drug. Exploratory endpoints include pharmacodynamics of zotatifin.

Scientists from eFFECTOR recently presented preclinical data at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) – also known as the Triple Meeting. The data demonstrated that solid tumor cell lines driven by alterations in FGFR1, FGFR2 and HER2 treated with zotatifin showed decreased MAPK and AKT signaling, potent inhibition of cell proliferation and apoptosis and strong in vivo anti-tumor activity, indicating potential for zotatifin to treat FGFR1/2 or HER2-driven cancers.

About Zotatifin (eFT226)

Zotatifin (eFT226) is a potent and selective inhibitor of eukaryotic translation initiation factor 4A (eIF4A). eIF4A is located downstream from key oncogenic mutations and their common resistance mechanisms. Zotatifin inhibits the translation of mRNA encoding several important oncogenes and survival factors, including several RTKS, KRAS, Cyclin D, MCL1 and BCL-2 resulting in potent in vivo efficacy in multiple tumor models dependent on these factors, including colorectal cancer, non-small cell lung cancer, breast cancer, hepatocellular carcinoma and B cell lymphomas. Since Zotatifin inhibits the translation of mRNA encoding KRAS and RTK, it is not limited to any mutation subtypes. The drug is currently being evaluated in a Phase 1/2 clinical trial in patients with solid tumors.

On November 5, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, reported that the United States Food and Drug Administration (FDA) has granted AUTO1 orphan drug designation for treatment of acute lymphoblastic leukemia (ALL) patients (Press release, Autolus, NOV 5, 2019, View Source [SID1234550683]).

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According to the National Institute of Health’s National Cancer Institute, in the United States, there will be an estimated 5,930 new cases of ALL and an estimated 1,500 related deaths in 2019. Patients are predominantly children; approximately 60% of cases occur at age < 20 years. ALL occurs when the bone marrow makes too many immature lymphocytes, which are a type of white blood cell. Despite a high rate of response to induction chemotherapy, only 30–40% of adult patients with ALL will achieve long-term remission. Similarly, pediatric patients typically respond well to first-line treatment (combination chemotherapy) but 10 to 20% of total patients relapse with chemotherapy-resistant disease, leading to a significant unmet need in pediatric patients with high-risk relapsed or refractory ALL.

"We are pleased to receive orphan drug designation for AUTO1 for acute lymphoblastic leukemia," said Dr. Christian Itin, chairman and chief executive officer of Autolus. "From the data reported in our ongoing studies, we have seen strong remission rates and excellent CAR T cell expansion and persistence without inducing high-grade CRS, a serious adverse event affecting a significant number of patients on currently available CAR T treatments. We look forward to presenting data on AUTO1 at ASH (Free ASH Whitepaper) at the end of the year."

Orphan drug designation is granted by the FDA Office of Orphan Products Development to drugs and biologics which are intended for the treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following marketing approval by the FDA. For more information about orphan designation, please visit the FDA website at www.fda.gov.

About AUTO1
AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells’ abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights from UCL Business plc (UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.