On November 14, 2018 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases, reported financial results for the third quarter ended September 30, 2018, and provided a business update (Press release, CohBar, NOV 14, 2018, View Source [SID1234531310]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As we previously discussed, we are moving forward with our plan to address the mild but persistent injection site reactions observed in our Phase 1 clinical trial of CB4211, under development for NASH and obesity," said Simon Allen, Chief Executive Officer of CohBar. "We will be sharing this plan shortly with the FDA, and look forward to the agency’s timely feedback and resuming clinical activities as expeditiously as possible."

"This first groundbreaking study of a mitochondria based therapeutic in humans will help inform us not only for the development of CB4211, but also for the development of other potential therapeutics from our large portfolio of novel peptides. We continue to believe in the potential of MBTs to treat a broad range of age-related diseases with large-population indications, including type 2 diabetes, cancer, neurodegenerative disease and cardiovascular disease," Mr. Allen concluded.

Third Quarter and Recent Business Highlights:

Clinical Study Update for CB4211. On November 5, 2018, the company announced the temporary suspension of its ongoing Phase 1 clinical study of CB4211, its lead MBT candidate under development as a potential treatment for non-alcoholic steatohepatitis (NASH) and obesity, in order to address mild injection site reactions that have been unexpectedly persistent. The company has a plan to address the persistent reactions and is seeking feedback from the FDA before resuming the clinical study.

Partnership with LifeSci Advisors. CohBar engaged LifeSci Advisors LLC ("LifeSci"), a leading New York-based investor relations consultancy serving life science companies, to implement a comprehensive investor outreach program that will include analyst and investor targeting/outreach, non-deal roadshows, corporate communications, and Key Opinion Leader (KOL) events in the field of mitochondria based therapeutics and age-related diseases.

Investment and Scientific Community Outreach. During the third quarter, CohBar’s CEO Simon Allen presented an overview of the Company and its clinical development program at the Bio Investor Forum. Additionally, company management met with investors during the H.C. Wainwright 20th Annual Global Investment Conference.
During the third quarter, CohBar’s founders, Dr. Pinchas Cohen and Dr. Nir Barzilai, continued to be recognized as international leaders in the study of aging, age-related diseases and mitochondrial science.

Dr. Cohen co-authored a number of papers published in scientific journals during the quarter including "Humanin Prevents Age-Related Cognitive Decline in Mice and is Associated with Improved Cognitive Age in Humans," in Nature Scientific Reports; "Chronic Treatment with the Mitochondrial Peptide Humanin Prevents Age-related Myocardial Fibrosis in Mice," in American Journal of Heart Circulation Physiology; "Characterizing the Protective Effects of SHLP2, a Mitochondrial-Derived Peptide, in Macular Degeneration," in Nature Scientific Reports; and "Mitochondrial biology and prostate cancer ethnic disparity," Carcinogenesis. Dr. Cohen also received a major grant from the National Institutes of Health recognizing his ground-breaking work in the field of mitochondrial science entitled, "Humanin is an AD resilience factor through its interaction with APOE4."

Dr. Barzilai delivered a keynote speech at the Aging Cell Conference in Sitges, Spain and co-authored a number of scientific papers published during the quarter including: "Aging as a Biological Target for Prevention and Therapy," in JAMA; "A framework for selection of blood-based biomarkers for geroscience-guided clinical trials: Report from the TAME Biomarkers Workgroup," in Geroscience; "Targeting Senescence," in Nature Medicine; "APOE Alleles and Extreme Human Longevity," Journals of Gerontology; "40 Years of IGF1: The Jekyll and Hyde of the Aging Brain," in Journal of Molecular Endocrinology; "Late-Life Targeting of the IGF1 Receptor Improves Healthspan and Lifespan in Female Mice," in National Communications.
Third Quarter 2018 Financial Highlights

Cash and Investments. CohBar had cash and investments of $24,223,712 on September 30, 2018, compared to $8,452,459 on December 31, 2017.

R&D Expenses. Research and development expenses were $3,435,509 in the three months ended September 30, 2018, compared to $2,316,454 in the prior year quarter. The increase was primarily due to the costs of our clinical activities and a net increase in stock-based compensation related to performance equity grants that vested within the current year quarter, offset by a decrease in costs related to the timing of IND-enabling activities incurred in the prior year period.

G&A Expenses. General and administrative expenses were $1,061,709 for the three months ended September 30, 2018, compared to $549,505 in the prior year quarter. The increase in general and administrative expenses was primarily due to an increase in stock-based compensation related to performance equity grants that vested within the current year quarter, the costs associated with new grants made since the prior year period and an increase in directors fees in the current quarter.

Net Loss. For the three months ended September 30, 2018, net loss was $4,613,042, or $0.11 per basic and diluted share, compared to a net loss of $2,861,107, or $0.07 per basic and diluted share, for the three months ended September 30, 2017.

Details for Conference Call

Date: November 14, 2018
Time: 5:00 p.m. Eastern Time

- Dial-in U.S. and Canada: (888) 394-8218
- Dial-in International: (323) 701-0225
- Conference ID Number: 1659879

We kindly request that you call into the conference audio approximately 10 minutes before the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on November 14, 2018, through 11:59 p.m. Eastern Time on December 5, 2018. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 1659879. The audio replay will also be available at www.cohbar.com from November 14 through December 5, 2018.

About CB4211
CohBar’s lead program is based on CB4211, a first-in-class mitochondria based therapeutic (MBT) that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide (MDP) which was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators and has been shown to play a significant role in the regulation of metabolism. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes a potential activity readout relevant to NASH and obesity. In November 2018, the company announced the temporary suspension of the trial to address mild injection site reactions that were unexpectedly persistent. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is currently no approved treatment for the disease.

On November 14, 2018 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of two Type II variation applications to the European Medicines Agency (EMA) seeking approval for the expanded use of IMBRUVICA (ibrutinib) (Press release, Johnson & Johnson, NOV 14, 2018, View Source [SID1234531468]). One application seeks to include use of ibrutinib in combination with obinutuzumab in previously untreated adults with chronic lymphocytic leukaemia (CLL) and to add long-term follow-up data from the existing label studies RESONATETM (PCYC-1112) and RESONATETM-2 (PCYC-1115). The second is for use of ibrutinib plus rituximab for the treatment of previously untreated and relapsed/refractory adults with Waldenström’s macroglobulinemia (WM).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Today’s news brings us one step closer to potentially offering ibrutinib in new combinations for patients where unmet needs still persist," said Dr. Catherine Taylor, Haematology Therapy Area Lead, Europe, Middle East and Africa (EMEA), Janssen-Cilag Limited. "Ibrutinib continues to demonstrate clinical benefit over the long term for a broad
group of patients living with blood cancer, and we look forward to working with relevant authorities to secure approval of these new combinations."

Ibrutinib, a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, is jointly developed and commercialised by Janssen Biotech, Inc., and Pharmacyclics LLC, an AbbVie company. The CLL submission is supported by positive results from the Phase 3 iLLUMINATE (PCYC1130) study which investigated ibrutinib in combination with obinutuzumab versus
chlorambucil plus obinutuzumab in patients with newly diagnosed CLL.

1 Study findings from iLLUMINATE will also be featured as an oral presentation (abstract #691), whilst further analysis of RESONATETM and RESONATETM-2 results in comparison with real-world evidence databases (abstract #4427) will be included at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place in San Diego next month.1,2 A supplemental New Drug Application (sNDA) which was also recently submitted to the U.S. Food and Drug Administration (FDA) received Priority Review.

2 In WM, the submission is supported by data from the Phase 3 iNNOVATE (PCYC-1127) study evaluating ibrutinib in combination with rituximab, versus rituximab with placebo, in patients with previously untreated and relapsed/refractory WM.

3 Follow-up efficacy and safety findings from the iNNOVATE study will also be presented at ASH (Free ASH Whitepaper) 2018 (abstract
#149).

4 In August 2018, the FDA approved ibrutinib in combination with rituximab for the treatment of WM based on the data from iNNOVATE.

5 Additional information about both studies can be found at www.ClinicalTrials.gov (NCT02264574 and NCT02165397).
6,7
#ENDS#

About ibrutinib
Ibrutinib is a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor, which works by forming a strong covalent bond with BTK to block the transmission of cell survival signals within the malignant B-cells.8 By blocking this BTK protein, ibrutinib helps kill and reduce the number of cancer cells, thereby delaying progression of the cancer.9 Ibrutinib is currently approved in Europe for the following uses:10

 Chronic lymphocytic leukaemia (CLL): As a single agent for the treatment of adult patients with previously untreated CLL, and as a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy.

 Mantle cell lymphoma (MCL): Adult patients with relapsed or refractory mantle cell MCL.

 Waldenström’s macroglobulinemia (WM): Adult patients who have received at least one prior therapy or in first-line treatment for patients unsuitable for chemoimmunotherapy.

The most common adverse reactions seen with ibrutinib include diarrhoea, neutropenia, haemorrhage (e.g., bruising), musculoskeletal pain, nausea, rash, and pyrexia.10

For a full list of side effects and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics for further information.

About CLL
CLL is typically a slow-growing blood cancer of the white blood cells.11 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year with rates amongst men and women approximately 5.87 and 4.01 cases per 100,000 persons per year, respectively.

12 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.13
CLL is a chronic disease; median overall survival ranges between 18 months and more than 10 years, according to the stage of disease.14 The disease eventually progresses in the majority of patients, and patients are faced with fewer treatment options with each relapse. Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.
3
About Waldenström’s macroglobulinemia
Waldenström’s macroglobulinemia (WM) is a rare form of non-Hodgkin’s lymphoma (NHL).15 It causes overproduction of a protein called monoclonal immunoglobulin M (IgM) antibody, which causes a thickening of the blood.
16 Incidence rates among men and women in Europe are approximately 7.3 and 4.2 per million persons, respectively.17 The causes of WM are unknown, with it typically affecting older adults and being slightly more
common in men than women.15,17

On November 14, 2018 Neuralstem, Inc. (Nasdaq: CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell and small molecule technologies, reported its financial results for the third quarter ended September 30, 2018 (Press release, Neuralstem, NOV 14, 2018, View Source [SID1234531311]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the third quarter of 2018 we have made progress with our programs, have taken steps to strengthen the balance sheet, and, perhaps most importantly, have determined how we will focus our efforts moving forward," said Jim Scully, Interim Chief Executive Officer of Neuralstem. "We believe that proceeds from our recent offering, combined with our efforts to reduce cash burn, will allow us to pursue key development initiatives that will be detailed in the coming weeks."

Clinical Highlights

In August, NSI-189 received from FDA the orphan designation for treatment of Angelman Syndrome. Angelman Syndrome (AS) is a rare congenital genetic disorder caused by a lack of function in the UBE3A gene on the maternal 15th chromosome. It affects approximately one in 15,000 people – about 500,000 individuals globally. Symptoms of AS include developmental delay, lack of speech, seizures, and walking and balance disorders. Patients with AS may never walk or speak and require life-long care. Life expectancy is normal which places a significant burden on patients and caregivers. There are currently no FDA-approved therapies for the treatment of Angelman syndrome.

In July, the company announced initiation of a Phase 2 clinical trial to further evaluate NSI-566 in ischemic stroke. This follows the positive results of the open-label Phase 1 stroke study disclosed in a 2018 ISSCR (International Society for Stem Cell Research) abstract on June 23, 2018. The Phase 2 study is a randomized, double-blind, sham-surgery controlled study. It is intended to further test the safety and efficacy of NSI-566 to reverse paralysis in stroke patients with half of their body partially paralyzed. The trial is taking place at BaYi Brain Hospital in Beijing, China, and commenced on August 1, 2018.

In October, the company announced publication of a manuscript in Scientific Reports showing that transplantation of NSI-532.IGF1 mitigates disease pathology and improves cognition in a mouse model of Alzheimer’s Disease. The study was performed at the University of Michigan by a team led by Dr. Eva Feldman, Director of the Program for Neurology Research and Discovery, and Research Director of the University of Michigan ALS Center of Excellence. NSI-532.IGF1 is the first candidate from the NSI-532 program, a second-generation cell therapy program which combines neural stem cells with neuroprotective proteins.
Corporate Highlights

In October, the company completed a registered direct offering of its securities which resulted in gross proceeds to Neuralstem of $2.1 million. Neuralstem intends to use the proceeds from this offering to further its clinical and preclinical programs, and for general working capital.
Financial Results for the Quarter Ended September 30, 2018

Cash Position and Liquidity: At September 30, 2018, cash and investments was $5.7 million as compared to $7.1 million at June 30, 2018. The $1.4 million decrease reflects a loss for the period, which was somewhat ameliorated by management’s cost reduction efforts. The company expects its existing cash, cash equivalents and short-term investments to fund its operations, based on its current operating plans, into the second quarter of 2019.

Operating Loss: Operating loss for the third quarter ended September 30, 2018 was $2.1 million compared to a loss of $2.6 million for the comparable period of 2017. The reduction in loss is attributed to reduced clinical development activity and management efforts to reduce overall expenses.

Net Loss: Net loss for the third quarter ended September 30, 2018 was $1.8 million, or $0.12 per share (basic), compared to a loss of $0.1 million, or $0.01 per share (basic), for the comparable period of 2017. The increase in net loss was primarily due to the non-cash gains related to the change in the fair value of our liability classified warrants in 2017, partially offset by a decrease in our operating loss.

Research and Development Expenses: The $0.9 million of research and development expenses for the quarter ended September 30, 2018 represents a 35% decrease over the comparable period of 2017. This decrease was primarily attributable to a decrease in personnel and facility expenses, and a decrease in clinical trial and related costs due to the completion of our NSI-189 Phase 2 clinical trial.

General and Administrative Expenses: The $1.2 million of general and administrative expenses for the third quarter ended September 30, 2018 represents a 2% decrease over the comparable period of 2017. This decrease was primarily attributable to lower payroll and related expenses due to corporate restructuring and cost reduction efforts coupled with a decrease in non-cash share-based compensation expense which was offset by an increase in tax and insurance expenses and consulting and professional fees.

On November 13 2018 FUJIFILM Corporation (President: Kenji Sukeno) reported that it has succeeded in stably encapsulating the anti-cancer agent topotecan*, approved for the treatment of several solid cancers, in newly invented liposome (Press release, Fujifilm, NOV 13, 2018, View Source [SID1234538525]). When preclinical studies in mice were conducted using a drug where topotecan was encapsulated in liposomes (development number: FF-10850), in addition to confirming high tumor shrinking effects, improved pharmacological efficacy was demonstrated when administered in combination with an immune checkpoint inhibitor**, with prolonged survival compared to monotherapy. Going forward, based on the results of these nonclinical studies, the company will prepare for the early initiation of clinical studies of the drug candidate.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Fujifilm conducts research and development on the potential use of encapsulating drugs in liposomes, artificially constructed vesicles made from organic phospholipids that make up cellular membranes, by harnessing its advanced nano-dispersion technology, analysis technology, and process technology cultivated through its wide range of product development. In another study the liposome drug candidate FF-10832, which encapsulates the anti-cancer agent gemcitabine*** , has been observed to protect gemcitabine from elimination and demonstrate an enhanced permeability and retention (EPR) effect*4 by which the FF-10850 accumulates within tumor and releases gemcitabine. A Phase I clinical trial of FF-10832 is underway in the U.S.
Topotecan has an extremely short half-life*5 in the blood, and also has the issue of causing serious bone marrow suppression*6 as a side effect in more than 80% of patients. A liposome drug where topotecan is encapsulated can be considered as a way to resolve these issues; however, topotecan has a tendency to pass through the liposome membrane, resulting in a problem where topotecan would leak into the blood before reaching the tumor. By enhancing the strength of the liposome membrane with the addition of new materials to the liposome ingredients, Fujifilm has succeeded in stably encupsulating topotecan. Fujifilm is conducting research on the drug as a candidate anti-cancer agent (development number: FF-10850), and has obtained the following results in studies in mice.

[Research Result 1] High tumor shrinking effects confirmed with monotherapy

1) Experiment:
Mice with transplanted human-derived ovarian cancer cells (ES-2) were administered with topotecan and FF-10850, respectively, and the efficacy and tolerability were confirmed for each dosage. The period of administration was five consecutive days for topotecan and two cycles of one administration per week for FF-10850.
2) Results:
With topotecan, efficacy was observed with 30 mg/m2 (6 mg/m2 x five times), while with FF-10850, efficacy was confirmed with 3 mg/m2 (1.5 mg/m2 x twice). When the two drugs were compared, FF-10850 demonstrated efficacy that was greater than or equal to topotecan with 1/10 of the total dose of topotecan.
With FF-10850, tumor shrinking effects were seen with 8 mg/m2 (4 mg/m2 x twice). Furthermore, the relative body weight change which is an indicator of tolerability, was less than that of topotecan.
[Chart] Tumor volume and relative body weight change upon administration of FF-10850 and topotecan
Based on the above, FF-10850 is expected to have high pharmacological efficacy that exceeds that of topotecan while maintaining tolerability even when dosage is increased.

[Research Result 2] Improved pharmacological efficacy observed when administered in combination with an immune checkpoint inhibitor

1) Experiment:
Monotherapies and combination therapy with an immune checkpoint inhibitor** and FF-10850 was conducted on mice transplanted with mouse-derived colorectal cancer cells (CT26), and efficacy and tolerability were confirmed. The period of administration was three cycles of two administrations per week for the immune checkpoint inhibitor (30 mg/m2) and three cycles of one administration per week for FF-10850 (6 mg/m2).
2) Results:
When administered as a monotherapy, the median value for mouse survival time was 19 days for the immune checkpoint inhibitor and 27.5 days for FF-10850. Meanwhile, when administered as a combination therapy, the median value for mouse survival time exceeded 40 days, demonstrating a statistically significant difference when compared with the monotherapies. Mouse survival rates for the combination therapy were 75% at 40 days after administration, which was higher than the monotherapies.
Even when administered as a combination therapy, noticeable side effects such as weight loss were not observed, and there were no issues with tolerability.
[Chart] Survival rates and median survival time when administered in combination with an immune checkpoint inhibitor
Based on the above, FF-10850 is expected to further suppress tumor proliferation through administration with an immune checkpoint inhibitor and prolong survival time.

Fujifilm will present its research results on FF-10850 at the "30th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) SYMPOSIUM", one of the world’s most prominent cancer related conferences, to be held in Dublin, Ireland from November 13th to 16th, 2018.

Fujifilm is harnessing its advanced technologies such as the nano-dispersion technology and ability to synthesize and design compounds to undertake the development of new drugs in the key areas of cancer, central nervous system diseases, and infectious diseases. The company is also focusing on developing drug delivery system (DDS) technologies including liposome drugs, undertaking research and development to apply DDS technologies not only to low-molecular-weight drugs but also to next-generation drugs such as nucleic acid drugs and gene therapy drugs. Going forward, the company will contribute to the resolution of social issues by developing and delivering innovative, high value-added pharmaceutical products.

* An anti-cancer agent (generic name: topotecan, product name: Hycamtin) developed by GlaxoSmithKline plc. Currently, the drug is distributed by Novartis. It is used as a treatment for ovarian cancer, small-cell lung cancer, and cervical cancer.
** The general term for drugs that have an effect by enabling activated immune cells to attack cancer cells by inhibiting the mechanism (immune checkpoints) that weakens the action of immune cells. Widely used in the treatment of malignant melanomas, lung cancer, stomach cancer, and kidney cancer. The immune checkpoint inhibitor used in the combination therapy with FF-10850 was an anti-PD-1 antibody.
*** An anti-cancer drug (generic name: gemcitabine, product name: Gemzar) developed by the U.S. company Eli Lilly and Company. It is used as a drug of first choice for the treatment of pancreatic cancer, and is also indicated for the treatment of a wide range of other cancers (such as lung cancer and ovarian cancer).
*4 As they grow, tumors generate surrounding blood vessels, but these newly generated blood vessels are not fully developed and have large gaps that are much smaller in normal blood vessels. When liposomes and polymers are retained within the blood, they do not permeate the walls of normal blood vessels, which have small gaps, permeating only the vascular walls around the tumor. In addition, as lymphatic vessels are not fully developed in tumors, the liposomes and polymers that have permeated are not easily eliminated, resulting in the accumulation of these liposomes and polymers in the tumor. This is called the EPR (enhanced permeability and retention) effect.
*5 The time required for the concentration of a drug in the blood to be reduced to half.
*6 The state where production of white blood cells, platelets, and red blood cells in the bone marrow is reduced, leading to increased risk of infection and bleeding and symptoms such as anemia.

On November 13, 2018 Medigene AG (FSE: MDG1, Frankfurt, Prime Standard, SDAX), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies for the treatment of cancer, reported financial results for the first 9 months of 2018 and provided a review of recent accomplishments and anticipated upcoming milestones (Press release, MediGene, NOV 13, 2018, View Source [SID1234531242]). The company further improved its financial guidance for the fiscal year 2018.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Major events in the first three quarters of 2018:
– Start of a Phase I/II clinical trial with T cell receptor-modified T cell therapy (TCR-T) MDG1011 in blood cancers including Acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and multiple myeloma (MM)
– Expansion of TCR alliance with bluebird bio from four to six TCR projects
– Raise of EUR 32.3 million in oversubscribed private placement to new and existing investors
– Strengthened patent portfolio with new TCR patents in USA and Europe
– Presented data on the successful production of dendritic cell (DC) vaccines for the current Phase I/II clinical trial
– Collaboration with Structured Immunity on improved T cell receptor development

Update Immunotherapies
– Authority approves simplified criteria for patient recruitment in Phase I/II trial with TCR-therapy MDG1011: The German regulatory authority, the Paul-Ehrlich-Institut (PEI), has approved Medigene’s amendment of the trial inclusion criteria. Initial trial protocol required inclusion of one patient per indication (AML, MDS, MM) in a dose cohort of three patients. The adjusted trial protocol requires at least one patient with MM and at least one patient with either AML or MDS. The adjusted trial protocol enables more flexibility and potentially more rapid enrollment of patients. Moreover, an analytical method for determining PRAME expression has been optimized, which results in an increase in the number of potential patients for the clinical trial.
– Successful manufacturing of the first personalized cell product MDG1011: The first personalized MDG1011 cell product was successfully manufactured with patient-specific T cells in compliance with the clinical trial protocol in the course of the trial. A sufficient number of therapeutic TCR-modified T cells with autologous T cells were generated despite the advanced disease stage of the patient. However, the patient could not be treated with the therapeutic product because he dropped out of the trial beforehand due to rapid progression of the underlying disease.
– Preparations to increase the number of trial centers started: Medigene is undertaking intensive preparations with a number of additional hospitals in order to increase the number of centers to allow for an accelerated patient recruitment.

Prof. Dolores Schendel, CEO of Medigene AG, comments: "We are delighted to show that the manufacturing of our highly innovative product MDG1011 worked well with a patient’s own cells in our first-in-man and first-in-country trial with a TCR-modified T cell therapy. The modified study protocol and the expansion of the study centers will improve the general admission for the treatment of these critically ill patients. Our financials are on track and the guidance reflects our intensive research and development activities in the further development of our immunotherapies."

Nine months’ financial results:
– Total revenue increased by 11% to EUR 8.0 million
– Revenue from the core business of immunotherapies increased by 38% to EUR 4.7 million
– Research and development (R&D) expenses increased as planned by 20% to EUR 13.3 million due to extension of the preclinical and clinical development activities for immunotherapy programs
– EBITDA loss increased as anticipated by 5% to EUR 10.7 million
– Net loss increased by 14 % to EUR 12.2 million due to higher R&D expenses and currency effects
– Cash & cash equivalents and time deposits of EUR 76.3 million as at September 30, 2018
– Further improvement of the financial guidance 2018

Financial guidance 2018:
Medigene further improves the financial guidance for 2018 which was raised in the 6M financial statement:
– The Company continues to expect total revenue of EUR 9.5 – 10.5 million in 2018.
– Due to lower than estimated clinical trial costs in 2018, the company now expects to spend EUR 19 – 21 million for the full year 2018 (previous guidance: EUR 21 – 23 million).
– As a result, Medigene is expecting to make a loss at EBITDA level of EUR 16 – 18 million (previous guidance: EUR 18 – 20 million).
– Excluding the proceeds from the capital increase conducted in May 2018, Medigene forecasts a total cash usage of EUR 12 – 14 million for 2018 (previous guidance: EUR 15 – 17 million)
– This forecast does not include potential future milestone payments or cash flows from existing or future partnerships or transactions, as the occurrence of such events and their timing and amount depend to a large extent on external parties and therefore cannot be reliably predicted by Medigene.
– Based on its current planning, the Company has sufficient financial resources to fund business operations beyond the planning horizon of two years.

Outlook immunotherapies:
T cell receptor-modified T cells (TCR-Ts)
The Company is planning to commence treatment of the first dose cohort in the Phase I/II clinical trial of Medigene’s TCR-T MDG1011 therapy for acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and multiple myeloma (MM). Phase I focuses on the safety and tolerability of the treatment with MDG1011.
In addition to the MDG1011 clinical trial, Medigene will also work on characterizing new TCR candidates for future Medigene-sponsored clinical trials and collecting preclinical data to prepare applications for further clinical TCR-T trials. In addition, Medigene will continue its successful and expanded collaboration with bluebird bio and expects to make further progress on TCR candidate discovery.

Dendritic cell vaccines (DCs)
Medigene continues its ongoing Phase I/II clinical trial with the DC vaccine to treat acute myeloid leukemia (AML). Data from all patients over a treatment duration of one year is expected in the fourth quarter of 2018. This corresponds to the half-way point of the full treatment period. This interim analysis is planned to be presented at scientific conferences in 2019. The final clinical data from the Phase I/II trial are expected at the conclusion of the two-year treatment for all patients towards the end of 2019.

The full version of the quarterly statement 9M-2018 can be downloaded here: www.medigene.com/investors-media/reports-presentations/

Conference call and webcast: A telephone conference (webcast) in English will be held today at 3:00 pm CET (Munich/Frankfurt) / 9:00 am EST (New York) and transmitted live via webcast. Access and transmission of the synchronized presentation slides and a recording of the presentation is available on the homepage of Medigene at www.medigene.com/investors-media/reports-presentations/webcasts/