On November 5, 2020 Genmab A/S (Nasdaq: GMAB) reported that Janssen Pharmaceutica NV (Janssen) has submitted a Type II variation application to the European Medicines Agency (EMA) for the subcutaneous (SC) formulation of daratumumab (daratumumab and hyaluronidase-fihj) in combination with bortezomib, cyclophosphamide, and dexamethasone (VCd) for the treatment of adult patients with light-chain (AL) amyloidosis (Press release, Genmab, NOV 5, 2020, View Source [SID1234570087]). In August 2012, Genmab granted Janssen Biotech, Inc. (Janssen) an exclusive worldwide license to develop, manufacture and commercialize daratumumab. The submission of this Type II variation application study triggers a USD 5 million milestone payment to Genmab.

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"We are extremely pleased about the submission for subcutaneous DARZALEX in patients with AL amyloidosis to the European Health Authorities based on the Phase 3 ANDROMEDA (AMY3001) study. We are hopeful that this will lead to the first approved treatment option for patients with this devastating disease which would also be the first approved indication for DARZALEX in Europe outside of multiple myeloma," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The submission is based on the Phase 3 ANDROMEDA study of daratumumab and hyaluronidase-fihj in combination with VCd as treatment for patients with newly diagnosed AL amyloidosis. The data were presented as a late-breaking abstract at the 25th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress in June 2020.

The milestone associated with this submission does not impact Genmab’s 2020 guidance.

About the ANDROMEDA (AMY3001) study
The Phase 3 study (NCT03201965) included 388 patients newly diagnosed with AL amyloidosis. Patients were randomized to receive treatment with either daratumumab and hyaluronidase-fihj in combination with bortezomib (a proteasome inhibitor), cyclophosphamide (a chemotherapy), and dexamethasone (a corticosteroid) (VCd) or treatment with VCd alone. The primary endpoint of the study was the percentage of patients who achieve hematologic complete response.

About Light-chain (AL) Amyloidosis
Amyloidosis is a disease that occurs when amyloid proteins, which are abnormal proteins, accumulate in tissues and organs. When the amyloid proteins cluster together, they form deposits that damage the tissues and organs. AL amyloidosis most frequently affects the heart, kidneys, liver, nervous system and digestive tract. There is currently no cure or existing approved therapies for AL amyloidosis though it can be treated with chemotherapy, dexamethasone, stem cell transplants and supportive therapies.1 It is estimated that in 2019 there were 4,388 diagnosed incident cases of AL amyloidosis in the five major European markets2

About DARZALEX(daratumumab)
DARZALEX (daratumumab) has become a backbone therapy in the treatment of multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with carfilzomib and dexamethasone for the treatment of patients with relapsed/refractory

multiple myeloma who have received one to three previous lines of therapy; in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.3 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma.

DARZALEX is indicated for the treatment of adult patients in Europe via intravenous infusion or subcutaneous administration: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy4. Daratumumab is the first subcutaneous CD38 antibody approved in Europe for the treatment of multiple myeloma. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S.

In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX FASPRO is the first subcutaneous CD38 antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).3,6,7,8,9

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

On November 5, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment, MDAX & TecDAX; NASDAQ: MOR) reported that it will publish its results for the third quarter and first 9-month of 2020 on November 11, 2020 at 10:00pm CET (9:00pm GMT; 4:00pm EST) (Press release, MorphoSys, NOV 5, 2020, View Source [SID1234570086]).

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MorphoSys’ Management team will host a conference call and webcast on November 12, 2020 at 2:00pm CET (1:00pm GMT; 8:00am EST) to present the third quarter and first 9-month financial results 2020 and the further outlook for 2020.Please dial in 10 minutes before the beginning of the conference.

A live webcast and slides will be made available at www.morphosys.com.

Approximately two hours after the call, a slide-synchronized audio replay of the conference and a transcript of the prepared remarks will be available on www.morphosys.com.

On November 5, 2020 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biotechnology company focused on developing and commercializing innovative medicines worldwide,reported recent business highlights, anticipated upcoming milestones, and financial results for the third quarter and first nine months of 2020 (Press release, BeiGene, NOV 5, 2020, View Source [SID1234570085]).
"Our commercial teams continue to execute and sales of our recently launched internally developed products drove total product revenue to $91 million for the third quarter, a 39 percent increase compared to last quarter," said John V. Oyler, Co-Founder, Chief Executive Officer, and Chairman of BeiGene. "We believe that we are well-positioned to accelerate the development of our deep pipeline, further expand our portfolio in oncology and into other therapeutic areas, and continue to build our capabilities and operations for our products to serve more patients worldwide. In the remainder of 2020 and 2021, we look forward to key clinical readouts, as well as potential expanded commercial opportunities for our products through approvals in additional indications and geographic markets and by growing our commercial-stage portfolio in China to up to 12 products."
Howard Liang, Ph.D., Chief Financial Officer and Chief Strategy Officer, plans to retire from BeiGene following a transition period and the appointment of a new chief financial officer, which is expected to occur around the end of the first quarter of 2021.
"Howard has contributed greatly to our success since joining BeiGene in 2015. Since that time, Howard has been instrumental in each milestone and accomplishment as we expanded our footprint and became a truly global commercial organization with world-class research, development and manufacturing. We are grateful for everything he has done for BeiGene," said John V. Oyler.
Recent Business Highlights and Upcoming Milestones
Commercial Operations
•Generated $91.08 million in product revenue in the three months ended September 30, 2020, representing an 81.6% increase compared to the comparable period of the prior year. Product revenue was driven by sales of our recently launched internally developed products tislelizumab in China and BRUKINSA in China and the United States.
Development Programs
BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects. BRUKINSA has received accelerated approval in the United States for the treatment of adult patients with MCL who have received at least one prior therapy; and approval in China in two indications – the treatment of adult patients with CLL/SLL who have received at least one prior therapy, and the treatment of adult patients with MCL who have received at least one prior therapy. BRUKINSA is under development globally for additional approvals.
•Received acceptance from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for a supplemental new drug application (sNDA) of BRUKINSA for the treatment of patients with Waldenström’s macroglobulinemia (WM) with priority review;
•Announced acceptance and priority review by Health Canada of a New Drug Submission (NDS) for BRUKINSA in WM; a subsequent NDS for patients with MCL in Canada has been accepted;
•Initiated patient enrollment in a Phase 1 trial (NCT04436107) of BRUKINSA in combination with lenalidomide, with or without rituximab, in patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma; and
•Received Fast Track designation in the U.S. for patients with marginal zone lymphoma (MZL).
Expected Milestones for BRUKINSA
•Present data from the pivotal Phase 2 MAGNOLIA trial (NCT03846427) in patients with R/R MZL, the pivotal Phase 2 trial in patients with WM in China (NCT03332173), the Phase 2 trial (NCT04116437) in patients with R/R B-cell malignancies who are intolerant to ibrutinib or acalabrutinib, and from Arm C of the SEQUOIA trial (NCT03336333) in patients with treatment-naïve CLL or SLL with del(17p) at the upcoming 62nd ASH (Free ASH Whitepaper) Annual Meeting being held virtually December 5-8, 2020;
•Announce top-line results from the SEQUOIA trial (NCT03336333) comparing BRUKINSA with bendamustine plus rituximab in patients with treatment-naïve CLL or SLL as early as the first half of 2021;
•Continue to discuss data from the Phase 3 ASPEN trial (NCT03053440) comparing BRUKINSA to ibrutinib in patients with WM with the U.S. Food and Drug Administration (FDA); and
•Complete enrollment in the Phase 3 ALPINE trial (NCT03734016) comparing BRUKINSA with ibrutinib in patients with R/R CLL/SLL in 2020.
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in two indications – the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, and the treatment of patients with locally advanced or metastatic urothelial carcinoma with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Tislelizumab is under development globally for additional approvals.
•Presented the first reported data from the Phase 3 RATIONALE 304 trial (NCT03663205) of tislelizumab combined with chemotherapy for the first-line treatment of patients with advanced non-squamous non-small cell lung cancer (NSCLC) at the 2020 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress. Data from this trial were included in the supplemental biologics license application (sBLA) currently under review by the NMPA;
•Began patient enrollment in a Phase 2 trial (NCT04401800) of tislelizumab in combination with lenvatinib in patients with hepatocellular carcinoma; and
•Completed patient enrollment in the Phase 3 clinical trial (NCT03924986) of tislelizumab combined with chemotherapy versus chemotherapy alone in recurrent or metastatic nasopharyngeal cancer.
Expected Milestones for Tislelizumab
•Announce top-line results from the global Phase 3 trial (NCT03358875) comparing tislelizumab versus docetaxel in second-or third-line patients with NSCLC and the global Phase 3 trial (NCT03430843) comparing tislelizumab versus chemotherapy in second-line patients with advanced esophageal squamous cell carcinoma (ESCC) before the end of 2020 or in 2021.
Pamiparib, an investigational selective small molecule inhibitor of PARP1 and PARP2
•Presented data from the pivotal Phase 2 trial (NCT03333915) of pamiparib in patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy, at the 2020 ESMO (Free ESMO Whitepaper) Virtual Congress. Data from this trial were included in the NDA currently under review by the NMPA.
Expected Milestones for Pamiparib
•Announce top-line results from the Phase 3 trial (NCT03519230) of pamiparib as a maintenance treatment in patients with platinum-sensitive recurrent ovarian cancer (OC) before the end of 2020 or in 2021.
BGB-A1217, an investigational TIGIT monoclonal antibody
•Continued to enroll patients in the global Phase 1 clinical trial (NCT04047862) in combination with tislelizumab. The recommended Phase 2 dose has been determined and pivotal trials are in planning globally.
Early-Stage Proprietary Programs
•Received acceptance of the investigational new drug (IND) submission in China for BGB-11417 (BCL-2 inhibitor) for mature B-cell malignancies; and
•Continued to advance our earlier-stage pipeline of internally-developed assets, including BGB-11417 (BCL-2 inhibitor in Phase 1 development for cancer), BGB-A445 (non-ligand competing OX40 monoclonal antibody in Phase 1 development in combination with tislelizumab for solid tumor), BGB-10188 (PI3Kδ inhibitor in Phase 1 development in combination with BRUKINSA or tislelizumab for cancer), and BGB-15025 (HPK1 inhibitor in preclinical development for cancer).
Collaboration Programs
Amgen
•Our collaboration with Amgen continues to progress, with ongoing commercial activities for XGEVA (denosumab) in China for patients with giant cell tumor of the bone (GCTB), as well as preparation for the launch of:
–XGEVA (120-mg denosumab) for the prevention of skeletal-related events in patients with bone metastases from solid tumors and in patients with multiple myeloma following potential approval expected in the fourth quarter 2020 or in early 2021;
–BLINCYTO (blinatumomab) for the treatment of adult patients with R/R B-cell precursor acute lymphoblastic leukemia (ALL) following potential approval expected in the fourth quarter 2020 or in early 2021; and
–KYPROLIS (carfilzomib) for patients with R/R multiple myeloma following potential approval expected in 2021;
•Amgen has advised BeiGene that its applications to the Human Genetic Resources Administration of China (HGRAC) to obtain approval to conduct clinical studies in China for assets that are part of the Amgen-BeiGene Collaboration, including its application for sotorasib (AMG 510), a first-in-class investigational KRAS G12C inhibitor, are currently delayed. Approval from the HGRAC is required for the initiation of clinical trials involving the collection of human genetic materials in China. BeiGene and Amgen continue to plan for the commencement of these clinical trials while Amgen awaits further information from HGRAC. BeiGene does not expect this to affect the conduct of the clinical trials in China for its drug candidates other than assets that are part of the Amgen-BeiGene collaboration.
Zanidatamab (ZW25), a novel investigational Azymetric bispecific antibody against HER2 currently in late-stage clinical development with Zymeworks Inc.
•Began patient enrollment in a registration-enabling Phase 2 clinical trial (NCT04466891) in patients with advanced or metastatic HER2-amplified biliary tract cancers.
DKN-01, a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, in development with Leap Therapeutics.
•Announced the first patient dosed in the DisTinGuish trial (NCT04363801), a Phase 2a clinical trial initiated by Leap, evaluating DKN-01 in combination with tislelizumab, with or without chemotherapy, in patients with gastric or gastroesophageal junction cancer (G/GEJ).
DXP-593 and DXP-604, SARS-CoV-2 neutralizing antibody drug candidates identified by Singlomics (Beijing DanXu) Biopharmaceuticals Co., Ltd. and licensed to BeiGene outside of Greater China. DXP-593 and DXP-604 can potentially be used as a cocktail treatment option that could avoid resistance due to viral mutation.
•Initiated a Phase 1 clinical trial of healthy volunteers (NCT04532294) in Australia.
BA3071, a novel, investigational conditionally-active CTLA-4 inhibitor discovered by BioAtla, Inc.
•Announced an amended agreement with BioAtla to license in BA3071 globally.
Manufacturing Operations
•Completed facility and process validation for the first plant of our biologics manufacturing facility in Guangzhou;
•Initiated expansion of the second and third plants of our biologics manufacturing facility in Guangzhou to significantly increase manufacturing capacity and introduce new manufacturing technology platforms, expected to be completed by the end of 2020 and 2021, respectively; and
•Entered into an agreement to acquire the 5% equity interest in BeiGene Biologics Co., Ltd. ("BeiGene Biologics") held by the Guangzhou High-tech Zone Technology Group Co., Ltd. (formerly Guangzhou GET Technology Development Co., Ltd.) ("GET"), an affiliate of Guangzhou Development District, and repay the related shareholder loan. Upon the update of the business license, which is expected to occur in the fourth quarter of 2020, our Guangzhou biologics facility will become a wholly-owned subsidiary of BeiGene Hong Kong Co., Limited.
on our ongoing and newly initiated late-stage pivotal clinical trials, R&D expense related to upfront license payments for our in-licensed assets, development expenses associated with the Amgen collaboration, the preparation for additional regulatory submissions, and manufacturing costs related to pre-commercial activities and supply. In-process R&D expense for in-licensed assets totaled $66.5 million in the three months ended September 30, 2020, compared to none in the prior year period. Our co-funding obligation for the development of the pipeline assets under the Amgen collaboration for the three months ended September 30, 2020 was $60.85 million, of which $30.80 million was recorded as R&D expense. The remaining $30.05 million was recorded as a reduction of the R&D cost share liability. R&D-related share-based compensation expense was $25.41 million for the three months ended September 30, 2020, compared to $20.67 million for the same period of 2019.
•SG&A Expenses for the three months ended September 30, 2020 were $160.84 million, compared to $105.00 million in the same period of 2019. The increase in SG&A expenses was primarily attributable to increased headcount, primarily related to the expansion of our commercial team to support the distribution of our products in China and the United States, as well as higher professional service fees and costs to support our growing operations. SG&A-related share-based compensation expense was $24.89 million for the three months ended September 30, 2020, compared to $16.14 million for the same period of 2019.
•Net Loss for the three months ended September 30, 2020 was $425.22 million, or $0.37 per share, and $4.81 per American Depositary Shares (ADS), compared to $307.36 million, or $0.39 per share, and $5.11 per ADS in the same period of 2019.
on our ongoing and newly initiated late-stage pivotal clinical trials, R&D expense related to upfront license payments for our in-licensed assets, development expenses associated with the Amgen collaboration, the preparation for additional regulatory submissions, and manufacturing costs related to pre-commercial activities and supply. In-process R&D expense for in-licensed assets totaled $66.5 million in the three months ended September 30, 2020, compared to none in the prior year period. Our co-funding obligation for the development of the pipeline assets under the Amgen collaboration for the three months ended September 30, 2020 was $60.85 million, of which $30.80 million was recorded as R&D expense. The remaining $30.05 million was recorded as a reduction of the R&D cost share liability. R&D-related share-based compensation expense was $25.41 million for the three months ended September 30, 2020, compared to $20.67 million for the same period of 2019.
•SG&A Expenses for the three months ended September 30, 2020 were $160.84 million, compared to $105.00 million in the same period of 2019. The increase in SG&A expenses was primarily attributable to increased headcount, primarily related to the expansion of our commercial team to support the distribution of our products in China and the United States, as well as higher professional service fees and costs to support our growing operations. SG&A-related share-based compensation expense was $24.89 million for the three months ended September 30, 2020, compared to $16.14 million for the same period of 2019.
•Net Loss for the three months ended September 30, 2020 was $425.22 million, or $0.37 per share, and $4.81 per American Depositary Shares (ADS), compared to $307.36 million, or $0.39 per share, and $5.11 per ADS in the same period of 2019.

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On November 5, 2020 vTv Therapeutics Inc. (Nasdaq:VTVT) reported financial results for the third quarter ended September 30, 2020, and provided an update on the progress of its clinical programs (Press release, vTv Therapeutics, NOV 5, 2020, View Source [SID1234570084]).

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"Following our decision to conclude enrollment in the Elevage Study at the end of September, we look forward to announcing topline results from this phase 2 study of azeliragon in December," said Steve Holcombe, president and CEO. "In addition, we are planning a mechanistic study of TTP399 to start early in the new year to better understand its impact on diabetic ketoacidosis. Finally, we are planning a phase 1 study of our PDE4 inhibitor compound, HPP737, as an initial step towards a proof-of-concept study in psoriasis. We expect to report topline results from both the mechanistic study of TTP399 and the phase 1 study of HPP737 during the second quarter of 2021."

Recent Achievements and Outlook

Type 1 Diabetes

FDA Engagement and Pivotal Study Planning. vTv continued its dialogue with the FDA during the third quarter surrounding the design and endpoints of its planned pivotal studies of TTP399 as an adjunct to insulin therapy for type 1 diabetes. Based upon the FDA’s feedback, the Company plans to conduct a placebo-controlled six-month clinical trial in approximately 400 subjects, followed by a second placebo-controlled six-month clinical trial to be initiated nine to twelve months after initiation of the first pivotal trial. The FDA also confirmed that the effect size of TTP399 on events of hypoglycemia as demonstrated in the Phase 2 SimpliciT-1 Study are clinically meaningful and that a reduction in events of hypoglycemia would be an acceptable clinical endpoint for evaluation of a therapy for the treatment of type 1 diabetes.

Development of Mechanistic Study of Ketoacidosis. To further support the hypothesis that TTP399 may help reduce the incidence of ketoacidosis, vTv plans to conduct a mechanistic study in a small number of patients with type 1 diabetes to determine the impact of TTP399 on ketone body formation during a period of acute insulin withdrawal. The Company proposed the mechanistic study to the FDA, and the FDA recommended that the study be performed in support of the planned pivotal trials. vTv expects the mechanistic study to be initiated in the first quarter of 2021 and to report topline results in the second quarter of 2021.

Two Oral Presentations at EASD of SimpliciT-1 Study Results. Members of vTv’s TTP399 clinical development team, including Chief Scientific Officer Dr. Carmen Valcarce, presented additional data at the 56th Annual European Association for the Study of Diabetes on the effects of TTP399 on hypoglycemia and ketone body formation from the positive SimpliciT-1 Study.
Dementia with Diabetes

Enrollment concluded for Phase 2 Elevage Study of azeliragon. On September 30, vTv concluded enrollment of patients in the Elevage Study. Forty-three (43) patients with mild probable Alzheimer’s disease and type 2 diabetes were enrolled in the study. The study is designed to evaluate the effect of six-months of treatment with azeliragon on cognitive performance. The Company plans to report top-line results for substantially all of the enrolled patients during December 2020, earlier than previously expected. The objective of the Elevage Study is to replicate in a randomized double-blind, placebo controlled study the results observed in a post hoc analysis of the phase 3 STEADFAST trial A-Study in which a subgroup of forty-seven (47) patients with mild Alzheimer’s disease and type 2 diabetes treated with azeliragon demonstrated nominally statistically significant improvements in cognition on the ADAS-cog11 scale of 5.5 points (p=0.006) at month 18 compared to the same subgroup of patients treated with placebo. Azeliragon associated improvement was nominally significant as early as month 6 on the ADAS-cog11 scale (4.9 points, p<0.001).

Elevage Study Update to be Presented at CTAD. Dr. Ann Gooch presented an update on the Elevage Study focusing on the demographic and baseline characteristics data of enrolled subjects at the 13th Clinical Trials on Alzheimer’s Disease conference on November 4, 2020.
Psoriasis

Multiple Ascending Dose Study with HPP737. The Company is planning a phase 1 multiple ascending dose study to assess the safety, tolerability, and pharmacokinetic profile of HPP737, a PDE4 inhibitor, in healthy volunteers. vTv expects to initiate the study in the first quarter of 2021 and to report topline results in the second quarter of 2021.
Third Quarter 2020 Financial Results

Cash Position: The Company’s cash position as of September 30, 2020, was $1.8 million compared to $6.4 million as of June 30, 2020.

Revenue: Revenues were insignificant for both the second and third quarters of 2020.

R&D Expenses: Research and development expenses were $1.8 million and $2.5 million for the three months ended September 30, 2020 and June 30, 2020, respectively. This decrease of $0.7 million was driven primarily by the reversal of certain performance-based compensation accruals that are no longer expected to be paid.

G&A Expenses: General and administrative expenses were $1.1 million for the third quarter of 2020 and $1.7 million for the second quarter, respectively. The decrease of $0.6 million was driven by the reversal of certain performance-based compensation accruals that are no longer expected to be paid.

Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $2.3 million for the third quarter of 2020 compared to $5.0 million for the second quarter of 2020.

Net Loss Per Share: GAAP net loss per share was $0.03 and $0.07 for the three months ended September 30, 2020 and June 30, 2020, respectively, based on weighted-average shares of 48.2 million and 45.7 million for the three-month periods ended September 30, 2020 and June 30, 2020, respectively. Non-GAAP net loss per fully exchanged share was $0.02 and $0.08 for the three months ended September 30, 2020 and June 30, 2020, respectively, based on non-GAAP fully exchanged weighted-average shares of 71.3 million and 68.8 million for the three months ended September 30, 2020 and June 30, 2020, respectively.

On November 5, 2020 CorMedix Inc. (NYSE American: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory disease, reported financial results for the third quarter and nine months ended September 30, 2020 and provided an update on recent developments (Press release, CorMedix, NOV 5, 2020, View Source [SID1234570083]).

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Recent Corporate and Regulatory Highlights:

CorMedix continues its interactions with the FDA regarding the New Drug Application, or NDA, for Defencath for the prevention of catheter related blood stream infections, or CRBSIs, in patients undergoing hemodialysis via central venous catheter. The FDA has tentatively scheduled the previously announced meeting of the Antimicrobial Drugs Advisory Committee for January 14, 2021 to discuss the Defencath NDA.
Recent changes to the CorMedix Board of Directors include the addition of Paulo Costa and Greg Duncan. These individuals further augment the expertise at the Board level on commercial leadership as well as corporate strategy. Each of Paulo and Greg has had successful careers and leadership roles in the biopharma industry.
CorMedix has continued to expand its efforts to prepare for the commercial launch of Defencath. This includes ongoing dialogue with key payors and dialysis providers and ongoing market research. The interactions have been positive and clearly position CorMedix to ensure that, once Defencath is approved by the FDA, it will be in the best possible position to successfully launch in the US market.
Cash and short-term investments, excluding restricted cash, at September 30, 2020 amounted to $37.0 million. Pro forma cash, including cash on the balance sheet at September 30, 2020 and the net proceeds from recent ATM issuance, is approximately $41.8 million.
Khoso Baluch, CorMedix CEO, commented, "We have continued to make progress on our goal of bringing Defencath to the U.S. market as a catheter lock solution for hemodialysis. We look forward to discussing Defencath with the Antimicrobial Drugs Advisory Committee in January, ahead of the February 28, 2021 PDUFA date for the product. We also are making necessary preparations for the launch of Defencath in the U.S. hemodialysis market, following FDA approval. We believe we have the team, the focus, the resources, and a novel catheter lock solution that will meaningfully improve patient outcomes and are excited about the opportunities in front of us."

Third Quarter and Nine Month 2020 Financial Highlights

For the third quarter of 2020, CorMedix recorded a net loss attributable to common shareholders of $6.6 million, or $0.22 per share, compared with a net loss of $5.3 million, or $0.22 per share, in the third quarter of 2019, excluding the impact of deemed dividends recognized in September 2019 as the result of an exchange agreement and warrant modification.

For the nine months ended September 30, 2020, CorMedix recorded a net loss attributable to common shareholders of $15.9 million, or $0.58 per share, compared with a net loss of $11.1 million, or $0.47 per share, in the first nine months of 2019, excluding the impact of deemed dividends recognized in September 2019 as the result of an exchange agreement and warrant modification. The increase in net loss in the first nine months of 2020 was driven primarily by increased operating expenses.

Operating expenses during the third quarter of 2020 were $6.6 million, compared with $5.2 million in the third quarter of 2019, an increase of approximately 28%. This increase was due to a $0.4 million, or 16%, increase in R&D expense and $1.1 million, or 40%, increase in SG&A expense. Operating expenses during the nine-month period ended September 30, 2020 amounted to $21.2 million compared with $15.6 million during the comparable period in 2019, an increase of $5.6 million, or 36%, due to a 32% increase in R&D expense and 40% increase in SG&A for this period. R&D expense for the first nine months of 2020 included approximately $3.8 million in costs related to the purchase of raw materials and manufacturing of Defencath prior to its potential marketing approval and also included increased staffing costs. Higher SG&A costs were primarily driven by higher staffing costs as well as costs related to market research studies in preparation for the potential market approval of Defencath.

In July 2020, CorMedix completed an underwritten public offering of its common stock, which yielded net proceeds of approximately $21.3 million. The public offering was made pursuant to an underwriting agreement relating to the issuance and sale of an aggregate of 5,111,110 shares of common stock, including 666,666 shares of common stock pursuant to the full exercise of the underwriters’ option, at a public offering price of $4.50 per share. During October 2020, CorMedix issued shares under its at-the-market ("ATM") program and realized net proceeds of approximately $4.6 million.

Total cash on hand and short-term investments as of September 30, 2020 amounted to $37.0 million, excluding restricted cash of $0.2 million. The Company believes that, based on the Company’s cash resources at September 30, 2020, and including the net proceeds received in October 2020 from ATM issuance, it has sufficient resources to fund operations for at least the coming 12 months, including the costs related to the initial preparations for the commercial launch of Defencath.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, November 5, 2020, at 4:30 PM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information is as follows: