On August 20, 2020 Genmab A/S (Nasdaq: GMAB) reported that the U.S. Food and Drug Administration (U.S. FDA) has approved the use of DARZALEX (daratumumab) in combination with carfilzomib and dexamethasone (DKd) for the treatment of adult patients with relapsed/refractory multiple myeloma who have received one to three previous lines of therapy (Press release, Genmab, AUG 20, 2020, View Source [SID1234563905]). A supplemental Biologics License Application (sBLA) for this indication was submitted by Genmab’s licensing partner, Janssen Biotech, Inc. (Janssen), in February 2020. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"We are extremely pleased that multiple myeloma patients in the U.S. will now have yet another treatment option as this is the eighth overall U.S. FDA approval for DARZALEX and the fifth in the relapsed/refractory setting. In addition, DARZALEX is now the first CD38 antibody approved for use in combination with carfilzomib," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The combination has been approved in two carfilzomib dosing regimens, 70 mg/m2 once weekly and 56 mg/m2 twice weekly, based on positive results from the Phase 3 CANDOR and Phase 1b EQUULEUS studies. CANDOR was an Amgen-sponsored study, co-funded by Janssen Research & Development, LLC. EQUULEUS was sponsored by Janssen Research & Development, LLC.

About the CANDOR study
The Phase 3 trial (NCT03158688) was a randomized, open-label study that included 466 patients with multiple myeloma who had relapsed after 1 to 3 prior therapies. Patients were randomized to receive either DKd or carfilzomib and dexamethasone (Kd) alone. In the daratumumab treatment arm, patients received 8 milligrams per kilogram (mg/kg) on days 1 and 2 of cycle 1, then 16 mg/kg once weekly for the remaining doses of the first 2 cycles, then every 2 weeks for 4 cycles (cycles 3 to 6), and then every 4 weeks for the remaining cycles or until disease progression. In both treatment arms carfilzomib was dosed twice weekly (20 mg/m2 on cycle 1 days 1 and 2 and 56 mg/m2 beginning on cycle 1 day 8 and thereafter) and dexamethasone was given weekly (40 mg orally or via IV infusion). The primary endpoint of the study was progression free survival (PFS).

About the EQUULEUS (MMY1001) Study
The Phase 1b EQUULEUS (NCT01998971) study was an open label, multi-cohort trial that evaluated the safety, tolerability, and dose regimen of daratumumab when administered in combination with various treatment regimens for the treatment of multiple myeloma. Among the regiments evaluated, the combination of DKd compared to Kd alone was studied in 85 patients with relapsed/refractory multiple myeloma who had received one to three prior lines of therapy using a once-weekly dosing regimen. DKd was evaluated at a starting dose of 20 mg/m2, which was increased to 70 mg/m2 on Cycle 1, Day 8 and onward.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were expected to be diagnosed with multiple myeloma and approximately 13,650 people were expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

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.6 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 therapy7. 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.8 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).6,9,10,11,12

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 August 20, 2020 Nkarta, Inc. (Nasdaq: NKTX), a clinical-stage biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported financial results for the second quarter ended June 30, 2020, and highlighted recent corporate accomplishments (Press release, Nkarta, AUG 20, 2020, View Source [SID1234563903]).

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"Nkarta made outstanding progress in the second quarter of 2020 as we pursue our goal of advancing engineered natural killer cells as the next foundation in anti-cancer cell therapy," said Paul J. Hastings, President and Chief Executive Officer of Nkarta. "With the success of our recently completed initial public offering and the resultant strengthening of our balance sheet, acceptance of our first IND by the FDA for NKX101, construction of our clinical manufacturing facility, and meaningful strides in our pipeline of allogeneic NK cell therapy candidates, we are off to a momentous start as a public company. We are grateful to our employees and collaborators who have supported this high level of execution in the midst of the difficult challenges posed by the COVID-19 pandemic."

Recent Developments

In March and April 2020, Nkarta appointed two new independent directors to its Board of Directors, Laura K. Shawver, Ph.D., President, Chief Executive Officer and Director of Silverback Therapeutics, and Leone Patterson, President of Adverum Biotechnologies.

Nkarta recently completed the construction of its cGMP manufacturing facility in South San Francisco, California, that is designed to enable Nkarta to supply clinical product for its early-stage clinical trials. Qualification of the facility is in process and Nkarta expects to manufacture NKX019 at this cGMP facility. In a second, final phase of this buildout, Nkarta expects to expand the capabilities of the facility to include the production of retroviral vector, stimulatory cell line and clinical manufacturing of NKX101.

In July 2020, the U.S. Food and Drug Administration cleared Nkarta’s Investigational New Drug Application (IND) for NKX101, an investigational NK cell therapy engineered to target NKG2D ligands.

In July 2020, Nkarta closed the second tranche of its Series B preferred stock financing, with gross proceeds of $64.4 million.

In July 2020, Nkarta completed an initial public offering (IPO) in which it issued and sold 16,100,000 shares of common stock, including full exercise of the underwriters’ over-allotment option, resulting in gross proceeds of $289.8 million, before deducting underwriting discounts and commissions and other offering expenses.
Anticipated Near-term Clinical Milestones

In 4Q 2020, Nkarta plans to begin dosing in a Phase 1 clinical trial of NKX101 for the treatment of relapsed or refractory (r/r) acute myeloid leukemia (AML) and r/r higher risk myelodysplastic syndrome (MDS).

In 1Q 2021, Nkarta expects to file an Investigational New Drug Application (IND) for NKX019, an investigational NK cell therapy engineered to target tumors expressing CD19 antigen for the treatment of B cell malignancies.
Second Quarter 2020 Financial Highlights

Cash and Cash Equivalents: As of June 30, 2020, Nkarta had cash, cash equivalents, restricted cash and short-term investments of $22.1 million, which excludes $54.2 million of the proceeds from the Company’s Series B Second Tranche Financing of $64.4 million which closed on July 1, 2020, as well as the net proceeds from the Company’s July 2020 IPO of $265.5 million.

R&D Expenses: Research & development expenses were $7.9 million for the second quarter of 2020, which includes $0.2 of non-cash stock-based compensation expense.

G&A Expenses: General and administrative expenses were $2.5 million for the second quarter of 2020, which includes $0.3 million of non-cash stock-based compensation expense.

Net Loss. Net loss was $51.1 million, or $30.06 per basic and diluted share, for the quarter ended June 30, 2020. This net loss includes a non-recurring $40.7 million non-cash change in fair value of preferred stock purchase liability.
Financial Guidance

Nkarta expects its current cash and cash equivalents will be sufficient to fund its current operating plan into at least the second half of 2023. The company expects cash and cash equivalents at December 31, 2020 to be in the range of $300 million to $310 million.
Upcoming Events

Executive management plans to participate in a fireside chat at the Cantor Virtual Healthcare Conference on Tuesday, September 15, 2020 at 2:00 PM ET. A webcast of the event will be available on the Investors section of the Nkarta website. http://bit.ly/Nkarta_Events

On August 20, 2020 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary gene engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported an operational update and financial results for the quarter ended June 30, 2020 (Press release, Poseida Therapeutics, AUG 20, 2020, View Source [SID1234563902]).

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"The first half of 2020 marked an important milestone in the growth of Poseida as we raised capital through a Series D preferred stock financing and prepared for our initial public offering," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida. "During our transition to a publicly traded company, we have maintained focus on research innovation and advancement of our clinical programs."

Operational Updates
Series D Financing and Initial Public Offering
In the second quarter, the Company raised net proceeds of $104.1 million in a Series D preferred stock financing led by funds advised by Fidelity Management Research Company, LLC. Following the end of the second quarter, the Company completed an initial public offering (IPO), raising net proceeds of $204.8 million. The capital raised in the Series D financing and IPO put the Company in a strong financial position to advance its clinical trials and pre-clinical studies and continue research and development in the Company’s cell and gene therapy programs.

Completion of Internal GMP Pilot Plant
Construction on the Company’s GMP pilot manufacturing facility adjacent to its San Diego headquarters was completed on schedule. The facility will be used to develop and manufacture preclinical materials and clinical supplies for certain of the Company’s Phase 1 and Phase 2 trials, increasing the Company’s capabilities and flexibility.

Initial Data Presentation from Expanded Phase 1 P-BCMA-101 Clinical Trial
The Company will provide an initial presentation of certain manufacturing improvements and related clinical data for the expanded Phase 1 P-BCMA-101 clinical trial for patients with multiple myeloma with a late breaking abstract by CEO, Eric Ostertag, to be presented at the CAR-TCR Digital Week virtual meeting on September 16, 2020.

Program Updates
P-BCMA-101
The Company’s most advanced product candidate, P-BCMA-101, is an autologous CAR-T therapy which is currently enrolling in an expanded Phase 1 clinical trial for the treatment of patients with relapsed/refractory multiple myeloma to inform the potentially registrational Phase 2 clinical trial.

P-PSMA-101
The Company’s second product candidate, P-PSMA-101, is a solid tumor autologous CAR-T product candidate being developed to treat patients with metastatic castrate resistant prostate cancer and started enrollment in May of 2020. On August 17, 2020 the Company announced that the clinical trial for P-PSMA-101 in metastatic castrate resistant prostate cancer was placed on clinical hold by the FDA following a patient death. The Company’s assessment of the event and evaluation of next steps is ongoing, including working with the FDA toward the objective of resuming the clinical trial.

P-BCMA-ALLO1
The Company’s first allogeneic CAR-T product candidate, P-BCMA-ALLO1 is in development for the treatment of relapsed/refractory multiple myeloma and is designed to be fully allogeneic, with genetic edits to eliminate or reduce both host-vs-graft and graft-vs-host alloreactivity. The program is proceeding with an expected IND filing in late 2020 or early 2021.

P-MUC1C-ALLO1
This allogeneic CAR-T product candidate is in preclinical development for solid tumor indications with the potential to treat a wide range of solid tumors. The program is proceeding with an expected IND filing and initiation of a Phase 1 clinical trial in 2021.

P-OTC-101 Gene Therapy Program
P-OTC-101 is the Company’s first liver-directed gene therapy program for in vivo treatment of urea cycle disease caused by congenital mutations in the OTC gene with a high unmet medical need. The program is proceeding with an expected IND submission in late 2021 or early 2022.

Financial Results
Research and Development Expenses
Research and development expenses were $25.2 million for the three months ended June 30, 2020, compared to $16.9 million for the same period in 2019. For the six months ended June 30, 2020, research and development expenses were $48.6 million, compared to $25.5 million for the same period in 2019. The increase in both periods was primarily due to increased headcount, external costs related to preclinical programs and clinical stage programs, including the ongoing P-BCMA-101 and P-PSMA-101 clinical trials.

General and Administrative Expenses
General and administrative expenses were $4.2 million for the three months ended June 30, 2020, compared to $4.0 million for the same period in 2019, with the increase primarily due to increased headcount. General and administrative expenses were $9.1 million for the six months ended June 30, 2020, compared to $10.4 million for the same period in 2019. The decrease was primarily due to a decrease in facility expense related to lease termination costs.

Net Losses
Net losses were $30.4 million and $59.2 million for the three and six months ended June 30, 2020, respectively, and $28.6 million and $42.0 million for the three and six months ended June 30, 2019, respectively.

Cash Position
As of June 30, 2020, we had cash, cash equivalents and short-term investments of $167.1 million. Net proceeds from the Company’s IPO, which closed in July 2020, were $204.8 million.

On August 20, 2020 Genmab A/S (Nasdaq: GMAB) reported that Novartis intends to transition availability of Arzerra (ofatumumab) to an oncology patient access program that will provide Arzerra at no cost to chronic lymphocytic leukemia (CLL) patients in the U.S (Press release, Genmab, AUG 20, 2020, View Source [SID1234563901]). This program will be facilitated through the Patient Access Novartis Oncology (PANO). As a consequence, Novartis will pay Genmab a lump sum of USD 30 million as payment for lost potential royalties. Arzerra was developed by Novartis under a license agreement between Genmab and Novartis Pharma AG.

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"We are pleased that patients with CLL who have benefitted from Arzerra can remain on treatment via Novartis’ patient access program," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Genmab is also improving its 2020 financial guidance last published on August 12, 2020, due to the inclusion of the payment from Novartis.

OUTLOOK

MDKK Revised Guidance Previous
Guidance
Revenue 9,250 – 9,850 9,100 – 9,700
Operating expenses (3,850) – (3,950) (3,850) – (3,950)
Operating income 5,350 – 5,950 5,200 – 5,800
About Ofatumumab (Arzerra)
Ofatumumab is a human monoclonal antibody that is designed to target the CD20 molecule found on the surface of normal B lymphocytes and on B cell malignancies (including chronic lymphocytic leukemia).

In more than 60 countries worldwide, including the United States and EU member countries, Arzerra was approved as monotherapy for the treatment of patients with CLL who are refractory after prior treatment with fludarabine and alemtuzumab. In the United States, Arzerra was also approved for use in combination with chlorambucil for the treatment of previously untreated patients with CLL for whom fludarabine-based therapy is considered inappropriate, in combination with fludarabine and cyclophosphamide for the treatment of patients with relapsed CLL, for extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive CLL and as monotherapy for the treatment of patients with CLL refractory to fludarabine and alemtuzumab. In the European Union, Arzerra was approved for use in combination with chlorambucil or bendamustine for the treatment of patients with CLL who have not received prior therapy and who are not eligible for fludarabine-based therapy and in combination with fludarabine and cyclophosphamide for adult patients with relapsed CLL. On January 22, 2018, it was announced that Novartis would transition Arzerra for the treatment of CLL indications from commercial availability to limited availability via compassionate use programs in markets outside of the U.S. and Japan. Subsequently, on August 7, 2020, it was announced that Novartis would transition availability of Arzerra in the U.S. to an oncology access program. Novartis obtained rights for ofatumumab from Genmab in all indications in December 2015.

On August 20, 2020 Vivoryon Therapeutics AG (Euronext Amsterdam: VVY, ISIN DE0007921835), reported that it will publish its Half Year Results for 2020 on Thursday, August 27, 2020 (Press release, Vivoryon Therapeutics, AUG 20, 2020, View Source [SID1234563900]). The company will host a conference call and webcast (in English) open to the public. The Half Year Results 2020 will be available to download on the company website (www.vivoryon.com/investors-news/financial-information/).

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Conference call details

Date: Thursday, August 27, 2020

Time: 3:00 pm CEST /09:00 am EDT

Webcast details

A live webcast and accompanying slides will be made available at: www.vivoryon.com/investors-news/financial-information/