On August 5, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the second quarter of 2021 (Press release, Kadmon, AUG 5, 2021, View Source [SID1234585868]).

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"The recent U.S. FDA approval of REZUROCK marked a transformative event for Kadmon and for patients living with cGVHD. REZUROCK represents a paradigm shift in the cGVHD treatment landscape by uniquely addressing both the immune and fibrotic components of the disease," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to bringing this meaningful new therapy to patients in the U.S. by the end of this month."

Dr. Waksal added, "Our momentum continues as we advance our portfolio of product candidates. Initial data from our open-label, Phase 2 trial of belumosudil for the treatment of systemic sclerosis is anticipated by year-end 2021. The recent positive initial safety data presented at ASCO (Free ASCO Whitepaper) on KD033, our anti-PD-L1/IL-15 fusion protein, supports our confidence in the therapeutic potential of IL-15 for cancer. We look forward to sharing additional clinical data from this trial in the fourth quarter of 2021."

2021 Program Updates and Milestones:

REZUROCK (belumosudil)

On July 16, 2021, the U.S. Food and Drug Administration (FDA) approved REZUROCK (belumosudil) for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy:
Commercial launch activities are underway, with a focus on generating awareness of REZUROCK’s differentiated clinical value and facilitating market access
Experienced hematology/oncology sales team and account directors are fully trained and have begun engaging with healthcare providers at the top 100 transplant centers, where ~90% of patients are treated
REZUROCK availability is anticipated by late August through a network of rare hematology/oncology specialty pharmacies and distributors
Kadmon launched Kadmon ASSIST, a comprehensive suite of patient financial and hub support services, including dedicated nurse practitioners on call to facilitate patient education and a successful clinical experience
Kadmon announced on August 4 that the National Comprehensive Cancer Network (NCCN) added REZUROCK tablets to its Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Hematopoietic Cell Transplantation (HCT) in the Pre-Transplant Recipient Evaluation and Management of Graft-Versus-Host Disease in the United States.
Results from the pivotal ROCKstar clinical trial of REZUROCK for the treatment of cGVHD were published in the journal Blood; available online here
Clinical

Belumosudil in SSc (systemic sclerosis)

Present initial data from the open-label Phase 2 clinical trial of belumosudil in patients with SSc (KD025-215) by year-end 2021
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in SSc (KD025-209)
KD033

Enrollment is ongoing in Cohort 4 (100 µg/kg) in the dose-escalation, Phase 1 clinical trial of KD033, Kadmon’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors (KD033-101)
Initial safety data presented at ASCO (Free ASCO Whitepaper) 2021 demonstrated that KD033 has been well tolerated at doses up to 50 µg/kg, with no dose-limiting toxicities reported
The Company plans to present additional clinical data from KD033-101 in the fourth quarter of 2021
Financial Results

Second Quarter 2021 Results

Loss from operations for the three and six months ended June 30, 2021 was $32.0 million and $59.4 million, respectively, compared to $26.9 million and $43.0 million for the same period in 2020. The six months ended June 30, 2020 included $6.0 million in one-time license revenues related to the Meiji strategic partnership.

The $5.6 million and $11.2 million increase in operating expenses for the three and six months ended June 30, 2021, respectively, as compared to 2020 was primarily related to belumosudil commercial launch readiness activities, non-cash stock compensation expenses and direct external research and development costs of developing our preclinical product candidates from our immuno-oncology platform.

Liquidity, Capital Resources and Cash Runway

At June 30, 2021, the Company’s cash, cash equivalents and marketable debt securities totaled $270.5 million, compared to $123.9 million at December 31, 2020. The Company expects its current financial position to be sufficient to fund its operations and capital expenditures into 2023.

About REZUROCK (belumosudil)

REZUROCK (belumosudil) is the first and only approved therapy targeting Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. REZUROCK is approved in the United States for the treatment of adult and pediatric patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy. For more information, visit www.REZUROCK.com.

Kadmon is also developing belumosudil for the treatment of systemic sclerosis. The FDA has granted Orphan Drug Designation to belumosudil for the treatment of systemic sclerosis.

On August 5, 2021 Acceleron Pharma Inc. (Nasdaq:XLRN), a biopharmaceutical company dedicated to the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Acceleron Pharma, AUG 5, 2021, View Source [SID1234585867]).

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"We were very pleased to highlight clinical updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept reported at the annual ATS medical meeting and outline our plans for future long-term growth in rare pulmonary diseases at our Research & Development Day during the second quarter," said Habib Dable, President and Chief Executive Officer of Acceleron. "Beyond our sotatercept clinical program in pulmonary arterial hypertension, most recently, we announced plans to expand the development of sotatercept into a Phase 2 trial in patients with Group 2 pulmonary hypertension and develop ACE-1334 in systemic sclerosis-associated interstitial lung disease. To date, our pulmonary pipeline has grown to include seven ongoing and planned clinical trials, showcasing our long-term commitment to becoming a global leader in rare pulmonary disease."

Added Mr. Dable: "With respect to our hematology program, along with our commercial partner Bristol-Myers Squibb Company (Bristol Myers Squibb), we continue to be pleased with product uptake. Our joint commercial teams are presently focused on expanding the reach to appropriate patients earlier in their MDS journey—where there is particular demand—along with the optimal dose of REBLOZYL to maximize patient benefit and increased duration of treatment in this population to drive further growth this year and beyond. In June, we presented results from the BEYOND Phase 2 trial of luspatercept during the Presidential Symposium at EHA (Free EHA Whitepaper). The study achieved its primary endpoint of an increase in hemoglobin of at least 1 gram per deciliter in the luspatercept treated group compared to placebo for the treatment of anemia in adults with non-transfusion dependent beta-thalassemia, supporting the rationale for its potential development in additional patient groups."

Program Highlights

Pulmonary

Sotatercept: Pulmonary Hypertension
Sotatercept acts as an investigational reverse-remodeling agent proposed to rebalance TGF-beta superfamily signaling. In preclinical models of PAH, sotatercept reversed pulmonary arterial wall and right ventricular remodeling that are hallmarks of the disease.

In May, Acceleron presented updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept in patients with PAH at the American Thoracic Society (ATS) 2021 International Conference.
Enrollment is ongoing in the registrational STELLAR Phase 3 trial in patients with PAH.
Study start up activities are underway for the HYPERION and ZENITH Phase 3 trials of sotatercept in expanded PAH patient populations.
Acceleron expects to initiate the CADENCE Phase 2 trial in patients with pulmonary hypertension with left heart disease this year.
ACE-1334: Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD)
ACE-1334 is an Acceleron-discovered, TGF-beta superfamily-based ligand trap designed to bind and inhibit TGF-beta 1 and 3 ligands but not TGF-beta 2. ACE-1334 has shown robust anti-fibrotic activity in multiple preclinical models of fibrosis.

Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD by year-end 2021.
Hematology

REBLOZYL (luspatercept-aamt):
REBLOZYL is the first and only approved erythroid maturation agent designed to promote late-stage red blood cell (RBC) production. REBLOZYL is part of the global collaboration between Acceleron and Bristol Myers Squibb.

Acceleron recognized approximately $25.6 million in royalty revenue from approximately $128 million in net sales of REBLOZYL in the second quarter of 2021. This compares with approximately $22.4 million in royalty revenue from approximately $112 million in net sales of REBLOZYL in the first quarter of 2021.
In June, the Companies presented results from multiple abstracts on luspatercept at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress.
Results from the BEYOND Phase 2 trial of luspatercept in adult patients with non-transfusion dependent beta-thalassemia were presented during the Presidential Symposium, which honors the top six research papers submitted for presentation at the meeting.
Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with first-line lower-risk MDS, with topline results expected in 2022+.
Enrollment is ongoing in the INDEPENDENCE Phase 3 trial in patients with anemia-associated with myelofibrosis.
Corporate Highlights

Acceleron published its inaugural Environmental, Social, and Governance (ESG) report in June, which provides a comprehensive summary of the Company’s broad range of ESG initiatives. Acceleron has adopted the accounting standards for the biotechnology and pharmaceuticals industry from the Sustainability Accounting Standards Board (SASB) to help develop and prioritize areas for inclusion in its ESG report. The full ESG report can be found on Acceleron’s Investor Relations website by clicking here.
On June 22, 2021, members of the Acceleron leadership team, along with external experts, reviewed the Company’s numerous ongoing and planned trials in rare pulmonary diseases, and highlighted Acceleron’s vision and strategy for long-term growth. An archived recording of the video webcast of the presentations and question and answer sessions is accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com.
Financial Results

Cash Position – Cash, cash equivalents and investments as of June 30, 2021 were $712.5 million, compared with $857.5 million as of December 31, 2020. Based on Acceleron’s current operating plan and projections, the Company believes that its current cash, cash equivalents and investments, along with the expected royalty revenue from REBLOZYL sales, will be sufficient to fund the Company’s projected operating requirements for the foreseeable future.
Revenue – Revenue for the second quarter of 2021 was $27.9 million, which includes $2.3 million of cost share revenue and $25.6 million of royalty revenue from net sales of REBLOZYL. All revenue was derived from the Company’s partnership with Bristol Myers Squibb.
R&D Expenses – GAAP R&D expenses were $56.1 million for the second quarter of 2021. Non-GAAP R&D expenses were $49.3 million for the second quarter of 2021, excluding $5.9 million and $0.9 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
SG&A Expenses – GAAP SG&A expenses were $35.5 million for the second quarter of 2021. Non-GAAP SG&A expenses were $27.8 million for the second quarter of 2021, excluding $7.6 million and $0.1 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Net Loss – The Company’s GAAP net loss for the second quarter of 2021 was $63.5 million, or $1.05 per share. Non-GAAP adjusted net loss for the second quarter was $49.0 million, or $0.81 per share, excluding $13.5 million and $1.0 million in non-cash, stock-based compensation and depreciation and amortization expense, respectively.
Non-GAAP Financial Measures

Acceleron supplements its results of operations prepared in accordance with U.S. generally accepted accounting principles, or GAAP, with certain non-GAAP financial measures, including non-GAAP R&D expense, non-GAAP SG&A expense, adjusted net loss and adjusted net loss per share, that exclude stock-based compensation expense and depreciation and amortization expense. These results should not be viewed as a substitute for the Company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures provide investors with additional insight into underlying trends of the Company’s ongoing business, and are important in comparing current results with prior period results. Investors are cautioned that there are material limitations associated with the use of non-GAAP financial measures. In addition, other companies may report similarly titled non-GAAP measures, but calculate them differently, which reduces their usefulness as a comparative measure. In the reconciliation tables below, Acceleron presents these non-GAAP financial measures reconciled to their comparable GAAP financial measures.

Conference Call and Webcast

The Company will host a webcast and conference call to discuss its second quarter 2021 financial results on August 5, 2021, at 5:00 p.m. EDT.

The webcast will be accessible under "Events & Presentations" in the Investors & Media page of the Company’s website at www.acceleronpharma.com. To participate in the conference call, please dial 833-494-1483 (domestic) or 236-714-2620 (international) and reference code #5736809.

An archived version of the webcast will be available for replay on the Company’s website for approximately one year.

On August 5, 2021 Coherus BioSciences, Inc. ("Coherus" or the "Company", Nasdaq: CHRS), reported financial results for the quarter ended June 30, 2021 (Press release, Coherus Biosciences, AUG 5, 2021, View Source [SID1234585866]). The Company also provided a progress update on its PD-1 blocking antibody, toripalimab, its lead immuno-oncology candidate for the potential treatment of various solid tumors, as well as other late-stage pipeline product candidates including CHS-201, a biosimilar Lucentis (ranibizumab), CHS-1420, a wholly owned biosimilar Humira (adalimumab), and CHS-305, a biosimilar Avastin (bevacizumab).

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"With two biosimilar BLAs currently under FDA review and a toripalimab BLA submission expected to be completed soon, we are making strong progress toward our objective to diversify and grow our product portfolio," said Denny Lanfear, Coherus CEO. "We project that within one year we will have four approved products, including UDENYCA, in the United States, and that in 2023 we will have five marketed products generating revenue to invest in our immuno-oncology business."

SECOND QUARTER 2021 FINANCIAL HIGHLIGHTS

Net product revenue, consisting of net sales of UDENYCA (pegfilgrastim-cbqv) was $88 million.
GAAP net loss of $29.9 million was primarily driven by increased R&D and regulatory expenses to support the advancement of toripalimab and the biosimilar pipeline product candidates.
Non-GAAP net loss was $27.3 million.
At June 30, 2021, Coherus had cash, cash equivalents and marketable securities of $454.4 million.
In April 2021, the Company received $50 million from Junshi Biosciences’ acquisition of 2,491,988 shares of Coherus stock.
PIPELINE HIGHLIGHTS

Coherus is planning an analyst day event in the fourth quarter of 2021

Toripalimab, a PD-1 blocking antibody product candidate, in collaboration with Junshi Biosciences:

Following a recent meeting with the United States Food and Drug Administration ("FDA"), Coherus’ immuno-oncology partner, Junshi Biosciences, plans to submit the biologics license application ("BLA") for toripalimab in combination with chemotherapy for 1st line treatment of metastatic or recurrent nasopharyngeal carcinoma ("NPC") concurrently with toripalimab monotherapy in second or third line treatment of recurrent or metastatic NPC. Junshi Biosciences expects to complete the BLA submission for these indications later this quarter.
Data from a Phase 3 clinical trial evaluating toripalimab for the treatment of non-small cell lung cancer will be presented in September at the World Conference on Lung Cancer.
Data from a Phase 3 clinical trial evaluating toripalimab for the treatment of esophageal squamous cell carcinoma will be presented in September at the annual meeting of the European Society for Medical Oncology.
CHS-201, a biosimilar Lucentis (ranibizumab) product candidate in collaboration with Bioeq AG:

Bioeq AG recently submitted the CHS-201 BLA. Pending acceptance of the BLA by the FDA, Coherus anticipates a mid-2022 target action date for the BLA review.
CHS-1420, a wholly owned biosimilar Humira (adalimumab) product candidate:

The review of the CHS-1420 BLA is progressing with a target action date of December 2021. Coherus plans to launch CHS-1420 on or after July 1, 2023, if approved.
CHS-305, a biosimilar Avastin (bevacizumab) product candidate in collaboration with Innovent Biologics (Suzhou) Co. Ltd:

Coherus is conducting the three-way pharmacokinetic study to facilitate the potential CHS-305 BLA submission.
SECOND QUARTER 2021 FINANCIAL RESULTS

Net product revenue, consisting of net sales of UDENYCA, was $87.6 million and $135.7 million during the three months ended June 30, 2021 and 2020, respectively, and $170.7 million and $251.9 million during the six months ended June 30, 2021 and 2020, respectively.

Cost of goods sold (COGS), increased to $16.7 million in the second quarter of 2021 as compared to $10.1 million in second quarter of 2020. Until the first quarter of 2021, Coherus sold inventory that was manufactured and expensed prior to the approval of UDENYCA in late 2018. This inventory was depleted in the first quarter of 2021, and the second quarter of 2021 is the first period with per unit acquisition costs fully reflected within COGS. UDENYCA COGS also includes a mid single digit royalty on net sales payable through the first half of 2024.

Research and development (R&D) expense for the three months ended June 30, 2021 was $54.8 million compared to $26.2 million for the same period in 2020, an increase of $28.6 million. The increase was mainly due to higher development and regulatory costs in support of the advancement of toripalimab and the biosimilar pipeline product candidates. For the six months ended June 30, 2021, R&D expense was $258.3 million compared to $59.3 million for the same period in 2020, an increase of $199.0 million which included the $136.0 million upfront license fee paid to Junshi Biosciences in 2021.

Selling, general and administrative (SG&A) expense for the three months ended June 30, 2021 was $40.3 million compared to $34.1 million for the three months ended June 30, 2020, an increase of $6.3 million which was primarily driven by increased UDENYCA commercialization expenses including an increase in sales personnel and travel. For the six months ended June 30, 2021, SG&A expense was $79.7 million compared to $69.4 million for the same period in 2020, an increase of $10.3 million, which was primarily due to an increase of $5.8 million in stock-based compensation expense and a $4.1 million increase in UDENYCA commercialization expenses.

Net loss for the second quarter of 2021 was $29.9 million, or $0.40 per share on a diluted basis, compared to a net income of $59.0 million, or $0.70 per share on a diluted basis for the same period in 2020.

Non-GAAP net loss for the second quarter of 2021 was $27.3 million, or $0.36 per share on a diluted basis, compared to non-GAAP net income of $68.3 million, or $0.81 per share on a diluted basis for the same period in 2020. See "Non-GAAP Financial Measures" below for a discussion on how Coherus calculates non-GAAP net (loss) income and a reconciliation to the most directly comparable GAAP measures.

Cash, cash equivalents and investments in marketable securities were $454.4 million as of June 30, 2021, compared to $399.5 million at March 31, 2021.

2021 FINANCIAL OUTLOOK

Excluding the upfront payment made to Junshi Biosciences in the first quarter, Coherus projects full year 2021 R&D and SG&A expenses in a range of $370 million to $400 million. R&D spending is focused on development, regulatory and other activities in preparation for the potential launch of toripalimab, as well as manufacturing-related and regulatory activities for CHS-1420, development activities for CHS-305, and additional presentations of UDENYCA. Increases in SG&A spending in 2021 are primarily driven by marketing activities and headcount to support UDENYCA and the potential launches in 2022 of toripalimab and CHS-201 (Lucentis biosimilar).

This financial guidance excludes the effects of any potential future strategic acquisitions, collaborations or investments, the exercise of rights or options related to collaboration programs, and any other transactions or items not yet identified or quantified. This guidance is subject to a number of risks and uncertainties. See Forward-Looking Statements described in the section below and the section titled "Risk Factors" in the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 to be filed with the Securities & Exchange Commission on August 5, 2021.

On August 5, 2021 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal third quarter ended June 30, 2021 (Press release, Arrowhead Research Corporation, AUG 5, 2021, View Source [SID1234585865]). The company is hosting a conference call today, August 5, 2021, at 4:30 p.m. ET to discuss the results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 7398304.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 7398304.

Selected Recent Events

Received Breakthrough Therapy designation from the U.S. Food and Drug Administration for ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency.
Presented additional positive interim 48-week liver biopsy results from the ongoing AROAAT2002 study, an open-label Phase 2 clinical study of ARO-AAT, at The International Liver Congress – The Annual Meeting of the European Association for the Study of the Liver (EASL). The results demonstrate that investigational ARO-AAT treatment led to improvements in multiple measures of liver health, including fibrosis, with substantial and sustained reductions in the level of mutant AAT protein. In addition, ARO-AAT treatment was generally well tolerated after up to 1 year of treatment.
Presented positive interim results from AROHSD1001, an ongoing Phase 1/2 clinical study of ARO-HSD, the company’s investigational RNAi therapeutic being developed as a treatment for patients with alcohol-related and nonalcohol related liver diseases, such as nonalcoholic steatohepatitis (NASH), at EASL. The data demonstrate that ARO-HSD is the first investigational therapeutic to achieve robust reductions in messenger RNA and protein levels of hepatic HSD17B13, leading to reductions in alanine aminotransferase (ALT), a liver enzyme typically elevated in liver diseases including NASH.
Announced positive interim results from the first two cohorts of AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNAi therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma. The data show clear signs of meaningful target engagement and some potentially early signs of efficacy in at least one patient.
Initiated and began dosing patients in AROANG3-2001, a Phase 2b clinical study of ARO-ANG3, the company’s investigational RNAi therapeutic being developed as a treatment for patients with mixed dyslipidemia.
Initiated and began dosing patients in AROAPOC3-2001, a Phase 2b clinical study of ARO-APOC3, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with severe hypertriglyceridemia (SHTG). Arrowhead also intends to initiate a Phase 2b study and a Phase 3 study of ARO-APOC3 in two additional patient populations in 2021.
Announced a global collaboration and license agreement with Horizon Therapeutics for ARO-XDH, a previously undisclosed discovery-stage RNAi therapeutic being developed by Arrowhead as a potential treatment for people with uncontrolled gout. Arrowhead received $40 million as an upfront payment from Horizon and is eligible to receive up to $660 million in potential development, regulatory and commercial milestones, and is further eligible to receive royalties in the low- to mid-teens range on net product sales.
Earned a $10 million option exercise fee from Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, for ARO-JNJ1.
Presented promising preclinical data on ARO-DUX4, Arrowhead’s first muscle-targeted program being developed as a treatment for patients with facioscapulohumeral muscular dystrophy (FSHD) at the 28th Annual FSHD Society International Research Congress. The data show that the TRiMTM muscle delivery platform achieved functional delivery to various types of skeletal muscle and achieved deep, durable, and dose-dependent knockdown of target genes. In addition, ARO-DUX4 improved multiple measures of FSHD-like muscle phenotype in relevant preclinical animal models.
Nominated ARO-C3, which is designed to reduce production of complement component 3 (C3) as a potential treatment for various complement mediated diseases, as a clinical candidate and initiated IND-enabling toxicology studies.

On August 5, 2021 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor-infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported second quarter 2021 financial results and corporate updates (Press release, Iovance Biotherapeutics, AUG 5, 2021, View Source [SID1234585863]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "During the first half of 2021 we advanced our TIL pipeline and presented clinical data across multiple solid tumor indications and treatment settings, including single-agent TIL in metastatic non-small cell lung cancer and melanoma, as well as initial clinical data for TIL in combination with pembrolizumab in early line melanoma. Our top priority remains our ongoing work to address FDA feedback regarding the potency assays for lifileucel to support our planned BLA submission. We are increasingly confident in the broad potential for TIL as the next class of paradigm-shifting therapy for cancer patients with significant unmet need."

Second Quarter 2021 Highlights and Recent Corporate Updates

Regulatory

Potency assays for lifileucel: Following FDA feedback regarding the potency assays for lifileucel, Iovance will continue ongoing work developing and validating its potency assays and plans to submit additional assay data and anticipates meeting with the FDA before the end of 2021. The company’s biologics license application (BLA) submission for lifileucel is now expected to occur during the first half of 2022. Resolution of the potency assay for lifileucel in melanoma is also a key step towards our regulatory plans in other indications.
Clinical

TIL therapy in melanoma:
Metastatic melanoma: follow up data from Cohort 2 in the C-144-01 study of lifileucel in advanced melanoma were presented at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting. As of the April 2021 data cutoff for the presentation, the overall response rate (ORR) was 36.4% (4.5% complete response rate and 31.8% partial response rate) and median duration of response (DOR) was not reached at 33.1 months of median study follow up as assessed by investigators (n=66). Detailed Cohort 2 data were also published in a manuscript in the Journal of Clinical Oncology, an ASCO (Free ASCO Whitepaper) journal.
Anti-PD-1 naïve melanoma: initial clinical data for lifileucel in combination with pembrolizumab were presented in a poster at ASCO (Free ASCO Whitepaper) 2021. The ORR was 86% and the complete response rate was 43% at a median follow up of 8.2 months in anti-PD-1 naïve melanoma patients in Cohort 1A in the IOV-COM-202 basket study (n=7).
TIL therapy in non-small cell lung cancer (NSCLC):

LN-145 clinical data in metastatic NSCLC (mNSCLC): clinical data for LN-145 showed a 21.4% ORR and 64.3% disease control rate in mNSCLC patients from Cohort 3B in the IOV-COM-202 study (n=28), including two responders with PD-L1 negative tumors. All Cohort 3B patients had received one or more prior systemic therapies, including anti-PD-1 therapy, and all responders also received prior chemotherapy. Detailed results are anticipated at a medical meeting in 2021.
LN-145 in second-line mNSCLC: the first patient was dosed and more than 15 U.S. clinical sites have been activated in the registration-supporting IOV-LUN-202 study of LN-145 in patients with mNSCLC.
Research

Iovance is committed to advancing the next generation of TIL and related therapies and technologies. Late preclinical programs in IND-enabling studies include a novel IL-2 analog (IOV-3001) as well as a genetically modified TIL (IOV-4001). IOV-4001 leverages TALEN technology licensed from Cellectis S.A. to genetically knock out PD-1 in TIL cells.
Manufacturing

TIL manufacturing success: to date, nearly 500 patients have been dosed with Iovance TIL products with more than a 90 percent manufacturing success rate.
Iovance Cell Therapy Center (iCTC): the investigational new drug (IND) application amendment has been cleared and clinical manufacturing of TIL is expected to commence at the iCTC in the near future. Commercial manufacturing remains on track to commence with a potential regulatory approval.
Corporate

Cash position of $708.7 million on June 30, 2021 is expected to be sufficient well into 2023.
A strong organization of nearly 270 employees with an average of more than 3.5 years of cell therapy experience is in place to advance research, development, manufacturing, and commercial launch preparations.
Iovance continues to expand its intellectual property portfolio and currently owns more than 25 granted or allowed U.S. and international patents for compositions and methods of treatment in a broad range of cancers relating to the Gen 2 manufacturing process. Iovance’s Gen 2 patent rights are expected to provide exclusivity through 2038. Iovance’s portfolio also includes patent applications and granted patents directed towards Gen 3 manufacturing, selected TIL products, stable and transient genetic TIL modifications, tumor digest and fragment compositions and methods (including cryopreservation), and combinations of checkpoint inhibitors and TIL products.
Second Quarter and First Half 2021 Financial Results

Iovance held $708.7 million in cash, cash equivalents, investments and restricted cash at June 30, 2021 compared to $635.0 million at December 31, 2020. The cash position as of the second quarter is expected to be sufficient for more than two years based on the current operating plan.

Jean-Marc Bellemin, Chief Financial Officer, stated, "Our balance sheet remains strong to advance our operating plan, including launch preparations and pipeline development, with no immediate need to raise additional capital."

Net loss for the second quarter ended June 30, 2021, was $81.4 million, or $0.53 per share, compared to a net loss of $63.0 million, or $0.47 per share, for the second quarter ended June 30, 2020. Net loss for the six months ended June 30, 2020, was $156.8 million, or $1.04 per share, compared to a net loss of $132.6 million, or $1.02 per share, for the same period ended June 30, 2020.

Research and development expenses were $62.1 million for the second quarter ended June 30, 2021, an increase of $12.8 million compared to $49.3 million for the second quarter ended June 30, 2020. Research and development expenses were $118.1 million for the six months ended June 30, 2021, an increase of $11.8 million compared to $106.2 million for the same period ended June 30, 2020.

The increase in research and development expenses in the second quarter 2021 over the prior year period was primarily attributable to an increase in costs associated with growth of the internal research and development team and increases in manufacturing and iCTC facility related costs. The increase in research and development expenses in the first half of 2021 over the prior year period was primarily attributable to growth of the internal research and development team, an increase in iCTC facility related costs, which were partially offset by lower manufacturing and clinical costs following the completion of enrollment in the pivotal cohorts for melanoma and cervical cancer.

General and administrative expenses were $19.3 million for the second quarter ended June 30, 2021, an increase of $5.0 million compared to $14.4 million for the second quarter ended June 30, 2020. General and administrative expenses were $38.9 million for the six months ended June 30, 2021, an increase of $10.7 million compared to $28.2 million for the same period ended June 30, 2020.

The increases in general and administrative expenses in the second quarter and first half of 2021 compared to the prior year periods were primarily attributable to growth of the internal general and administrative team and higher stock-based compensation expenses.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss the second quarter 2021 financial results and corporate updates. The conference call dial-in numbers are 1-(844) 646-4465 (domestic) or 1-(615) 247-0257 (international) and the access code is 1489438. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.