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 [SID1234585853]).

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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.

On August 5, 2021 Vaxart, Inc. (Nasdaq: VXRT) reported its business update today for the second quarter of 2021, reporting strong forward momentum in development of oral tablet vaccines that it believes can revolutionize public health (Press release, Aviragen Therapeutics, AUG 5, 2021, View Source [SID1234585870]).

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Vaxart, a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, announced its business progress and provided financial results for the second quarter ended June 30, 2021.

"By any measure, Vaxart showed real forward momentum during the last quarter," CEO Andrei Floroiu said. "We strengthened our balance sheet, made major research advances, deepened our scientific knowledge, grew the company, and took the company closer to our goal of developing disruptive oral tablet vaccines that can have a material impact on the world’s public health."

Recent Business Highlights

Corporate Developments

Vaxart raised $36.2 million in net proceeds from its $250 million at-the-market facility in the three months ended June 30, 2021.
Vaxart signed an exclusive worldwide licensing agreement with Altesa Biosciences, Inc. to allow Altesa to develop and commercialize Vaxart’s Vapendavir, a clinical-stage broad spectrum antiviral.
The licensing agreement provides for milestone payments up to $130 million and royalties for global Vapendavir sales.
Building out the infrastructure necessary to support its groundbreaking research, Vaxart added to its management team and strengthened its research, clinical, and manufacturing groups and R&D infrastructure. The number of R&D employees grew by 36% in the quarter to 49 full-time employees.
Pre-Clinical and Clinical

Platform-Wide Developments

Vaxart completed its first boosting study, which showed in clinical trials that its norovirus vaccine built on the VAAST platform can successfully boost immune responses in subjects previously vaccinated with a Vaxart oral vaccine more than a year earlier.
The research supports the company’s thesis that its oral tablet vaccines have the potential to be used annually for indications that may require a boost such as flu or COVID-19.
The data announced that Vaxart’s vaccines may not suffer from certain antibody response challenges that can occur with viral vector-based vaccines.
COVID-19 Vaccine Developments

While the nation’s death and illness tolls have fallen markedly since the beginning of the last quarter, new variants such as the Delta strain continue to worry national political and health leaders. Vaxart broadened its research into the various COVID-19 strains while continuing its development of an oral tablet vaccine. Among the most significant developments in the second quarter:

The U.S. Food and Drug Administration cleared Vaxart to move to its next phase of COVID-19 testing with a study of its next generation S-1 construct.
Vaxart is manufacturing the vaccine necessary to start the Phase 2 clinical study of its S-Wuhan construct and expects to begin this study shortly.
A Non-Human Primate study of the S&N construct along with S-Wuhan and S-South Africa constructs showed optimal performance by the S-Wuhan construct and also cross reactivity against all variants tested. The decision was made to the S-Wuhan vaccine construct into Phase 2.
Norovirus Vaccine Developments

Norovirus is a highly infectious illness that affects around 20 million Americans annually, with an annual economic impact of approximately $10.5 billion in the United States.

In addition to its boosting study, Vaxart enrolled the first subjects in a Phase 1b placebo-controlled, dose-ranging, repeat dose trial investigating its oral norovirus vaccine candidate in elderly subjects aged 55 – 80 years.
This study is designed to evaluate the safety and immunogenicity of Vaxart’s candidate, which is the only clinical stage norovirus oral tablet vaccine actively being developed.
Financial Results for the Three Months Ended June 30, 2021

Vaxart ended the quarter with cash, cash equivalents, and available-for-sale debt securities of $198.9 million, compared to $177.3 million as of March 31, 2021. The increase was primarily due to net receipts of $36.2 million from the Company’s $250 million at-the-market facility entered into in October 2020 and $0.9 million from the exercise of warrants and options, partially offset by $13.2 million of cash used in operations and $2.2 million spent on property and equipment.
Vaxart reported a net loss of $16.1 million for the second quarter of 2021 compared to $9.0 million for the second quarter of 2020. Net loss per share for the second quarter of 2021 was $0.13, compared to a net loss of $0.12 in the second quarter of 2020. The small increase in net loss per share was due to the increase in net loss being mostly offset by the increase in the weighted average number of shares outstanding.
Revenue for the second quarter of 2021 was $112,000 compared to $523,000 in the second quarter of 2020. The decrease was principally due to the absence of royalty revenue related to Relenza sales in Japan as a result of the patent expiring and a reduction in royalty revenue related to Inavir sales in Japan as a result of lower incidences of seasonal influenza.
Research and development expenses were $10.7 million for the second quarter of 2021 compared to $5.1 million for the second quarter of 2020. The increase was mainly due to manufacturing and clinical trial expenses related to the COVID-19 and norovirus vaccine candidates.
General and administrative expenses were $5.2 million for the second quarter of 2021 compared to $3.9 million for the second quarter of 2020. The increase was mainly due to higher insurance expenses and an increase in headcount and related costs.

On August 5, 2021 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Harpoon Therapeutics, AUG 5, 2021, View Source [SID1234585885]).

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"The clinical data emerging for our proprietary TriTAC portfolio continues to be encouraging as HPN424, HPN536 and HPN328 have shown cancer target engagement, significant treatment duration, and either tumor size reductions or stable disease," said Jerry McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "Looking ahead into to the second half of 2021, we remain focused on increasing dose levels across all four programs and providing additional interim clinical pipeline data by year end."

Second Quarter 2021 Business Highlights and Other Recent Developments

•In June 2021, Harpoon presented interim clinical data from the ongoing dose-escalation portion of the Phase 1/2a trial for HPN424—a TriTAC targeting prostate-specific membrane antigen (PSMA)—in patients with metastatic castration-resistant prostate cancer (mCRPC) at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. At the time of the ASCO (Free ASCO Whitepaper) presentation data cutoff, 89 patients had been treated in 13 cohorts. Key findings include:
•HPN24 was active and generally well-tolerated.
•Antitumor activity included a confirmed PR per RECIST, PSA declines and circulating tumor cell reductions.
•Cytokine release syndrome (CRS) has been transient and manageable with 4% of patients experiencing Grade 3 CRS.
•CRS events observed most often in Cycle 1, with diminished frequency and severity in subsequent cycles.
•Introduction of step dose regimens has allowed for the administration of higher target doses, which had reached 300ng/kg at the time of the update.
•Expanding on the data presented at ASCO (Free ASCO Whitepaper), with a May 31 data cutoff date, 19 patients had been enrolled in the 300 ng/kg step dose cohort, which includes patients treated with different step regimens to obtain the target dose of 300 ng/kg. Four of 19 patients in this cohort showed PSA declines, including two PSA30.

Dose escalation is ongoing, and we have recently opened a 450 ng/kg step dose cohort. The first patient has successfully reached the target dose of 450 ng/kg.

•In June 2021, Harpoon provided a corporate pipeline update. In addition to reiterating and expanding upon HPN424 data presented at ASCO (Free ASCO Whitepaper)21, Harpoon provided updates on the following TriTAC clinical programs:
•HPN 536 (mesothelin TRiTAC) – Phase 1/2a clinical trial continues dose escalation for treating late-stage ovarian, pancreatic, and peritoneal and pleural mesothelioma cancers. Findings demonstrated that 11 of 20 relapsed/refractory ovarian cancer patients showed stability of target lesions, including three with target lesion shrinkage. Additionally, five of 27 ovarian cancer patients had a duration of treatment of greater than 24 weeks. As of the May 31, 2021 data cutoff date, two DLTs had been observed, which did not limit escalation. An MTD has not been identified and escalation to higher doses is underway. We recently opened and are actively recruiting a 1800 ng/kg cohort.
•HPN 217 (BCMA TriTAC) – Dose escalation for Phase 1/2 clinical trial is progressing. Relapsed/refractory multiple myeloma patients (N=20) have been treated across eight fixed dose cohorts of 5 to 2150 µg weekly, reflecting rapid dose expansion since the trial began, and HPN 217 has been well tolerated. Pharmacokinetic analysis shows half-life extension to support at least once weekly dosing.

•HPN328 (DLL3 TriTAC) – Dose escalation for Phase 1/2 clinical trial initiated in late 2020 and has shown rapid progress. The first single patient cohort began with a flat dose of 15µg of HPN328 administered once weekly by intravenous infusion and has proceeded to the fourth cohort at a dose of 405µg. Eligible patients include small cell lung cancer patients who have relapsed after platinum chemotherapy and patients with other tumors associated with DLL3 expression. One SCLC patient previously treated with chemotherapy enrolled in the 45µg cohort, demonstrated a 38% reduction in target lesion, consistent with an unconfirmed partial response. A subsequent scan indicated stable disease as best response. MTD has not been identified and escalation to higher doses is underway. We recently opened a 3.6 mg flat dose cohort and have successfully treated the first patient in that dose cohort.

•In addition, Harpoon provided an update on HPN601 (EpCAM ProTriTAC), a conditionally active T cell engager. IND-enabling studies for HPN601 are progressing as planned. EpCAM is expressed in a broad range of solid tumors, including gastrointestinal cancers, potentially enabling HPN601 to address multiple indications with high unmet medical need.

Second Quarter 2021 Financial Results

•Harpoon ended the second quarter of 2021 with $175.2 million in cash, cash equivalents, and marketable securities compared to $150.0 million as of December 31, 2020. The cash balance at the end of the second quarter includes Harpoon’s follow-on financing that closed on January 11, 2021 resulting in net proceeds of approximately $107.6 million.

•Revenue for the second quarter ended June 30, 2021 was $5.8 million compared to $2.8 million for the quarter ended June 30, 2020. For the six months ended June 30, 2021, revenue was $14.8 million compared to $6.1 million for the six months ended June 30, 2020. For the second quarter ended June 30, 2021, the increase in revenue was primarily due to an increase in revenue recognized related to Harpoon’s Development and Option Agreement with AbbVie, for research and development services performed. For the six months ended June 30, 2021,the increase in revenue was primarily due to an increase in revenue recognized due to the delivery of the second initial target under Harpoon’s Amended and Restated Discovery Collaboration Agreement with AbbVie, where all remaining deferred revenue associated with that target was recognized as we had no further continuing performance obligations, as well as an increase in revenue recognized related to Harpoon’s Development and Option Agreement with AbbVie, for research and development services performed.

• Research and development expense for the second quarter ended June 30, 2021, was $18.3 million compared to $11.9 million for the quarter ended June 30, 2020. For the six months ended June 30, 2021, R&D expense was $34.5 million compared to $24.4 million for the six months ended June 30, 2020. The increase for both periods, primarily arose from higher clinical development and personnel-related expense, which included conducting preclinical studies and the continuation and preparation of the clinical trials for HPN424, HPN536, HPN217 and HPN328.

• General and administrative expense for the second quarter ended June 30, 2021 was $4.3 million compared to $3.9 million for the quarter ended June 30, 2020. For the six months ended June 30, 2021, G&A expense was $8.9 million compared to $7.9 million for the six months ended June 30, 2020. The increase for both periods was primarily attributable to an increase in personnel-related expenses due to an increase in headcount and other professional services to support Harpoon’s operations as a public company.

•Net loss for the second quarter ended June 30, 2021 was $16.8 million compared to $12.7 million for the quarter ended June 30, 2020. The net loss for the six months ended June 30, 2021 was $78.5 million compared to $25.2 million in the first six months of the prior year.

Anticipated 2021 Milestones

•HPN424 – initiate a dose expansion cohort of the Phase 1/2a trial by year end 2021
•HPN536 – present interim data from the dose escalation phase of the trial by year end 2021 and initiate a dose expansion cohort of the ongoing Phase 1/2 trial in the second half of the year
•HPN217 – present interim data from the dose escalation phase of the trial by year end 2021 and initiate a dose expansion cohort of the ongoing Phase 1/2 trial in the second half of the year
•HPN328 – present interim data from the dose escalation phase of the trial by year end 2021
COVID-19 Business Update

In response to the ongoing COVID-19 pandemic, Harpoon has established testing and other protocols for personnel access to its headquarter offices and laboratory although the majority of the company’s employees continue to telecommute. Harpoon is currently continuing its clinical trials, and has not yet experienced any material delays or impacts as a result of the COVID-19 pandemic. In addition, Harpoon’s third-party contract manufacturers continue to operate at or near normal levels. Harpoon continues to assess the potential impact of the COVID-19 pandemic on its business and operations, including its programs, expected timelines, expenses, manufacturing activities and preclinical and clinical trials. The full extent to which the COVID-19 pandemic may have a negative impact on Harpoon’s business, assets, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted.

On August 5, 2021 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the second quarter ended June 30, 2021 and provided recent corporate updates (Press release, Bicycle Therapeutics, AUG 5, 2021, View Source [SID1234585901]).

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"We have made significant progress executing on our ongoing company-sponsored clinical programs over the first half of the year and, looking forward, intend to announce interim clinical data from the Phase I portion of our Phase I/II clinical trials of BT5528 and BT8009, along with the expected Phase I/II BT7840 trial initiation, in the second half of the year," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "Additionally, we believe our recent license and collaboration agreement with Ionis provides further validation for our contention that Bicycles have the potential to become a leading technology for the development of precision medicines. We remain well-funded and look forward to announcing multiple upcoming key clinical milestones in the second half of the year."

Second Quarter 2021 and Recent Highlights

Entered into an Exclusive License and Collaboration Agreement with Ionis Pharmaceuticals to Develop Targeted Oligonucleotide Therapeutics. In July 2021, Ionis exercised its option under the terms of a December 2020 evaluation and option agreement and entered into an exclusive worldwide license and collaboration agreement for tissue-targeted delivery of oligonucleotide therapeutics using Bicycles with high affinity to the transferrin receptor (TfR1). Bicycle received $45 million upfront, which included a license fee, an option fee, and an $11 million equity investment. Bicycle is also eligible to receive development, regulatory and commercial milestone payments and royalties for programs developed under the collaboration.
Continued to Strengthen the Balance Sheet in the Second Quarter of 2021. The Company recognized gross proceeds from Bicycle’s at-the-market (ATM) offering program during the second quarter of 2021 totaling $14.4 million as of June 30, 2021, with an additional $30.8 million received in July 2021. Cash as of June 30, 2021 does not include $42 million of proceeds from the Ionis license and collaboration agreement or net proceeds from the ATM offering program received subsequent to the end of the second quarter of 2021.
Financial Results

Cash was $198.7 million as of June 30, 2021, compared to $136.0 million as of December 31, 2020. The increase in cash during the first six months of 2021 is primarily due to net proceeds of $72.8 million from the ATM offering program and net proceeds of $15.0 million from the debt facility with Hercules Capital, Inc., offset by cash used in operating activities. Cash of $198.7 million at June 30, 2021 is expected to provide financial runway through multiple clinical milestones and into 2024.
Research and development expenses were $11.7 million for the three months ended June 30, 2021, compared to $8.0 million for the three months ended June 30, 2020. The increase in expense of $3.7 million for the three months ended June 30, 2021 as compared to the same period in the prior year was primarily due to increased clinical program expenses for BT8009, a second-generation Bicycle Toxin Conjugate (BTC) targeting Nectin-4, increased other unallocated discovery and platform related expenses due to the timing of development activities, and increased personnel-related expenses, including $0.6 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $7.3 million for the three months ended June 30, 2021, compared to $6.2 million for the three months ended June 30, 2020. The increase of $1.1 million for the three months ended June 30, 2021 as compared to the same period in the prior year was primarily due to an increase in personnel-related costs, including $0.7 million of incremental non-cash share-based compensation expense, offset by a decrease in professional and consulting fees.
Net loss was $17.9 million, or $(0.74) basic and diluted net loss per share, for the three months ended June 30, 2021, compared to net loss of $12.1 million, or $(0.67) basic and diluted net loss per share for three months ended June 30, 2020.

On August 5, 2021 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases with significant unmet needs, reported financial results for the second quarter ended June 30, 2021 and provided a business update (Press release, Protara Therapeutics, AUG 5, 2021, View Source [SID1234585916]).

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"We continue to make significant progress executing against our development plans for TARA-002 in non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs), and we look forward to achieving multiple important milestones in the second half of this year," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "With the successful completion of required non-clinical studies for TARA-002, we remain on track to submit our Investigational New Drug (IND) application and initiate our Phase 1 clinical study by year-end. In addition, our Good Manufacturing Practice (GMP) scale up and comparability work remain on track to be completed by year-end, and we look forward to working with the U.S. Food and Drug Administration (FDA) on a clinical trial of TARA-002 in patients with LMs, a rare pediatric indication for which there are currently no U.S. FDA-approved therapies."

Recent Highlights and Upcoming Milestones

TARA-002 Comparability

The Company remains on track to complete confirmatory, large-scale GMP manufacturing comparability by year-end.
TARA-002 in NMIBC

Protara successfully completed required non-clinical IND-enabling studies to characterize local toxicity of intravesical administration of TARA-002. The Company remains on track to submit an IND application in the second half of 2021 and, subject to the FDA acceptance of the IND application, the Company plans to commence a Phase 1 trial by the end of 2021 to assess the safety and tolerability of TARA-002 in patients with NMIBC, including patients with carcinoma in situ (CIS).
TARA-002 in LMs

Following the completion of confirmatory, large-scale GMP manufacturing comparability, the Company plans to align with the FDA on the design, and subsequently initiate a clinical trial in pediatric LM patients.
IV Choline Chloride in Intestinal Failure Associated Liver Disease (IFALD)

The Company is currently executing a prevalence study in partnership with a large home health organization in the U.S. to enhance understanding of the appropriate patient population and will use this information to define the next steps for the development program.
Corporate Update

In June 2021, the Company announced the appointment of Jane Huang, M.D., to its Board of Directors. Dr. Huang is an experienced biotech executive and proven leader throughout the development lifecycle of multiple oncology therapeutics globally and currently serves as Chief Medical Officer, Hematology at BeiGene, Ltd.
Second Quarter 2021 Financial Results

As of June 30, 2021, cash, cash equivalents and investments totaled $145 million.

Research and development expenses for the second quarter of 2021 increased to $5.9 million from $2.5 million during the second quarter of 2020. The increased R&D expenses were primarily due to increases in manufacturing and regulatory expenses associated with TARA-002.

General and administrative expenses for the second quarter of 2021 increased to $6.9 million from $4.8 million during the second quarter of 2020. The increase was primarily due to increases in stock-based compensation, headcount, and costs associated with the new Company headquarters in New York, NY.

For the second quarter of 2021, Protara reported a net loss of $12.8 million, or $1.14 per share, compared with a net loss of $7.1 million, or $1.22 per share, for the second quarter of 2020. Net loss for the second quarter of 2021 included approximately $3.0 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully demonstrated initial manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-6, IL-8, IL-12, interferon (IFN)-gamma, tumor necrosis factor (TNF)-alpha, and vascular endothelial growth factor (VEGF) are secreted by immune cells to induce a strong local inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the 6th most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations

Lymphatic malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride and Intestinal Failure-associated Liver Disease (IFALD)

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN) who have IFALD. Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. If approved, IV Choline Chloride would be the first approved therapy for IFALD. It has been granted Orphan Drug Designations (ODDs) by the FDA for the treatment of IFALD and the prevention of choline deficiency in PN patients.