On November 4, 2021 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported that its third quarter 2021 financial results will be released after the markets close on Wednesday, November 10th (Press release, DiaMedica, NOV 4, 2021, View Source [SID1234594497]). DiaMedica will host a live conference call on Thursday, November 11th at 7:00 AM Central Time to discuss its business update and financial results.

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

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

                  Schedule Your 30 min Free Demo!

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on our website, under investor relations – events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until November 18, 2021, by dialing (800) 770-2030 (US Toll Free) and entering the replay passcode: 4814247.

On November 4, 2021 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported financial results for the third quarter ended September 30, 2021 and highlighted recent corporate milestones (Press release, Akebia, NOV 4, 2021, View Source [SID1234594496]).

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

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

                  Schedule Your 30 min Free Demo!

The Company will host a conference call on Tuesday, November 9, 2021, at 9:00 a.m. Eastern Time. Executives will highlight publications and scientific presentations of vadadustat Phase 3 clinical data, which will be presented at American Society of Nephrology Kidney Week 2021 beginning today. The Company will also discuss ongoing pre-commercialization activities ahead of a potential first-in-class U.S. launch for vadadustat, Akebia’s investigational oral therapeutic for the treatment of anemia due to chronic kidney disease (CKD). Vadadustat is currently under review by the U.S. Food and Drug Administration (FDA) with a scheduled Prescription Drug User Fee Act (PDUFA) date of March 29, 2022.

"We are within five months of vadadustat’s PDUFA date, which has the potential to be a pivotal catalyst for the company, and, if approved, would mean the availability of a novel, oral therapeutic for people living with anemia due to chronic kidney disease," said John P. Butler, Chief Executive Officer, Akebia Therapeutics. "We continue to value opportunities to share vadadustat efficacy and safety data with nephrologists through scientific congresses such as ASN Kidney Week. While our review with the FDA is ongoing, we remain confident that the data we have compiled and submitted for review makes a compelling case for approval in dialysis patients."

"Akebia has made substantial progress on pre-commercialization activities to prepare for a first-in-class product launch for vadadustat in the U.S. in 2022, subject to regulatory approval," added Dell Faulkingham, Chief Commercial Officer, Akebia Therapeutics. "As part of our commercialization preparation, we are focused on broad access initiatives, and are fortunate that, if approved, our specialized renal sales force will be ready to add a second product for use in the dialysis market. We are working closely with our partners at Vifor and Otsuka to ensure we are well-positioned to execute a successful launch, including preparedness for reimbursement for vadadustat under Transitional Drug Add-on Payment Adjustment (TDAPA) for dialysis organizations, which will be key to our launch."

Recent Business Highlights:

Akebia announced that Otsuka submitted a marketing authorization application to the European Medicines Agency for vadadustat for the treatment of anemia due to CKD in adults.
Vadadustat efficacy and safety data is being presented at the American Society of Nephrology Kidney Week 2021 beginning today, November 4, 2021. Eleven abstracts were accepted for presentation at Kidney Week 2021.
Akebia has settled all patent litigation proceedings related to Abbreviated New Drug Applications filed with respect to Auryxia (ferric citrate) tablets, which allows for generic versions of Auryxia beginning in March 2025.
Akebia announced that Ron Frieson, Chief Operating Officer of Children’s Healthcare of Atlanta (CHOA), joined Akebia’s Board of Directors. Mr. Frieson previously held leadership roles in external affairs, public policy, and diversity at CHOA, the Atlanta Urban League and BellSouth Corporation.
Third Quarter Financial Results

Revenues: Total revenue was $48.8 million for the third quarter of 2021 compared to $60.0 million for the third quarter of 2020. The decrease compared to the same period in 2020 was primarily due to lower collaboration revenue from Otsuka driven by lower development costs incurred subject to cost share provisions under both the Otsuka U.S. Agreement and the Otsuka International Agreement as the Company successfully completed the INNO2VATE and PRO2TECT global Phase 3 clinical programs and is currently engaged in close-out activities with respect to the programs. Collaboration revenue will continue to reflect collaboration development activities.
Collaboration revenue was $12.0 million for the third quarter of 2021 compared to $25.6 million for the third quarter of 2020.
Net product revenue for Auryxia was $36.8 million for the third quarter of 2021 compared to $34.4 million for the third quarter of 2020, an increase of 7.0% due to lower volume rebates related to the negative impact to sales volume as a result of COVID-19, and improved payor mix.
COGS: Cost of goods sold was $15.9 million for the third quarter of 2021 and included a $6.0 million decrease to the liability for excess purchase commitments primarily due to the settlement of all patent litigation proceedings related to Abbreviated New Drug Applications filed with respect to Auryxia, which allows for generic versions of Auryxia beginning in March 2025. Cost of goods sold was $30.3 million for the third quarter of 2020 and included $8.4 million in non-cash charges related to the fair-value inventory step-up from the application of purchase accounting and $8.5 million primarily related to the write-down of inventory. These charges for the third quarter of 2020 were partially offset by a $0.7 million reduction to the excess purchase commitments liability within cost of goods sold.
R&D Expenses: Research and development expenses were $40.5 million for the third quarter of 2021 compared to $46.9 million for the third quarter of 2020. The decrease compared to the prior year period was primarily due to the completion of the INNO2VATE and PRO2TECT global Phase 3 clinical programs.
SG&A Expenses: Selling, general and administrative expenses were $46.4 million for the third quarter of 2021 compared to $40.2 million for the third quarter of 2020. The increase compared to the prior year period was due primarily to increases in headcount related costs and one-time legal costs.
Net Loss: Net loss was $59.5 million for the third quarter of 2021 compared to $60.0 million for the third quarter of 2020.
Cash Position: Cash, cash equivalents and available-for-sale securities as of September 30, 2021, were $207.2 million. The Company believes that its cash resources will be sufficient to fund its current operating plan through at least the next twelve months.
"We continue to be judicious in spend as we invest in pre-commercialization activities in anticipation of the U.S. launch of vadadustat in 2022, if approved, while also supporting the marketing, sales, and payor strategies necessary to continue to drive Auryxia revenue growth," said David A. Spellman, Chief Financial Officer, Akebia Therapeutics. "We’re proud of the great work our renal sales and support teams have done to get a critical therapy to eligible kidney patients, especially in a market that has seen a decline over the past year due in part to the COVID-19 pandemic."

Conference Call

Akebia will host a conference call on Tuesday, November 9, 2021, at 9:00 a.m. Eastern Time to discuss its third quarter financial results and recent business highlights. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 5389484. The call will also be webcast LIVE and can be accessed via the Investors section of the Company’s website at View Source

A replay of the conference call will be available two hours after the completion of the call through November 15, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5389484. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

On November 4, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the third quarter of 2021 (Press release, Kadmon, NOV 4, 2021, View Source [SID1234594495]).

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

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

                  Schedule Your 30 min Free Demo!

"The third quarter was an exceptional period for Kadmon culminating in the availability of REZUROCK to patients living with cGVHD and the significant milestones that were achieved in the months following our strategic commercial launch," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "Our previously-announced merger with Sanofi is expected to close as soon as November 9, 2021, with a special meeting of stockholders scheduled for November 5, 2021. We are pleased that Sanofi has recognized the value of belumosudil and, pending the close of the merger, look forward to leveraging their expertise and resources to continue to build on our momentum."

Dr. Waksal added, "In addition to our commercial efforts, our clinical and preclinical work in other therapeutic areas remain underway. We continue to advance belumosudil forward in systemic sclerosis and continue to dose patients with KD033, our anti-PD-L1/IL-15 fusion protein. Our research and development efforts have also moved forward two product candidates (KD050 and KD045) that we anticipate will be in the clinic in 2022. I am so proud of the work taking place at Kadmon, and look forward to seeing the programs developed here continuing to progress."

2021 Program Updates and Milestones:

Sanofi Acquisition

On September 7, 2021, Kadmon entered into a definitive merger agreement with Sanofi:
The acquisition supports Sanofi’s strategy to continue to grow its General Medicines core assets and will immediately add REZUROCK to its transplant portfolio
Shareholders of Kadmon common stock will receive $9.50 per share in cash, which represents a total equity value of approximately $1.9 billion (on a fully diluted basis)
The acquisition remains subject to satisfaction or waiver of certain conditions to closing, as set forth in the definitive merger agreement
The Sanofi and Kadmon Boards of Directors unanimously approved the transaction and the transaction is expected to close in the fourth quarter of 2021
REZUROCK (belumosudil)

On August 19, 2021 REZUROCK became commercially available for shipment to prescribed patients in the United States through a network of rare hematology/oncology specialty pharmacies and distributors
Execution of commercial launch remain underway, with a focus on generating awareness of REZUROCK’s differentiated clinical value and facilitating market access
Experienced field team call on all target accounts, including engagement with 100% of the top 80 transplant centers, where ~90% of chronic graft-versus-host disease (cGVHD) patients in the U.S. are treated
Strong sales performance and demand during initial launch period; $12.2 million net sales achieved in 3Q 2021
The Company plans to present data on belumosudil at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, to be held December 11 – 14, 2021
Pipeline

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 5 (200 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)
The Company plans to present data from KD033-101 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting, to be held November 10 – 14, 2021
KD050

Continue ongoing Investigational New Drug Application (IND)-enabling activities of KD050, Kadmon’s anti-PD1/IL15 fusion molecule; IND target for 1H 2022
KD045

Continue ongoing Investigational New Drug Application (IND)-enabling activities of KD045, Kadmon’s next-generation ROCK inhibitor, for the treatment of fibrotic diseases; IND target for 4Q 2021
Financial Results

Third Quarter 2021 Results

Net sales for the three and nine months ended September 30, 2021 were $12.2 million and $12.6 million, respectively, compared to $0.3 million and $1.2 million for the same periods in 2020. The increases are attributable to the approval and commercialization of REZUROCK in the United States, which generated approximately $12.2 million of net sales for the three and nine months ended September 30, 2021.

Other revenues for the three and nine months ended September 30, 2021 were $2.5 million and $2.9 million, respectively, compared to $0.2 million and $6.4 million for the same periods in 2020. Other revenues during the three and nine months ended September 30, 2021 include $2.0 million in one-time license revenues related to the BioNova strategic partnership and other revenues during the nine months ended September 30, 2020 include $6.0 million in one-time license revenues related to the Meiji strategic partnership.

Operating expenses increased by approximately $12.3 million and $23.5 million for the three and nine months ended September 30, 2021, respectively, as compared to 2020, primarily related to REZUROCK commercial launch 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 September 30, 2021, the Company’s cash, cash equivalents and marketable debt securities totaled $251.6 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.

INDICATIONS AND USAGE

REZUROCK is a kinase inhibitor indicated for the treatment of adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Embryo-Fetal Toxicity: Based on findings in animals and its mechanism of action, REZUROCK can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential of the potential risk to a fetus and to use effective contraception.

Adverse Reactions

The most common (≥20%) adverse reactions, including laboratory abnormalities, in patients receiving REZUROCK were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

To report suspected adverse reactions, contact Kadmon at 1-877-377-7862 or the FDA at (800) FDA-1088 or www.fda.gov/medwatch.

Use in Specific Populations

Lactation: Advise not to breastfeed.

Please visit www.REZUROCK.com to see the full Prescribing Information for REZUROCK.

On November 4, 2021 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, reported financial results for the fiscal second quarter ended September 30, 2021 and provided a business update (Press release, Replimune, NOV 4, 2021, View Source [SID1234594494]).

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

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

                  Schedule Your 30 min Free Demo!

"Replimune continues to build towards a data rich 2022 where we are expecting to reach multiple inflection points culminating in the primary data analysis trigger for our registration-directed randomized controlled clinical trial with RP1 in cutaneous squamous cell carcinoma (CSCC) and the presentation of interim data from our registration directed cohort in anti-PD1 failed melanoma from the IGNYTE clinical trial," said Philip Astley-Sparke, CEO of Replimune. "In the first quarter of 2022 we will be presenting initial data in anti-PD1 failed non-melanoma skin cancers (NMSC) and in NMSC transplant patients with single agent RP1, both including CSCC. Our objective is to establish a broad skin cancer franchise with RP1. In addition to an update on RP2 at SITC (Free SITC Whitepaper) 2021, we look forward to presenting data during 2022 from the expansion of our Phase 1 studies with RP2 and RP3 with a focus on patients with liver metastases from prevalent tumor types. We also look forward to presenting a comprehensive Phase 2 development plan with RP2 and/or 3 in the first quarter of 2022, with study initiation intended around midyear, as we continue to make progress towards positioning our programs as a cornerstone of cancer treatment."

Recent Events and Corporate Updates

Replimune to present three ‘Trial-In-Progress’ posters and a poster including new data with RP2 at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting. The Company is scheduled to present four posters at SITC (Free SITC Whitepaper) being held November 10-14, 2021. Included in these posters will be a data update from its 30-patient Phase 1 clinical trial evaluating RP2 alone and combined with Opdivo in difficult-to-treat cancers.
Program Highlights*

CERPASS – Registration directed Phase 2 clinical trial in CSCC

RP1 in combination with Libtayo (cemiplimab) in CSCC: The Company is actively enrolling patients in CERPASS, its registration directed, global, randomized controlled Phase 2 clinical trial of RP1 in combination with Libtayo vs. Libtayo alone in 180 patients with advanced CSCC. The Company expects to complete enrollment such that the primary data analysis is expected to be triggered in late 2022.
IGNYTE – multi cohort Phase 2 clinical trial of RP1 combined with Opdivo

Anti-PD-1 failed melanoma cohort: The Company continues to enroll patients in the 125-patient cohort of the IGNYTE Phase 2 clinical trial in patients with anti-PD1 failed melanoma. The company continues to expect to release interim data from this cohort in late 2022 but, as previously disclosed, in order to document sufficient durability of response, an important secondary endpoint of the study, the primary analysis is expected to be triggered in mid-year 2023.
Non-melanoma skin cancer (NMSC) cohort: The Company has completed enrollment of 30-patients with anti-PD-1 naïve NMSC and continues to enroll patients with anti-PD-1 failed NMSC. The Company remains on track to provide updated data from the anti-PD1 naïve patients and initial data from the anti-PD-1 failed patients in the first quarter of 2022.
Anti-PD(L)-1 failed non-small cell lung cancer (NSCLC) cohort: Dosing is underway in a 30-patient cohort of RP1 in combination with Opdivo in anti-PD(L)-1 failed NSCLC. A recently filed amendment to the IGNYTE protocol includes modifications to the patient eligibility criteria which are expected to enhance enrollment into this cohort of the clinical trial. The Company had planned to provide initial data from this cohort in the first quarter of 2022. However, the changes made to facilitate enrollment are not expected to take effect in time to meet prior guidance and initial data is now expected to be released later in 2022.
ARTACUS – Phase 1b/2 clinical trial of RP1 as monotherapy in solid organ transplant recipients with skin cancers

The Company continues to enroll patients in this 65-patient clinical trial, with potential registrational intent, assessing the safety and efficacy of RP1 in liver and kidney transplant recipients with skin cancers, including CSCC. The Company remains on track to present initial data from this clinical trial in the first quarter of 2022.
RP2 and RP3

RP2 alone and in combination with Opdivo in difficult-to-treat cancers: The Company remains on track to provide a data update from the Phase 1 clinical trial in patients treated as monotherapy with RP2 and RP2 combined with Opdivo at SITC (Free SITC Whitepaper) in the fourth quarter of 2021. The Company has filed an amendment to expand this trial to, in particular, provide data in patients with liver metastases from various prevalent tumor types with a focus on patients with lung, breast and gastrointestinal cancers including colorectal cancer.
RP3 alone and in combination with anti-PD-1 therapy: The Company is enrolling patients in its Phase 1 clinical trial with RP3, with initial monotherapy data expected to be presented in the first quarter of 2022. The Company expects to start enrolling patients to be treated with RP3 in combination with anti-PD-1 therapy early in the new year, including patients with liver metastases from various prevalent tumor types such as lung, breast, head and neck cancer and gastrointestinal cancers including colorectal cancer.
RP2 and/or RP3 next stage development with focus on patients with liver metastases from a range of tumor types: The Company remains on track to initiate a broad clinical development program with RP2 and/or RP3, intended to include a range of prevalent tumor types with focus on patients with liver metastases, around mid-year 2022. Details of this plan are intended to be presented in the first quarter of 2022.

*Program Highlight dates are on a calendar-year basis.
Financial Highlights

Cash Position: As of September 30, 2021, cash, cash equivalents and short-term investments were $435.8 million, as compared to $476.3 million as of March 31, 2021. This decrease was primarily related to cash utilized in operating activities in advancing the Company’s expanded clinical development plan.

Based on the current operating plan, Replimune believes that existing cash and cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements into the second half of 2024, excluding any confirmatory trial required by the FDA or other regulatory body.
R&D Expenses: Research and development expenses were $19.9 million for the second quarter ended September 30, 2021, as compared to $14.1 million for the second quarter ended September 30, 2020. This increase was primarily due to increased clinical and manufacturing expenses driven by the Company’s lead programs and increased personnel expenses. Research and development expenses included $2.2 million in stock-based compensation expenses for the second quarter ended September 30, 2021.

G&A Expenses: General and administrative expenses were $9.3 million for the second quarter ended September 30, 2021, as compared to $5.6 million for the second quarter ended September 30, 2020. The increase was primarily driven by personnel-related costs, professional fees, and facility expansion. General and administrative expenses included $4.1 million in stock-based compensation expenses for the second quarter ended September 30, 2021.

Net Loss: Net loss was $29.4 million for the second quarter ended September 30, 2021, as compared to a net loss of $20.1 million for the second quarter ended September 30, 2020.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial will enroll 180 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD-1 therapy. The trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two primary efficacy endpoints as assessed by independent review, as well as duration of response, progression-free survival (PFS), and overall survival (OS) as its secondary endpoints. The study is being conducted under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi.
Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo. There are 4 tumor specific cohorts currently enrolling in this trial including a 125-patient cohort in anti-PD-1 failed cutaneous melanoma. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same trial of approximately 30 patients with melanoma. The additional cohorts are in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in anti-PD1 failed microsatellite instability high, or MSI-H/dMMR tumors and anti-PD(L)-1 failed non-small cell lung cancer, or NSCLC. This trial is being conducted under a collaboration and supply agreement with Bristol-Myers Squibb Company.
Opdivo is a registered trademark of Bristol-Myers Squibb Company.

About RP1
RP1 is Replimune’s lead Immulytic product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.

On November 4, 2021Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Protara Therapeutics, NOV 4, 2021, View Source [SID1234594493]).

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

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

                  Schedule Your 30 min Free Demo!

"We have made significant progress advancing our TARA-002 clinical programs in the third quarter, most notably the completion of our confirmatory large-scale GMP comparability work and the clearance of our Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for our non-muscle invasive bladder cancer (NMIBC) clinical development program. NMIBC is one of the most recurrent and difficult to treat cancers with limited treatment options, and we look forward to initiating our Phase 1 clinical trial of TARA-002 in adults with high-grade NMIBC by the end of the year," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are also working to align with the FDA on the design of a clinical trial of TARA-002 in patients with lymphatic malformations (LMs), a rare pediatric indication for which there are currently no U.S. FDA-approved therapies."

Recent Highlights and Upcoming Milestones

TARA-002 in NMIBC

In October 2021, Protara announced that the FDA cleared the Company’s IND application for TARA-002, an investigational cell-based therapy in development for the treatment of NMIBC. The confirmatory, GMP-scale comparability data for TARA-002 in relation to OK-432, the originator therapy for TARA-002, have been completed and were reviewed by the FDA as part of the clearance of the IND.
The Company plans to commence a Phase 1 clinical trial by the end of 2021 to assess the safety, tolerability, and preliminary signs of anti-tumor activity of TARA-002 in adults with high-grade NMIBC.
TARA-002 in LMs

In October 2021, the Company updated its IND submission for TARA-002 for the treatment of LMs with completed confirmatory, GMP-scale comparability data, and plans to engage 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)

In September 2021, the Company announced the completion of a retrospective prevalence study designed to enhance understanding of the incidence of IFALD in patients dependent on parenteral nutrition (PN). The study found that approximately 30% of patients who are dependent on PN have cholestasis, despite the use of medical management in these patients.
The Company plans to use the results from the completed retrospective study and ongoing prospective study to inform next steps for the IV Choline Chloride development program.
Corporate Update

In October 2021, the Company hired Mary Grendell as its new General Counsel. Ms. Grendell has an extensive track record as a legal executive, most recently serving as Vice President, Deputy General Counsel and Corporate Secretary, at Intercept Pharmaceuticals. Previously, she held positions at Mylan (now part of Viatris) and Amgen following her early legal career at Sullivan & Cromwell and Covington & Burling. Ms. Grendell received her J.D. from the University of Pennsylvania Law School and her B.A. from Yale University.
Third Quarter 2021 Financial Results

As of September 30, 2021, cash, cash equivalents and marketable debt securities totaled $138.4 million.

Research and development expenses for the third quarter of 2021 increased to $4.1 million from $2.8 million during the third quarter of 2020. The increased R&D expenses were primarily due to increases in non-clinical, clinical and regulatory expenses associated with TARA-002, headcount and stock-based compensation, and other employee-related expenses.

General and administrative expenses for the third quarter of 2021 increased to $6.7 million from $5.3 million during the third quarter of 2020. The increase was primarily due to increases in headcount and employee-related expenses, development of commercial capabilities, and costs associated with the new corporate office in New York, NY.

For the third quarter of 2021, Protara reported a net loss of $10.8 million, or $0.96 per share, compared with a net loss of $8.0 million, or $1.26 per share, for the third quarter of 2020. Net loss for the third quarter of 2021 included approximately $2.7 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and 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 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 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

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 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.