On August 5, 2021 Aeglea BioTherapeutics, Inc. (Nasdaq:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported financial results for the second quarter ended June 30, 2021, and reviewed recent corporate updates and program highlights (Press release, Aeglea BioTherapeutics, AUG 5, 2021, View Source [SID1234585789]).

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"2021 is proving to be a pivotal year for Aeglea. In the second quarter, we achieved a significant milestone in our second clinical development program. With the initiation of dosing in our Phase 1/2 clinical trial of AGLE-177, Aeglea is making progress for patients who suffer from Homocystinuria, a rare and progressive disease which currently has limited treatment options," said Anthony Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. "Additionally, with the strengthening of our leadership team, deepening relationship with the rare disease community and advancing our scientific communications platform, we continue to prepare for the data readout for our Phase 3 clinical trial of pegzilarginase in Arginase 1 Deficiency in the fourth quarter as well as lay the groundwork for potential approval and commercialization."

Second Quarter and Recent Highlights and Updates

AGLE-177 in Homocystinuria

Initiated dosing with AGLE-177 in a Phase 1/2 clinical trial in people with Homocystinuria. The trial is anticipated to enroll 16–20 patients at sites located in the United Kingdom and Australia. Aeglea expects to provide a clinical update prior to the end of 2021.
Pegzilarginase in Arginase 1 Deficiency

Published 20-week data from a Phase 1/2 and open-label extension studies of pegzilarginase in the Journal of Inherited Metabolic Disease. The article, titled "Clinical Effect and Safety Profile of Pegzilarginase In Patients with Arginase 1 Deficiency," is available in the July issue.
Corporate

Strengthened the leadership team with appointment of Jonathan Alspaugh as chief financial officer and Jim Kastenmayer as general counsel.
Mr. Alspaugh joined Aeglea from Evercore, where he most recently served as a managing director in the firm’s corporate advisory business.
Mr. Kastenmayer joined Aeglea from Viela Bio, where he provided strategic guidance and legal advice, including advising Viela in connection with the FDA approval and launch of its first commercial product.
Expanded the board of directors with the appointment of Marcio Souza. Mr. Souza serves as the president & chief executive officer of Praxis Precision Medicines.
Participated in Rare Disease Awareness Week on Capitol Hill 2021, meeting with patient advocates and law makers to raise awareness and discuss the specific needs facing the rare disease community.
Supported the launch of the Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, of which Aeglea is a founding member.
Upcoming Investor Events

Wells Fargo 2021 Virtual Healthcare Conference, September 9–10
HC Wainwright 23rd Annual Global Investment Conference, September 13–15
2021 Cantor Virtual Healthcare Conference, September 27–30
Second Quarter 2021 Financial Results

As of June 30, 2021, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $130.4 million inclusive of the $21.5 million upfront cash payment received from Immedica Pharma AB pursuant to the March 2021 license and supply agreement. The Company expects its cash, cash equivalents and investments will enable it to fund its operating expenses and capital expenditure requirements into 2023.

Aeglea recognized license and development revenues of $13.7 million in the second quarter of 2021, as a result of its license and supply agreement with Immedica for the commercial rights of pegzilarginase in certain territories outside the United States. The revenues recorded in the second quarter of 2021 are related to the transfer of the license and delivery of trial and regulatory services. Aeglea recognized no revenue for the corresponding period of 2020.

Research and development expenses totaled $13.6 million for the second quarter of 2021 and $16.9 million for the second quarter of 2020. The decrease was primarily associated with completing certain pre-commercial manufacturing activities for Aeglea’s lead product candidate, pegzilarginase.

General and administrative expenses totaled $6.8 million for the second quarter of 2021 and $4.7 million for the second quarter of 2020. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure.

Net loss totaled $6.8 million and $21.4 million for the second quarter of 2021 and 2020, respectively, with non-cash stock compensation expense of $2.1 million and $1.6 million for the second quarter of 2021 and 2020, respectively.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme, which has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. Aeglea’s Phase 1/2 and Phase 2 open-label extension data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. The Company’s ongoing single, global pivotal Phase 3 PEACE trial is designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of plasma arginine reduction. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough, Fast Track and Orphan Drug Designations from the FDA as well as Orphan Drug Designation from the European Medicines Agency.

About AGLE-177 in Homocystinuria

AGLE-177 is a novel recombinant human enzyme, which degrades the amino acid homocysteine and its related homocystine dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of homocysteine and homocystine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177, which is designed to lower abnormally high blood levels of homocysteine, improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.

On August 4, 2021 Shanghai Huaota Biopharmaceutical Co., Ltd. (hereinafter referred to as "Huaota") reported the "Approval Notice for Drug Clinical Trial" approved and issued by National Medical Products Administration for the company’s injection HB0030 (anti-TIGIT monoclonal antibody) (Press release, Huabo Biopharm, AUG 4, 2021, View Source [SID1234656059]).

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HB0030 is an anti-TIGIT humanized monoclonal antibody of lgG1, independently developed by Huaota. It can bind to TIGIT with high affinity, thereby blocking the binding to its ligands (such as CD155). The binding of HB0030 to TIGIT on the surface of T cells and NK cells effectively blocks the binding of TIGIT to CD155, then relieves immunosuppression and reactivates the tumor-killing effect of T cells and NK cells.

Preclinical studies have shown that HB0030 has higher affinity and strong in vitro ADCC/CDC activity and demonstrated strong anti-tumor activity in preclinical studies. HB0030 is planned to use in combination with other product of Huaota, such as HOT-1030 (anti-CD137 monoclonal antibody) and HB0025 (anti-PD-L1/VEGF bispecific antibody) to achieve synergistic or additive effect on tumor inhibition.

Potential MoA of HB0030

There is currently no anti-TIGIT drugs on the market , and the monoclonal antibody Tiragolumab developed by Roche is in the first class in Phase III clinical trials at present. In January 2021, the combination of Tiragolumab and Tecentriq (anti-PD-L1 monoclonal antibody) for the treatment of NSCLC with high PD-L1 expression was granted by the FDA as a breakthrough therapy. Vibostolimab, developed by Merck, is also currently in a Phase III clinical study in combination with Pembrolizumab (an anti-PD-1 monoclonal antibody) for the treatment of NSCLC. There are different anti-TIGIT antibodies developed by four domestic companies that have clinical launched trials, including BeiGene.

On August 4, 2021 Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, reported that it will host a webcast and conference call to provide a corporate and financial update for the second quarter of 2021 on Wednesday, August 11, 2021, at 4:30 p.m. ET (Press release, Shattuck Labs, AUG 4, 2021, View Source [SID1234591766]).

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The live call may be accessed by dialing (833) 614-1555 (domestic) or (516) 575-8754 (international) and entering the conference code: 7172288. The live and archived webcast will be available on the Events & Presentations section of the Company’s website. A replay of the webcast will be archived for up to 90 days following the presentation date.

On August 4, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported that the Centers for Medicare & Medicaid Services (CMS) has granted a new technology add-on payment (NTAP) for COSELA (trilaciclib) when administered to Medicare beneficiaries in the hospital inpatient setting (Press release, G1 Therapeutics, AUG 4, 2021, View Source [SID1234587577]). It will become effective for provider billing on October 1, 2021. An NTAP provides additional payment to hospitals above the standard Medicare Severity Diagnosis-Related Group (MS-DRG) payment amount.

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This grant follows the receipt of a C Code for pass through hospital outpatient system use (effective July 1, 2021) and a permanent J Code for all sites of care (effective October 1, 2021).

The NTAP will provide hospitals with a payment, in addition to the standard-of-care DRG reimbursement, of up to 65 percent of the average cost of the technology if the cost of the discharge exceeds the full DRG payment. As such, beginning on October 1, 2021, CMS will provide an additional maximum payment of $5,526.30 for COSELA when used in the inpatient hospital setting for fiscal year 2022. Congress created the NTAP program to ensure that Medicare beneficiaries have timely access to innovative therapies while the agency collects data about them to use in future rate-setting.

"CMS’ issuance of an NTAP for COSELA recognizes its potential to address an urgent need for proactive multilineage myeloprotection in patients living with extensive stage small cell lung cancer," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "We believe the decision to include COSELA in the NTAP program is an important step that will help increase patient access to this important drug. We applaud CMS for their decision; the need for effective tools to help clinicians reduce or prevent myelosuppressive events is critical for extensive stage small cell lung cancer patients undergoing chemotherapy."

About COSELA (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication
COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information
COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

On August 4, 2021 G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, reported a corporate and financial update for the second quarter ended June 30, 2021 (Press release, G1 Therapeutics, AUG 4, 2021, View Source [SID1234587576]).

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"The second quarter of 2021 was a period of solid progress across G1, as we seek to develop COSELA for patients suffering from a variety of cancers," said Jack Bailey, Chief Executive Officer of G1 Therapeutics. "In our first full quarter of sales of COSELA, the commercial team continued to build the commercial foundation for this important myeloprotection drug for patients with ES-SCLC undergoing chemotherapy. We believe that COSELA is a paradigm-changing product, allowing cancer patients to be treated proactively to reduce the impact of the multilineage myelosuppression side effects of chemotherapy. So far, the enthusiasm for COSELA is encouraging. We also initiated three new COSELA clinical trials during the second quarter, including a registrational trial in metastatic TNBC and two Phase 2 trials, one in NSCLC and the other in bladder cancer. With the ongoing commercial launch, the expansion of our innovative tumor-agnostic pipeline, and a strong financial position, we are excited about the opportunities ahead for COSELA."

Second Quarter 2021 and Recent Highlights

Financial

Achieved Net COSELA (trilaciclib) Revenue of $2.5 Million.
Ended the Second Quarter with Cash and Cash Equivalents of $244.0 million: The current financial position expected to be sufficient to fund G1’s operations and capital expenditures into 2023.
Medical

Presented New Data Describing the Estimated Economic Impact of Treating Myelosuppression in Patients with ES-SCLC at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR): The first poster used a cost-benefit model to predict an estimated economic value from a general U.S. commercial payer perspective of using COSELA prior to chemotherapy in ES-SCLC to project a significant payer cost savings based on assumptions that myelosuppressive adverse events and their associated treatment costs would be reduced. The second poster quantified the significant health burden, economic toll, and health-related quality-of-life effects of chemotherapy-induced myelosuppression among Medicare patients diagnosed with SCLC. (Press release here)
Presented Results of Analyses Evaluating the Immune Effects of COSELA in Patients with ES-SCLC at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting: Patients receiving COSELA prior to chemotherapy had greater peripheral T-cell clonal expansion than patients receiving placebo. The data suggest that, among patients treated with COSELA plus either etoposide and carboplatin (E/C) or E/C plus atezolizumab, increased clonal expansion is associated with clinical response, indicating that COSELA may enhance antitumor immunity in patients with ES-SCLC treated with chemotherapy. (Press release here)
Presented Positive Data Suggesting Strong Safety Profile and Evidence of Antitumor Activity of Rintodestrant Combined with Palbociclib in ER+/HER- Advanced Breast Cancer at ASCO (Free ASCO Whitepaper): In the 40-patient combination arm of the Phase 1 trial, rintodestrant was very well tolerated and did not result in additional or more severe toxicities when added to Palbociclib. The clinical benefit rate (CBR; percentage of patients with either confirmed complete or partial response or stable disease lasting ≥ 24 weeks) doubled from 30 percent with rintodestrant monotherapy to 60 percent with the combination of rintodestrant and Palbociclib; among patients with early relapse, the CBR was 73%. (Press release here)
Clinical

Initiated Pivotal Trial of COSELA in Locally Advanced Unresectable or Metastatic TNBC: Patient enrollment is underway in PRESERVE 2, a randomized, double-blind, placebo-controlled Phase 3 registrational trial of COSELA in patients receiving first- or second-line gemcitabine/carboplatin chemotherapy for locally advanced unresectable or metastatic TNBC. (Press release here)
Received Fast Track Designation for COSELA in TNBC: The FDA granted Fast Track designation to COSELA investigation for use in combination with chemotherapy for the treatment of locally advanced or metastatic TNBC. Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill unmet medical needs. (Press release here)
Initiated Phase 2 Trial of COSELA in Metastatic NSCLC: Patent enrollment is underway in PRESERVE 4, a randomized double-blind placebo-controlled Phase 2 trial of COSELA administered prior to docetaxel in patients with NSCLC in the 2nd and 3rd line setting who have previously been treated with a checkpoint inhibitor and chemotherapy. The primary endpoint of the trial is to evaluate the anti-tumor effect of COSELA on overall survival (OS) compared to placebo. (Press release here)
Initiated Phase 2 Trial of COSELA in Bladder Cancer: Patent enrollment is underway in PRESERVE 3, a randomized double-blind placebo-controlled Phase 2 trial of COSELA administered with first-line platinum-based chemotherapy and the immune checkpoint inhibitor avelumab maintenance therapy in patients with untreated, locally advanced or metastatic urothelial carcinoma. (Press release here)
Corporate

Announced Andrew Perry as Chief Commercial Officer: Mr. Perry, formerly the Vice President of US Marketing for ViiV Healthcare NA, brings nearly 25 years of leadership experience in commercial launch strategy, digital marketing, and co-promotion management to G1, with extensive capabilities in launching and growing brands in multiple areas including oncology. (Press release here)
Second Quarter 2021 Financial Results

As of June 30, 2021, cash and cash equivalents totaled $244.0 million, compared to $207.3 million as of December 31, 2020.

Total revenues for the second quarter of 2021 were $6.6 million, including $2.5 million in net product sales of COSELA and license revenue of $4.1 million, primarily related to a development milestone payment from the Company’s license agreement with Simcere, clinical trial reimbursements from EQRx, and delivery of clinical drug supply and manufacturing services to Simcere, EQRx and Genor. Total revenues for the six months ended June 30, 2021 were $20.8 million.

Operating expenses for the second quarter of 2021 were $44.8 million, compared to $33.0 million for the second quarter of 2020. GAAP operating expenses include stock-based compensation expense of $5.7 million for the second quarter of 2021, compared to $4.4 million for the second quarter of 2020. Total operating expenses for the six months ended June 30 was $84.5 million.

Cost of goods sold expense for the second quarter of 2021 were $0.8 million, compared to $0 for second quarter of 2020. The increase related to the Company’s period costs for the sales of COSELA. Cost of goods sold for the six months ended June 30 was $1.1 million.

Research and development (R&D) expenses for the second quarter of 2021 were $18.8 million, compared to $18.5 million for the second quarter of 2020. The increase in R&D expenses was primarily due to an increase in clinical trial spend, which is offset by a decrease in costs associated with the manufacturing of active pharmaceutical ingredients and drug product to support clinical trials, as well as external costs related to discovery and pre-clinical development. R&D expenses for the six months ended June 30 were $35.3 million.

Selling, general and administrative (SG&A) expenses for the second quarter of 2021 were $25.2 million, compared to $14.4 million for the second quarter of 2020. The increase in SG&A expenses was largely due to an increase in commercialization activities, an increase in compensation due to increases in headcount, and increased spend on information technology, professional services, and other administrative costs. SG&A expenses for the six months ended June 30 were $48.2 million.

The net loss for the second quarter of 2021 was $39.4 million, compared to $31.2 million for the second quarter of 2020. The basic and diluted net loss per share for the second quarter of 2021 was $(0.94) compared to $(0.83) for the second quarter of 2020. The net loss for the six months ended June 30 was $65.9 million. The basic and diluted net loss per share for the six months ended June 30 was $(1.59).

Financial Guidance

The Company expects its current financial position to be sufficient to fund its operations and capital expenditures into 2023.

Webcast and Conference Call

G1 will host a webcast and conference call at 4:30 p.m. ET today to provide a corporate and financial update for the second quarter 2021 ended June 30, 2021. The live call may be accessed by dialing (866) 763-6020 (domestic) or (210) 874-7713 (international) and entering the conference code: 3553037. A live and archived webcast will be available on the Events & Presentations page of the company’s website: www.g1therapeutics.com. The webcast will be archived on the same page for 90 days following the event.

About COSELA (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication
COSELA (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information
COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. View Source

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit