On August 5, 2021 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose of bettering the lives of people impacted by kidney disease, reported financial results for the second quarter ended June 30, 2021 and highlighted recent corporate milestones (Press release, Akebia, AUG 5, 2021, View Source [SID1234585790]). The Company will host a conference call today, Thursday, August 5, 2021, at 9:00 a.m. Eastern Time.

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"The first half of 2021 has been marked by significant milestones that have further strengthened Akebia’s position and the potential market opportunity for vadadustat, an investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), setting the stage for an exciting and catalyst-rich year ahead for us. During the quarter, these achievements included publication of our global Phase 3 results for vadadustat in the New England Journal of Medicine and, more recently, FDA acceptance of the vadadustat NDA for filing with a PDUFA target action date of March 29, 2022," stated John P. Butler, Chief Executive Officer of Akebia. "As there are currently no approved HIF-PHIs to treat anemia due to chronic kidney disease (CKD) in the U.S., we believe vadadustat is positioned as a potential first-in-class product with a broader opportunity in the dialysis market than originally anticipated. With vadadustat’s PDUFA date set for March, we are highly focused on pre-launch activities to ensure that we are well positioned for a successful U.S. launch in 2022, and are excited to be one step closer to having a novel, oral therapeutic available for people living with this disease, subject to regulatory approval."

Steven K. Burke, M.D., Senior Vice President, Research & Development and Chief Medical Officer of Akebia stated, "We remain confident in the clarity and quality of our data, and continue to be encouraged by the safety profile of vadadustat demonstrated in our global Phase 3 program for the treatment of anemia due to CKD. As seen in the recent New England Journal of Medicine publications of our global Phase 3 results, the data showed no significant safety signals on adverse events, including thromboembolic events, seizures and infections. More specifically, the data showed that these events were very similar for vadadustat as compared to darbepoetin alfa."

Akebia is also working in close collaboration with its partner, Otsuka Pharmaceutical Co. Ltd., to prepare a Marketing Authorization Application for vadadustat for submission to the European Medicines Agency, expected this year.

Recent Business Highlights:

In late May, the FDA accepted for filing the NDA for vadadustat for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis. The FDA assigned the application a standard review and a Prescription Drug User Fee Act (PDUFA) target action date of March 29, 2022.
In April, the New England Journal of Medicine published the results of Akebia’s global Phase 3 program for vadadustat, which consisted of two programs that evaluated the efficacy and safety of vadadustat versus darbepoetin alfa for the treatment of anemia due to CKD in adult patients on dialysis (INNO2VATE) and not on dialysis (PRO2TECT).
In June, Akebia presented data regarding the hematologic efficacy of vadadustat for anemia in patients with kidney failure on dialysis and not on dialysis from the global Phase 3 program for vadadustat at the European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) Virtual Congress 2021.
In March, Akebia submitted an NDA to the FDA for vadadustat for the treatment of anemia due to CKD in both adult patients on dialysis and adult patients not on dialysis.
Second Quarter Financial Results

Revenues: Total revenue was $52.9 million for the second quarter of 2021 compared to $90.1 million for the second quarter of 2020. The decrease compared to the same period in 2020 was primarily due to lower collaboration revenue consistent with the Company successfully completing the INNO2VATE and PRO2TECT global Phase 3 clinical programs.
Collaboration revenue was $20.0 million for the second quarter of 2021 compared to $59.4 million for the second quarter of 2020.
Net product revenue for Auryxia was $33.0 million for the second quarter of 2021 compared with $30.7 million for the second quarter of 2020, an increase of 7.4 percent.
COGS: Cost of goods sold was $52.5 million for the second quarter of 2021 and included a $30.3 million non-cash charge related to an increase to the liability for excess purchase commitments consistent with the Company’s long-term payor contract strategy, which remains focused on contract economics and net product revenue growth. Cost of goods sold was $174.6 million for the second quarter of 2020 and included a non-cash impairment charge of $115.5 million related to the Auryxia intangible asset, $19.9 million in non-cash charges related to the fair-value inventory step-up from the application of purchase accounting, $11.0 million in non-cash charges related to an increase to the liability for excess purchase commitments and $9.9 million primarily related to the write-down of inventory.
R&D Expenses: Research and development expenses were $37.2 million for the second quarter of 2021 compared to $52.8 million for the second 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 $41.7 million for the second quarter of 2021 compared to $35.5 million for the second quarter of 2020. The increase compared to the prior year period was due primarily to higher marketing expenses.
Net Loss: Net loss was $83.0 million for the second quarter of 2021 compared to $175.8 million for the second quarter of 2020. The improvement in net loss compared to the prior year period was due primarily to the non-recurrence of a $115.5 million non-cash impairment charge in the prior year period, as well as lower operating expenses in the 2021 period, partially offset by lower collaboration revenue in the 2021 period.
Cash Position: Cash, cash equivalents and available-for-sale securities as of June 30, 2021 were $247.0 million. This balance includes net proceeds of $37.3 million from sales of common stock under the Company’s at-the-market offering program during the second quarter of 2021. The Company also received net cash proceeds of $16.1 million from sales of common stock under this program subsequent to quarter end through July 16, 2021. The Company believes that its cash resources will be sufficient to fund its current operating plan through at least the next twelve months. Additionally, the Company believes its cash runway would extend beyond the next twelve months assuming timely regulatory approval of vadadustat and the receipt of associated regulatory milestones.
"We continue to be encouraged by Auryxia’s revenue growth, which we believe is a great illustration of our commercial team’s execution in this ongoing COVID-19 environment," stated David A. Spellman, Chief Financial Officer of Akebia. "We believe this performance also highlights Auryxia’s favorable product profile and the critical nature of this therapy. While we remain cautious due to COVID-19, we believe the team’s focus and execution on marketing, sales, and payor strategies will continue to drive net product revenue growth."

Conference Call

Akebia will host a conference call today, Thursday, August 5, 2021, at 9:00 a.m. Eastern Time to discuss its second 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 5529396. 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 August 11, 2021. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 5529396. An online archive of the conference call can be accessed via the Investors section of the Company’s website at View Source

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.