2seventy bio Reports First Quarter Financial Results and Recent Operational Progress

On May 12, 2022 2seventy bio, Inc. (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, reported financial results and recent highlights for the first quarter ended March 31, 2022 (Press release, 2seventy bio, MAY 12, 2022, View Source [SID1234614340]).

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"2seventy bio has started 2022 in a strong position, and we’ve already begun to execute on some key clinical milestones," said Nick Leschly, chief kairos officer. "This quarter, we enrolled the first patients in clinical studies of SC-DARIC33 and bbT369. We look forward to continuing to enroll patients in these studies and providing an update at the appropriate time. In addition, we continue to focus on other foundational elements of 2seventy bio: growth of ABECMA and a sound financial position. Despite a challenging external environment, I’m pleased with the steady progress on both fronts with continued demand for ABECMA as well securing additional capital in March that we anticipate will provide cash runway into 2025. We’re proud of the momentum we’ve established as a team at 2seventy bio because we know that every day matters as we focus on delivering more time to patients with cancer and their families."

COMMERCIAL PROGRESS
Bristol Myers Squibb reported total U.S. ABECMA (idecabtagene vicleucel; ide-cel) first quarter revenues of $56 million, consistent with our 2022 plan. 2seventy bio and Bristol Myers Squibb share equally in all profits and losses related to developing, manufacturing and commercializing ABECMA in the U.S. In 2022, 2seventy anticipates total U.S. ABECMA revenues of $250-$300 million and we are continuing to track to the high end of the range bolstered by continued high demand for a proven treatment and increasing manufacturing capacity.

Demand for ABECMA is expected to continue to fully utilize the expanding manufacturing capacity throughout 2022 and 2023.

RECENT HIGHLIGHTS

SC-DARIC33 FIRST PATIENT ENROLLED – Last quarter, the first patient with acute myeloid leukemia (AML) was enrolled in PLAT-08, a phase 1 study of SC-DARIC33 led by Seattle Children’s Therapeutics in relapsed or refractory pediatric and young adult AML. This is the first-in-human application of 2seventy bio’s proprietary DARIC T cell platform.
BBT369 FIRST PATIENT ENROLLED – Last quarter, the first patient with B cell non-Hodgkin lymphoma (B-NHL) was enrolled in CRC-403, a phase 1/2 study of bbT369 in patients with relapsed and/or refractory B-NHL. This study serves as a safety and proof-of-concept assessment of 2seventy bio’s proprietary megaTAL gene editing platform, dual-targeting strategies and split co-stimulation signaling technology.
bbT369 PRECLINICAL DATA AT AACR (Free AACR Whitepaper) – New preclinical data on bbT369 was presented in a poster session (poster #581) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 in New Orleans, LA on Sunday, April 10. The data presented at AACR (Free AACR Whitepaper) show the anti-lymphoma activity of bbT369 observed to date and suggest that, as intended in the design, bbT369 has the potential to overcome failure modes of anti-CD19 CAR therapies.
ASCO ABSTRACTS ACCEPTED– In April 2022, the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) released the abstract titles for its 2022 Annual Meeting. 2seventy bio will present updates from its portfolio of oncology cell therapies at the meeting, including a correlative analysis, in partnership with Bristol Myers Squibb, defining patient profiles associated with manufacturing and clinical endpoints in patients treated with ide-cel, and a trial in progress poster on CRB-403, a phase 1/2 study on bbT369.
Poster Discussion [#8021]: Correlative analysis to define patient profiles associated with manufacturing and clinical endpoints in relapsed/refractory multiple myeloma (RRMM) patients treated with idecabtagene vicleucel (ide-cel; bb2121), an anti-BCMA CAR T cell therapy.
Presenting Author: Julie Rytlewski, PhD, Bristol Myers Squibb
Date/Time: Saturday, June 4, 2022, 5:30 PM – 7:00 PM ET
Poster [#TPS7580]: CRC-403: A phase 1/2 study of bbT369, a dual targeting CAR T-cell drug product with a gene edit, in relapsed and/or refractory B-cell non-Hodgkin lymphoma (NHL).
Presenting Author: Frederick L. Locke, MD, H. Lee Moffitt Cancer Center & Research Institute
Date/Time: Saturday, June 4, 2022, 9:00 AM – 12:00 PM ET
UPCOMING ANTICIPATED MILESTONES

ABECMA

Anticipated $250-300 million total U.S. commercial revenue in 2022; profits and losses shared with Bristol Myers Squibb
Increasing manufacturing capacity expected over 2022 and 2023
KarMMa-2 study in high-risk multiple myeloma proof-of-concept data in 2022
KarMMa-3 study in 3L+ registrational data in 2023 with potential FDA approval in 2023-2024
Pipeline

Initial assessment of feasibility of bbT369 drug product manufacturing and patient safety in 2H 2022
Initial assessment of feasibility of SC-DARIC33 drug product manufacturing and drug regulated anti-CD33 activity in 2H 2022
SELECT FIRST QUARTER 2022 FINANCIAL RESULTS

Bristol Myers Squibb reported total U.S. revenues of $56 million for ABECMA for the three months ended March 31, 2022. 2seventy bio and Bristol Myers Squibb share equally in all profits and losses related to development, manufacturing and commercializing ABECMA in the U.S. We reported share of collaboration loss of $5.4 million for the three months ended March 31, 2022, which includes our share of gross profit/loss less costs associated with the commercialization of ABECMA in the U.S. The collaboration reported a loss this quarter due to continued investment in manufacturing scale-up and commercialization.
Total revenues were $8.4 million for the three months ended March 31, 2022, compared to $11.9 million for the three months ended March 31, 2021. The decrease for the three-month period was primarily driven by a decrease in royalty and other revenue as a result of the termination of the Company’s license agreement with Novartis in March 2021.
Research and development expenses were $69.2 million for the three months ended March 31, 2022, compared to $77.6 million for the three months ended March 31, 2021. The decrease for the period was primarily driven by decreased collaboration research costs, which represent the Company’s share of research and development costs under the collaboration with Bristol Myers Squibb.
Selling, general and administrative expenses were $23.9 million for the three months ended March 31, 2022, compared to $24.6 million for the three months ended March 31, 2021. The slight decrease was primarily driven by a decrease in employee compensation expense. In 2021, the Company recorded higher stock-based compensation and bonus expense related to a retention plan that was enacted during the separation of 2seventy bio from bluebird bio. The retention plan was completed at the end of 2021.
Net loss was $85.7 million for the three months March 31, 2022, compared to $87.2 million for the three months ended March 31, 2021.
2seventy bio ended the first quarter of 2022 with cash, cash equivalents and marketable securities of $452.5 million, including net proceeds from our March 2021 private placement of $165.7 million, after deducting placement agent fees and other offering expenses payable by the Company.

HTG Molecular Diagnostics Reports First Quarter 2022 Results

On May 12, 2022 HTG Molecular Diagnostics, Inc. (Nasdaq: HTGM) (HTG), a life science company advancing precision medicine through its innovative transcriptome-wide profiling technology, reported recent business highlights and financial results for the quarter ended March 31, 2022 (Press release, HTG Molecular Diagnostics, MAY 12, 2022, View Source [SID1234614357]).

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Recent Business Highlights

Released a proof-of-approach white paper, demonstrating the utility of HTG’s proprietary profiling technologies as a key component of its novel drug discovery and design platform.

Expanded the HTG Therapeutics business unit leadership team with the addition of Christina M. Carruthers, Ph.D., as Vice President of Target Strategy and Early Development.

Expanded the utility of the HTG EdgeSeq technology by harmonizing sample preparation protocols for the miRNA Whole Transcriptome Assay and the HTG Transcriptome Panel (HTP), allowing customers to use a single tissue section and one lysate method to run both panels.

Completed a private placement with a leading healthcare investor. The gross proceeds to HTG from the private placement, after deducting the placement agent fees and other expenses related to the transaction, were approximately $7.2 million.

First Quarter 2022 Financial Highlights:

Revenue for the quarter ended March 31, 2022 was $1.2 million, compared with $1.4 million for the same period in 2021, and was comprised entirely of product and product-related services revenue in both periods. Sales of the HTP to new and existing customers as consumables and sample processing services represented over 40% of revenue for the quarter ended March 31, 2022.

Net loss from operations for the quarter ended March 31, 2022 was $6.3 million, compared with $4.6 million for the same period in 2021. Net loss per share was $(0.81) for the quarter ended March 31, 2022 compared with $(0.80) for the first quarter of 2021.

Cash, cash equivalents and short-term available-for-sale securities totaled $21.6 million as of March 31, 2022, with current liabilities of approximately $8.5 million and non-current liabilities of $8.7 million.

Conference Call and Webcast:

HTG will host a conference call for the investment community today beginning at 4:30 p.m. Eastern Time. Conference call and webcast details are as follows:

Chemomab Therapeutics Announces First Quarter 2022 Financial Results and Provides Corporate Update

On May 12, 2022 Chemomab Therapeutics, Ltd. (Nasdaq: CMMB), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, reported financial and operating results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Chemomab, MAY 12, 2022, View Source [SID1234614374]).

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"During the quarter we continued to make good progress in refining and implementing revisions to our CM-101 clinical program aimed at decreasing overall development risk, maximizing the clinical data obtained to facilitate clinical decision-making, and generating critical data to support advancement to registration trials," said Dale Pfost, PhD, Chief Executive Officer of Chemomab. "We are expanding our efforts in primary sclerosing cholangitis (PSC) with an enlarged clinical trial that is recruiting patients from new sites in the U.S. and Europe, while adding an important dose finding component and an open-label extension. We will be performing an interim analysis of the currently enrolling dose cohort in the PSC study to assess safety and to confirm the planned sample sizes for the CM-101 dose cohorts. This planned interim read-out is expected late this year."

Dr. Pfost added, "In systemic sclerosis (SSc), we expect the revised Phase 2 trial design to enable an expedited path to proof-of-concept data, and, importantly, to provide additional information on CM-101’s activity in modifying the skin, lung and vascular pathophysiology seen in SSc patients. We are designing this trial with the assistance of key SSc opinion leaders and are on track to launch the trial by the end of the year. I am also pleased to report that we have now completed enrollment in our Phase 2 safety, pharmacokinetic and biomarker liver fibrosis study, with final readouts expected near year’s end. We continue to use our capital efficiently and look forward to providing further details on our progress with the PSC and SSc Phase 2 trials this summer."

Recent Highlights:

Named Jack Lawler Vice President of Global Clinical Development Operations. Mr. Lawler, who is based in the U.S., brings the company more than 20 years of diverse global clinical drug development experience.
Chief Scientific Officer Dr. Adi Mor presented "Blocking CCL24, a novel target regulating inflammation fibrosis and endothelial damage, shows promising potential as treatment for Systemic Sclerosis" at the biennial International Rheumatology Conference in Israel. Study data from experimental models and patient samples demonstrated that CCL24, the target for CM-101, is overexpressed in skin and serum samples of diffuse SSc patients compared to healthy individuals. CCL24 levels also correlated with fibrotic biomarkers and disease progression. In an experimental mouse model of SSc, CM-101 profoundly reduced skin and lung fibrosis.
Professor Francesco Del Galdo of the University of Leeds presented "CCL24 as a Marker of Worse Prognosis in diffuse cutaneous SSc: a Promising Novel Biological Target" at the 7th Systemic Sclerosis World Congress. Professor Del Galdo’s findings support the role of CCL24 as a potential therapeutic target, demonstrating elevated serum levels of CCL24 in diffuse cutaneous SSc patients. High CCL24 serum levels were correlated with disease activity and worse prognosis as reflected by high fibrotic activity and deterioration of lung function over time in a longitudinal patient cohort.
Participated in the 32nd Annual Oppenheimer Healthcare Conference
At the Cantor Fitzgerald Rare Orphan Disease Summit, Dr. Adi Mor described how Chemomab’s CM-101 "pipeline in a product" strategy offers important synergies and efficiencies in the drug development process.
First Quarter 2022 Financial Highlights

Cash Position: Cash and cash equivalents were $57.5 million as of March 31, 2022, compared to $61.2 million as of December 31, 2021.
Research and Development (R&D) Expenses: R&D expenses were $2.7 million for the first quarter ended March 31, 2022, compared to $1.2 million for the same quarter in 2021.
General and Administrative (G&A) Expenses: G&A expenses were $2.6 million for the first quarter ended March 31, 2022, compared to $0.5 million for the same quarter in 2021. The current quarter figure includes a $0.9 million non-cash stock-based compensation payment.
Net Loss: Net loss was $5.1 million, or a net loss of $0.02 per basic and diluted share, for the first quarter ended March 31, 2022, compared to $1.7 million, or a net loss of $0.01 per basic and diluted share, for the quarter ended March 31, 2021. The weighted average number of Ordinary Shares outstanding, basic and diluted were 228,090,300 (equal to 11,404,515 American Depository Shares) and 156,751,771 (equal to 7,837,589 American Depository Shares) for the quarters ended March 31, 2022, and March 31, 2021, respectively.
For further details on the company’s financial results for the quarter ended March 31, 2022, refer to the Form 10-Q, which will be filed with the SEC today, May 12, 2022.

Conference Call
Chemomab management will host a conference call for investors today, Thursday, May 12, 2022, beginning at 8:00 a.m. Eastern Time to discuss these results and answer questions. Shareholders and other interested parties may participate in the conference call by clicking this Webcast link to access the live webcast or replay, or by dialing 877-407-9208 (in the U.S.) or 201-493-6784 (outside the U.S. and in Israel) and entering passcode 13728593. Ask for the Chemomab conference call. The call also will be webcast live on the company’s website at View Source

A replay of the call will be available on the company website for 90 days at www.chemomab.com.

X4 Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Corporate Update

On May 12, 2022 X4 Pharmaceuticals, (Nasdaq: XFOR), a leader in the discovery and development of novel oral CXCR4-targeted small molecule therapeutics to benefit people with rare immune system disorders, reported financial results for the first quarter ended March 31, 2022 (Press release, X4 Pharmaceuticals, MAY 12, 2022, View Source [SID1234614391]).

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"As we quickly approach top-line results from our 4WHIM pivotal Phase 3 trial for WHIM syndrome by the end of this year, the X4 team continues to identify and expand the breadth of individuals diagnosed with this rare disease. Initiatives aimed at supporting diagnosis and driving disease awareness, such as our PATH4WARD program offering free genetic sequencing to patients, and publishing peer-reviewed research to help define novel pathogenic genetic variants, are adding to the population of those known to be affected by WHIM and chronic neutropenia," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Specifically, to support those affected by chronic neutropenia, we are expanding the availability of genetic testing through PATH4WARD and evaluating patients with congenital, idiopathic, and cyclic chronic neutropenia in our ongoing Phase 1b study. We look forward to providing both clinical data and regulatory updates over the coming months on this important initiative."

Recent Highlights

Presented posters at the CIS 2022 Annual Meeting that detailed the PATH4WARD genetic testing program to aid in the diagnosis of WHIM syndrome and chronic neutropenia, and the characterization of a novel pathogenic CXCR4 variant (S341Y).
Presented preclinical data on mavorixafor’s ability to significantly enhance the tumor-cell killing activity of the leading commercial and clinical stage Bruton Tyrosine Kinase Inhibitors (BTKi) including ibrutinib, zanubrutinib, pirtobrutinib (LOXO-305) and nemtabrutinib (ARQ-531), at the 2022 AACR (Free AACR Whitepaper) Annual Meeting.
Completed enrollment in the ongoing Phase 1b study of mavorixafor in combination with ibrutinib in patients with Waldenström’s Macroglobulinemia (WM) whose tumors express mutations in MYD88 and CXCR4. A total of 16 patients were enrolled and dose escalation to the 600 mg dose of mavorixafor has been completed. Results from this study are expected to be reported during the second half of 2022.

First Quarter 2022 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $67.7 million in cash, cash equivalents, and restricted cash as of March 31, 2022. The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
Research and Development (R&D) Expenses were $14.1 million for the first quarter of 2022 as compared to $12.1 million for the comparable period in 2021. R&D expenses include $0.7 million and $0.6 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Selling, General and Administrative Expenses (SG&A) were $7.7 million for the first quarter of 2022 as compared to $5.8 million for the comparable period in 2021. SG&A expenses include $0.8 million and $0.7 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Net Loss: X4 reported a net loss of $22.0 million for the first quarter of 2022, as compared to $18.7 million for the comparable period in 2021. Net losses include $1.5 million and $1.3 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 4382059. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

Aptevo Therapeutics Reports First Quarter 2022 Financial Results and Business Highlights

On May 12, 2022 Aptevo Therapeutics Inc. ("Aptevo" or the "Company") (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported financial results and business highlights for the quarter ended March 31, 2022 (Press release, Aptevo Therapeutics, MAY 12, 2022, View Source [SID1234614410]).

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Business Highlights

Earned and collected a $10 million non-dilutive milestone payment related to 2021 sales of RUXIENCE. Additionally, based on RUXIENCE 2021 fourth quarter and full-year sales results, the Company is optimistic about the potential to earn additional non-dilutive milestones totaling $22.5 million over the next two years
Reported that two patients with relapsed/refractory acute myeloid leukemia (AML) in its on-going Phase 1b trial evaluating adult patients with AML, achieved transplant-eligible status, and have received allogeneic stem cell transplants:
One patient received APVO436 in a monotherapy cohort of the trial and experienced significant reduction in bone marrow blasts
Another patient received APVO436 in a combination therapy cohort of the trial and experienced a complete remission
Continued enrollment in the Company’s Phase 1b expansion trial evaluating APVO436 in adult patients with AML
Presented preclinical data for APVO442 in a poster session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting
The poster highlighted the potential of APVO442 to treat prostate cancer indications such as metastatic castration-resistant prostate cancer with increased benefit and decreased side effects relative to other potential therapeutics in the bispecific category
APVO442 is a bispecific therapeutic candidate targeting prostate-specific membrane antigen (PSMA) and CD3, designed to redirect the patient’s T cell-mediated tumor-fighting responses against PSMA-expressing solid tumors (prostate cancer)
"The first quarter was productive as we continued to advance both clinical and pre-clinical assets, all of which are based on the Company’s proprietary ADAPTIR and ADAPTIR-FLEX platforms. Our Phase 1b expansion trial continues to enroll patients and we reported that two patients advanced to transplant after receiving APVO436 – one as a monotherapy and the other as a combination therapy. We were also happy to report we earned a $10 million milestone payment related to 2021 sales of RUXIENCE. Receipt of this non-dilutive payment underscores our ongoing commitment to delivering value to shareholders," said Marvin White, President and CEO of Aptevo. "Looking ahead, we continue to progress the ALG.APV-527 program toward the clinic and remain on track for an IND submission and clinical trial initiation, later this year. We also plan to release interim data from the APVO436 expansion trial and announce the addition of a new molecule to the preclinical pipeline later this year."

First Quarter 2022 Financial Results Summary

Cash Position: Aptevo had cash and cash equivalents as of March 31, 2022 totaling $36.3 million, including restricted cash of $1.3 million. $0.5 million of the restricted cash was released in April 2022 and the remaining $0.8 million is expected to be released over the next twelve months.

Royalty Revenue: For the three months ended March 31, 2022, royalty revenue increased by $0.7 million, or 29%, to $3.1 million from $2.4 million for March 31, 2021. Royalty revenue relates to the royalty from Pfizer on global net sales of RUXIENCE (rituximab-pvvr), a biosimilar to the drug RITUXAN, launched by Pfizer in early 2020.Due to the nature of the transaction, which includes a cap on HCR’s return from royalties, constituting continuing involvement under the Collaboration and License Agreement originally between Trubion and Wyeth, we continue to recognize royalty revenue on net sales of RUXIENCE and record the royalty payments to HCR as a reduction of the liability when paid. As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the HCR royalty purchase agreement. RUXIENCE is a trademark of Pfizer; RITUXAN is a trademark of Biogen.

Research and Development Expenses: For the three months ended March 31, 2022, research and development expenses decreased by $0.5 million, to $4.9 million from $5.4 million for March 31, 2021. The decrease was primarily due to lower spending on preclinical projects and employee costs. The decrease was partially offset by higher spending on our APVO436 clinical trial as we continue to advance that trial and continue to dose patients in our Phase 1b Expansion program.

General and Administrative Expenses: For the three months ended March 31, 2022 and 2021, general and administrative expenses were $3.9 million.

Other Expense, Net: Other expense, net consists primarily of gains or losses realized on foreign currency revaluation, costs related to debt extinguishment, accrued exit fees on debt, non-cash interest on financing agreements, and interest on debt. Other expense, net was $2.3 million for the three months ended March 31, 2022 and $0.8 million for the three months ended March 31, 2021. The increase in other expense, net is primarily related non-cash interest expense for the HCR royalty purchase agreement. The increase was partially offset by lower interest expense for the MidCap Credit Agreement due to payments made towards principal.

Discontinued Operations: For the three months ended March 31, 2022, we collected $0.2 million in deferred payments from Medexus related to IXINITY sales. For the three months ended March 31, 2021, we collected $0.2 million related to the sale of hyperimmune business to Saol as a result of the collection of certain accounts receivable and a deferred payment of $0.2 million received from Medexus related to IXINITY sales. Pursuant to our LLC Purchase Agreement, the rate for deferred payments will increase from 2% to 5% of net sales no later than June 30, 2022.

Net Loss: Aptevo’s net loss for the three months ended March 31, 2022 was $7.7 million or $1.55 per share, as compared to a net loss of $7.3 million or $1.64 per share for the corresponding period in 2021.

Liability Related to Sale of Future Royalties: We treat the Royalty Purchase Agreement with HCR as a debt financing, amortized under the effective interest rate method over the estimated life of the related expected royalty stream. The liabilities related to sale of future royalties and the debt amortization are based on our current estimates of future royalties expected to be paid over the life of the arrangement. We will periodically assess the expected royalty payments using projections from external sources. To the extent our estimates of future royalty payments are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will adjust the effective interest rate and recognize related non-cash interest expense on a prospective basis. We are not obligated to repay the proceeds received under the Royalty Purchase Agreement with HCR.