On May 5, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2022 and reaffirmed its financial guidance for the year (Press release, Ultragenyx Pharmaceutical, MAY 5, 2022, View Source [SID1234613752]).

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"Product based revenue continues to grow driven by increased traction for Crysvita in Latin America," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In the clinical portfolio, we are now dosing patients in three of our four concurrent pivotal programs and are on track for updated data from the Phase 1/2 for Angelman syndrome mid-year."

First Quarter 2022 Financial Results

Net Revenues
For the first quarter of 2022, Ultragenyx reported $79.9 million in total revenue. Ultragenyx recognized $54.6 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $45.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $9.4 million. Total royalty revenue related to European Crysvita sales was $4.8 million. Dojolvi (triheptanoin) product sales in the first quarter of 2022 were $12.4 million. Mepsevii (vestronidase alfa) product sales for the first quarter of 2022 were $4.9 million.

Total revenue for the first quarter of 2022 also includes $3.2 million related to technical assistance following the successful completion of technology transfer activities with Daiichi Sankyo. This compares to total revenue in the first quarter of 2021, which includes $42.8 million related to the technology transfer services which were ongoing at the time.

Operating Expenses
Total operating expenses for the first quarter of 2022 were $216.6 million, including non-cash stock-based compensation of $29.4 million.

Net Loss
For the first quarter of 2022, Ultragenyx reported net loss of $152.3 million, or $2.19 per share basic and diluted, compared with a net loss for the first quarter of 2021 of $136.1 million, or $2.03 per share, basic and diluted. Net cash used in operations for the quarter ended March 31, 2022 was $117.5 million.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were $813.8 million as of March 31, 2022.

2022 Financial Guidance

The company continues to expect 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.

First Quarter 2022 Revenue and Selected Financial Data Tables

Recent Updates

Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): European marketing authorization transitioned to Ultragenyx, reimbursement processes initiated
The European Commission has completed transfer of the marketing authorization of Evkeeza from Regeneron. Ultragenyx is preparing reimbursement dossiers for various national health authorities in Europe. Ultragenyx received rights to commercialize and distribute Evkeeza in countries outside the U.S. in January 2022.

UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing has been initiated for the Phase 2/3 Orbit study; Phase 2 study in children under age five planned for second half of 2022
Ultragenyx has begun dosing patients in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI ages five to <26 years. A dosing update on the Phase 2 portion of the Orbit study and transition to Phase 3 is expected in the second half of 2022.

In addition, Ultragenyx intends to initiate an additional study in children with OI under age 5 years in the second half of 2022 and will continue to evaluate adult patients who were previously treated in the ASTEROID study, a Phase 2b study conducted by our partner Mereo.

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 GlucoGene study dosing patients
Dosing and enrollment of the Phase 3 study of DTX401 is ongoing. The pivotal GlucoGene study has a 48-week primary efficacy analysis period and the company plans to enroll approximately 50 patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control.

UX701 for Wilson Disease: Cyprus2+ pivotal Phase 1/2/3 study dosing patients
The company is dosing patients in the Phase 1/2 stage of the seamless Phase 1/2/3 Cyprus2+ study of UX701. During the first stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary efficacy endpoints are change in 24-hour urinary copper concentration and percent reduction in standard of care medication by Week 52.

DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 eNH3ance study expected to initiate in mid-2022
Ultragenyx expects to initiate the Phase 3 eNH3ance study of DTX301 in patients with OTC in mid-2022. The 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

GTX-102 for Angelman Syndrome: Patients continue to be treated in the Phase 1/2 study in Canada and the U.K. and under a separate protocol in the U.S.
Dosing is ongoing in cohorts 4 and 5 of the Phase 1/2 study in the U.K. and Canada, as well as for four additional patients in the U.S. under a separate protocol. To date, no treatment-related serious adverse events or lower extremity weakness adverse events have been observed in these patients. An interim update on the safety and efficacy of GTX-102 is planned for mid-2022.

UX053 for Glycogen Storage Disease Type III (GSDIII) Debrancher Deficiency: Phase 1/2 study currently dosing patients; Preliminary data from first part of study and initiation of second part of study anticipated in second half of 2022
Ultragenyx has begun to dose patients in the two-part Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of UX053 in adults age 18 years and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. Preliminary data from the Part 1 single ascending dose phase of the study is expected in the second half of the year.

1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 5, 2022, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2022 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 7951356. The replay of the call will be available for one year.

On May 5, 2022 NextCure, Inc. (Nasdaq: NXTC), a clinical-stage biopharmaceutical company committed to discovering and developing novel, first-in-class immunomedicines to treat cancer and other immune-related diseases, reported first quarter 2022 financial results and provided a business update (Press release, NextCure, MAY 5, 2022, View Source [SID1234613819]).

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"In the first quarter, we continued to advance our pipeline of four product candidates towards key clinical milestones," said Michael Richman, NextCure’s President and Chief Executive Officer. "In the second half of 2022, we anticipate clinical trial updates for NC318, NC410, and NC762, as well as an IND filing for NC525. We also expect our quarter-end cash position of $201.3 million will fund our operations into the first quarter of 2024."

Expected Milestones

We believe we are on track to meet the following milestones during the second half of 2022:

NC318 Phase 2 update: fourth quarter of 2022 (Amended Phase 2 with patient selection and increased dosing).
NC318 anti-PD-1 combo initial data second half of 2022 (Yale University Investigator-Initiated trial).
NC410 Phase 1 update: second half of 2022.
NC762 Phase 1 initial data: second half of 2022.
NC525 Investigational New Drug Application (IND) filing: fourth quarter of 2022.
Financial Guidance

Based on its current research and development plans, NextCure expects its existing cash, cash equivalents and marketable securities will enable it to fund operating expenses and capital expenditures into the first quarter of 2024.

Financial Results for First Quarter March 31, 2022

Cash, cash equivalents, and marketable securities, excluding restricted cash, as of March 31, 2022, were $201.3 million as compared with $219.6 million as of December 31, 2021. The decrease of $18.3 million primarily reflects cash used to fund operations.
Research and development expenses were $15.0 million for the quarter ended March 31, 2022 as compared to $12.4 million for the quarter ended March 31, 2021. The increase of $2.6 million was driven primarily by additional clinical and lab-related costs.
General and administrative expenses were $5.8 million for the quarter ended March 31, 2022 as compared with $4.9 million for the quarter ended March 31, 2021. The increase of $0.9 million was primarily related to additional personnel-related costs.
Net loss was $20.6 million for the quarter ended March 31, 2022 as compared with $16.5 million for the quarter ended March 31, 2021. The changes in net loss from the previous year’s quarter were primarily due to increased research and development expenses.

On May 5, 2022 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported financial results for the three months ended March 31, 2022 and provided an update on key milestones (Press release, Lexicon Pharmaceuticals, MAY 5, 2022, View Source [SID1234613619]).

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"We plan to resubmit our New Drug Application (NDA) for sotagliflozin for the treatment of heart failure this month having received confirmation from the FDA in late April that it is aligned with our resubmission plans. With the recently announced loan facility, we have committed access to sufficient resources to support our commercial preparations for launch in the first half of 2023," said Lonnel Coats, Lexicon’s chief executive officer. "In addition, we are looking forward to completing and announcing top-line results by the end of this quarter for our proof-of-concept Phase 2 study of LX9211 in diabetic peripheral neuropathic pain."

First Quarter Highlights

Sotagliflozin

A new analysis of data from the SCORED Phase 3 clinical trial of sotagliflozin was presented at the American College of Cardiology’s 71st Annual Scientific Session. The analysis showed that treatment with sotagliflozin resulted in a significant reduction in major adverse cardiovascular events (MACE) of cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke in the entire cohort as compared to placebo, with consistent results in patients with and without prior cardiovascular disease.
New guidelines for the management of heart failure were issued jointly by the American Heart Association, the American College of Cardiology and the Heart Failure Society of America, recommending the use of SGLT2 inhibitors in the prevention and treatment of heart failure with the highest-level recommendations among classes of therapy in each category.
Lexicon entered into a loan facility with Oxford Finance LLC that provides up to $150 million in borrowing capacity designed primarily to support commercial preparations and the potential launch of sotagliflozin in heart failure. An initial $25 million tranche was funded at closing.
LX9211

Top-line results for the ongoing Phase 2 proof-of-concept studies of LX9211 are expected by the end of the second quarter of 2022 for the treatment of diabetic peripheral neuropathic pain (RELIEF-DPN-1) and the third quarter of 2022 for the treatment of post-herpetic neuralgia (RELIEF-PHN-1).
First Quarter 2022 Financial Highlights

Research and Development (R&D) Expenses: Research and development expenses for the first quarter of 2022 increased to $14.9 million from $12.6 million for the corresponding period in 2021, primarily due to increases in professional and consulting costs related to the company’s NDA for sotagliflozin.

Selling, General and Administrative (SG&A) Expenses: Selling, general and administrative expenses for the first quarter of 2022 increased to $8.5 million from $8.3 million for the corresponding period in 2021, primarily due to increases in personnel and external expenses relating to preparations for the commercial launch of sotagliflozin.

Net Loss: Net loss for the first quarter of 2022 was $23.5 million, or $0.16 per share, as compared to a net loss of $21.0 million, or $0.15 per share, in the corresponding period in 2021. For the first quarters of 2022 and 2021, net loss included non-cash, stock-based compensation expense of $2.8 million and $2.9 million, respectively.

Cash and Investments: As of March 31, 2022, Lexicon had $86.5 million in cash and investments, as compared to $86.7 million as of December 31, 2021.

Conference Call and Webcast Information

Lexicon management will hold a live conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a general business update. The dial-in number for the conference call is 888-645-5785 (U.S./Canada) or 970-300-1531 (international). The conference ID for all callers is 3334447. The live webcast and replay may be accessed by visiting Lexicon’s website at www.lexpharma.com/events. An archived version of the webcast will be available on the website for 14 days.

On May 5, 2022 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies and cytokines for the treatment of cancer and autoimmune diseases, reported financial results for the first quarter ended March 31, 2022 and provided a review of recent business and clinical highlights (Press release, Xencor, MAY 5, 2022, View Source [SID1234613648]).

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"Our XmAb Fc domains and protein engineering expertise have enabled a broad portfolio of clinical-stage drug candidates, which we and our partners are investigating across many therapeutic areas. Internally we are focused on efficiently using our resources to advance the most promising clinical-stage programs, as well as the next wave of Xencor innovations to enter the clinic – more reduced-potency XmAb cytokines, CD28 T cell engagers and 2+1 CD3 T cell engagers," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "Today we are pleased to announce we have dosed the first patient in a potentially registration-enabling study for plamotamab to evaluate the chemotherapy-free, triple combination with tafasitamab and lenalidomide for patients with an aggressive form of non-Hodgkin’s lymphoma."

Dr. Dahiyat continued, "As we progress through the year, we remain on track to present additional clinical data from our vudalimab and plamotamab programs and initial data from our IL2-Fc autoimmune program, XmAb564, in healthy volunteers. In addition, we will present data from our XmAb104 program at the ASCO (Free ASCO Whitepaper) Annual Meeting. In 2022, we also expect to start Phase 1 studies for XmAb819, our ENPP3 x CD3 bispecific antibody for renal cell carcinoma engineered with our multivalent 2+1 format for greater tumor selectivity, and for XmAb808, our B7-H3 x CD28 bispecific antibody. This broad development pipeline is supported by the recent strong revenue from our marketed partnered programs and our robust financial position."

Recent Portfolio Highlights

Plamotamab (CD20 x CD3): Xencor has dosed the first patient in a potentially registration-enabling Phase 2 study, evaluating plamotamab in combination with tafasitamab plus lenalidomide, in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The study consists of two parts, a safety run-in intended to establish the safety of the triple combination (Part 1) and a two-arm, open-label cohort where patients will be randomized to receive either the triple combination or tafasitamab plus lenalidomide (Part 2). Xencor is conducting the clinical study in collaboration with MorphoSys AG and Incyte Corporation. Tafasitamab is co-marketed by Incyte and MorphoSys under the brand name Monjuvi in the United States and is marketed by Incyte under the brand name Minjuvi in the European Union. Incyte has exclusive commercialization rights to tafasitamab outside the U.S.

Expansion cohorts in the Phase 1 study of plamotamab are actively recruiting patients with DLBCL and follicular lymphoma (FL) and are dosing using the recommended Phase 2 regimen to further evaluate the safety and efficacy of plamotamab as a monotherapy. Subcutaneous administration of plamotamab will be introduced this year, and the Company plans to present data from the expansion cohorts in the second half of 2022.
Vudalimab (PD-1 x CTLA-4): Xencor is supporting two newly initiated investigator-sponsored studies of vudalimab, in patients with advanced biliary tract cancers and in patients with advanced rare cancers.

The Company is currently enrolling a Phase 2 study in patients with metastatic castration-resistant prostate cancer (mCRPC), in which vudalimab is being evaluated as a monotherapy or in combination with chemotherapy or a PARP inhibitor depending on the tumor’s molecular subtype. The Company plans to present initial data from the study in the second half of 2022 and is currently initiating a Phase 2 study evaluating vudalimab monotherapy in patients with advanced pelvic tumors, including clinically defined high-risk mCRPC and certain gynecologic malignancies.
XmAb306 (IL15/IL15Rα-Fc): Xencor’s co-development partner Genentech, a member of the Roche Group, has initiated a Phase 1 study to evaluate the combination of XmAb306 (RO7310729) and daratumumab, an anti-CD38 monoclonal antibody, in patients with relapsed/refractory multiple myeloma.

XmAb306 is a potency-reduced IL15/IL15Rα-Fc fusion protein targeting NK and T cells for the treatment of patients with cancer. In an ongoing Phase 1 dose-escalation study of XmAb306 in patients with advanced solid tumors, XmAb306 has promoted high levels of sustained NK cell expansion and evidence of peripheral effector T cell proliferation. Additional studies of XmAb306 in combination with other therapeutic agents are also being planned.
XmAb104 (PD-1 x ICOS): An abstract with initial dose-escalation data from the Phase 1 study of XmAb104 in patients with advanced solid tumors was accepted for a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2022.
Preclinical Data Presentations: New data from two preclinical-stage XmAb cytokine programs, an IL18-Fc (XmAb143) and a LAG-3 targeted IL15/IL15Rα-Fc, were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.
Tidutamab (SSTR2 x CD3) and XmAb841 (CTLA-4 x LAG-3): The Company will stop internal development of the tidutamab and XmAb841 programs. Neither program demonstrated a competitive clinical profile in recent trials, and the Company has decided to focus resources on new clinical programs. The Company will continue to support patients currently enrolled and being treated.
Progress Across Partnered Programs

Vir Biotechnology, Inc.: In the first quarter of 2022, Xencor recognized $70.3 million in royalty revenue under the Company’s agreement with Vir. Sotrovimab, an antibody that targets the SARS-CoV-2 virus and incorporates Xencor’s Xtend Fc domain for longer duration of action, has been made available by Vir and its partner Glaxo Wellcome UK Limited and GlaxoSmithKline Biologicals S.A. Due to the rapid emergence of the sotrovimab-resistant Omicron BA.2 subvariant in the first quarter, sotrovimab’s authorization was ended in all U.S. regions.
Alexion Pharmaceuticals, Inc.: In April 2022, Ultomiris (ravulizumab-cwvz), which incorporates an Xtend Fc domain, was approved by the U.S. Food and Drug Administration for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. In the first quarter of 2022, Xencor earned $6.1 million from Alexion on net sales of Ultomiris.
Astellas Pharma, Inc.: Astellas has advanced ASP2138, a CLDN18.2 x CD3 XmAb bispecific antibody, into Phase 1 clinical development for the treatment of patients with gastric, gastroesophageal, and pancreatic cancers. Under the Astellas agreement, Xencor applied XmAb bispecific Fc technology to an antigen pair provided by Astellas.
Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc. Monjuvi and Minjuvi are registered trademark of MorphoSys AG.

Financial Results for the First Quarter Ended March 31, 2022

Cash, cash equivalents, receivables and marketable debt securities totaled $683.6 million as of March 31, 2022, compared to $664.1 million on December 31, 2021. During the first quarter of 2022, the Company received milestone payments and royalties from partners of $83.7 million, which offset spending on operations and resulted in a higher cash balance relative to the 2021 year-end amount.

Revenues for the first quarter ended March 31, 2022 were $85.5 million, compared to $34.0 million for the same period in 2021. Total revenues earned in the first quarter of 2022 included revenues earned from Xencor’s Janssen collaboration, milestone revenue from Astellas, and royalties from the Alexion, MorphoSys and Vir agreements, compared to revenue earned from the Janssen collaboration and royalties and milestones from the Alexion and MorphoSys agreements in the first quarter of 2021.

Research and development expenses for the first quarter ended March 31, 2022 were $47.8 million, compared to $41.4 million for the same period in 2021. Increased research and development spending for first quarter of 2022 compared to 2021 reflects increased spending on the Company’s new development programs including XmAb808 (B7-H3 x CD28) and XmAb662 (IL-12).

General and administrative expenses for the first quarter ended March 31, 2022 were $11.3 million, compared to $8.2 million in the same period in 2021. Increased general and administrative spending for the first quarter of 2022 compared to 2021 reflects increased staffing and additional lease expenses.

Non-cash, stock-based compensation expense for the first quarter ended March 31, 2022 was $10.8 million, compared to $8.3 million for the same period in 2021.

Net income for the first quarter ended March 31, 2022 was $23.6 million, or $0.39 on a fully diluted per share basis, compared to a net loss of $2.5 million, or $(0.04) on a fully diluted per share basis, for the same period in 2021. Net income reported for the first quarter of 2022 compared to net loss for the same period in 2021 is primarily due to increased royalties from partners in 2022.

The total shares outstanding were 59,529,192 as of March 31, 2022, compared to 58,221,953 as of March 31, 2021.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations through the end of 2025. While future royalty revenues are uncertain, based on recent guidance from GSK, Xencor expects the amount of royalty revenue that it receives from sales of sotrovimab to substantially decline in future reporting periods. The Company expects to end 2022 with between $500 million and $550 million in cash, cash equivalents, receivables and marketable debt securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss the first quarter 2022 financial results and provide a corporate update.

The live call may be accessed by dialing (877) 359-9508 for domestic callers or +1 (224) 357-2393 for international callers and referencing conference ID number 2583486. A live webcast of the conference call will be available online from the Investors section of the Company’s website at www.xencor.com. The webcast will be archived on the company’s website for 30 days.

On May 5, 2022 Aileron Therapeutics (Nasdaq: ALRN), a chemoprotection oncology company that aspires to make chemotherapy safer and thereby more effective to save more patients’ lives today, reported financial results and business highlights for the first quarter ended March 31, 2022 (Press release, Aileron Therapeutics, MAY 5, 2022, View Source [SID1234613663]).

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"Building on our positive proof-of-concept trial last year in patients with small cell lung cancer treated with topotecan, we view 2022 as a year of validation and execution for Aileron as we seek to bring selective chemoprotection to all patients with p53-mutated cancers regardless of the type of cancer or chemotherapy. Our ongoing NSCLC clinical trial and upcoming breast cancer clinical trial are designed to help us further understand ALRN-6924’s chemoprotective effect against chemotherapy-induced hematologic toxicities as well as other toxicities," said Manuel Aivado, M.D., Ph.D., President and Chief Executive Officer of Aileron. "We believe that evaluating ALRN-6924 across these two major cancer types treated with different chemotherapies that carry distinct toxicity profiles may provide us with an expanded regulatory opportunity based on established precedent for other supportive care drug approvals."

Nearly 1 million patients are diagnosed with a p53-mutated cancer in the US each year. Aileron is pioneering a precision medicine-based approach known as selective chemoprotection to exclusively treat patients with p53-mutated cancers receiving chemotherapy. ALRN-6924 is designed to selectively protect these patients’ healthy cells from chemotherapy without interfering with chemotherapy’s effects on cancer cells. Aileron’s vision is to bring chemoprotection to all patients with p53-mutated cancers regardless of the type of cancer or chemotherapy.

First Quarter 2022 Highlights and Recent Updates

On track to report interim results from ongoing randomized, double-blind, placebo-controlled Phase 1b clinical trial of ALRN-6924 in advanced p53-mutated NSCLC in June 2022. Aileron plans to enroll 60 patients with advanced p53-mutated NSCLC undergoing treatment with first-line carboplatin plus pemetrexed with or without immune checkpoint inhibitors. Patients are randomized 1:1 to receive carboplatin/pemetrexed plus 0.3 mg/kg ALRN-6924 or placebo for four 21-day treatment cycles.

The company anticipates reporting interim results on the first 20 patients enrolled in June 2022 and topline results on 60 patients in 4Q 2022. The interim readout will include an initial evaluation of the trial’s composite endpoint, composed of the proportion of treatment cycles free of severe toxicities, blood transfusions, and the use of growth factors, as well as dose reductions or dose delays, in patients treated with ALRN-6924 versus placebo.
Completed blinded safety evaluation of NSCLC clinical trial. In 1Q 2022, Aileron conducted a blinded safety evaluation of the first ten patients enrolled in the NSCLC clinical trial who completed the first cycle of treatment with ALRN-6924 and carboplatin plus pemetrexed. The evaluation did not identify any safety concerns, consistent with ALRN-6924’s previously demonstrated safety and tolerability profile.
On track to initiate Phase 1b randomized trial of ALRN-6924 in patients with p53-mutated ER+/HER2- neoadjuvant breast cancer in 2Q 2022. The planned breast cancer trial will evaluate ALRN-6924’s protection against chemotherapy-induced bone marrow toxicities, as well as other toxicities, including alopecia, in patients with p53-mutated ER+/HER2- breast cancer treated with a doxorubicin + cyclophosphamide and docetaxel chemotherapy regimen. The Phase 1b trial is anticipated to enroll up to 30 patients in a parallel group design trial with a dose expansion cohort.
Announced management team updates. In April 2022, Aileron announced the appointment of Christopher Zergebel as Vice President, Program Management and Clinical Operations. Mr. Zergebel previously served as Vice President, R&D Services at Taiho Oncology, Inc., overseeing clinical project management, clinical operations, data management, medical writing, and clinical supplies through all phases of clinical development and through regulatory approvals. Aileron also recently announced the pending departure of Richard Wanstall, Chief Financial Officer. Mr. Wanstall joined Aileron in July 2018 and has been instrumental in Aileron’s successful evolution to a chemoprotection oncology company and helped expand Aileron’s finance, operations, and human resources infrastructure. Prior to joining Aileron, Mr. Wanstall served for four years as Vice President, Finance at Moderna. Aileron has launched a formal search process to identify Mr. Wanstall’s successor.
Upcoming virtual investor event on Thursday, May 19th. Aileron’s management team, joined by guest speakers, will host an investor event to discuss ALRN-6924’s revolutionary potential as the first precision medicine-based supportive care drug, the landscape of chemotherapy-induced toxicities, and the company’s clinical trials and planned 2022 data readouts. To register, visit View Source
First Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents, and investments on March 31, 2022, were $38.1 million, compared to $45.9 million on December 31, 2021. Based on its current operating plan, the company expects its existing cash, cash equivalents, and investments will fund operations into the fourth quarter of 2023.
Research and Development (R&D) Expenses: R&D expenses for the quarter ended March 31, 2022, were $5.9 million, compared to $4.3 million for the quarter ended March 31, 2021. The $1.6 million increase in R&D spending primarily resulted from $0.4 million of increased spending for clinical development of ALRN-6924 related to the Phase 1b clinical trial in patients with advanced p-53 mutated NSCLC and startup activities related to the Phase 1b randomized trial of ALRN-6924 in patients with p53-mutated ER+/HER2- neoadjuvant breast cancer, partially offset by lower spending for the ongoing Phase 1 trial in healthy human volunteers and the completion of the Phase 1b trial in small cell lung cancer in 2021. R&D expenses also increased in the first quarter of 2022 compared to the first quarter of 2021 resulting from increased ALRN-6924 manufacturing activities.
General and Administrative (G&A) Expenses: G&A expenses for the quarter ended March 31, 2022, were $2.5 million compared to $2.7 million for the quarter ended March 31, 2021. The decrease in general and administrative expenses was the result of lower professional services fees during the first quarter of 2022 as compared to the first quarter of 2021.
Net Loss: Net loss for the quarter ended March 31, 2022, was $8.4 million, compared to $7.0 million for the corresponding quarter in 2021. The basic and diluted net loss per share for the first quarter of 2022 was $0.09 compared to $0.08 for the first quarter of 2021.