On May 9, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported its financial results for the first quarter ended March 31, 2022 (Press release, Castle Biosciences, MAY 9, 2022, View Source [SID1234613966]).

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"We saw significant progress and execution on our growth initiatives in the first quarter, with record test report volume," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "We remain laser-focused on the execution of our three operational growth pillars — our strong core dermatology business, pipeline initiatives and strategic opportunities — and are pleased with our results.

"In 2021, we made commercial investments, including a significant sales team expansion, in our proprietary skin cancer test business to further our position of strength in 2022 and beyond. We believe these investments, coupled with the demonstrated utility of our tests, are the drivers behind the nearly 70% increase in total test volume during the first quarter.

"We reported initial proof of concept data on our sample collection method for our pipeline test for inflammatory skin diseases, and we remain on track to launch this test in 2025, which would add an additional $1.9 billion to our estimated U.S. total addressable market (TAM), if successful.

"We are encouraged with the initial progress on our gastroenterology franchise and TissueCypher Barrett’s Esophagus (BE) test. We successfully hired and trained our commercial team for this test, who were in the field in February 2022, and received new Advanced Diagnostic Laboratory Test (ADLT) status from the Centers for Medicare & Medicaid Services (CMS) in March 2022. Further, the recent acquisition of AltheaDx and the IDgenetix pharmacogenomic test (PGx), in alignment with our M&A strategy to diversify our portfolio and create near- and long-term revenue growth opportunity, adds approximately $5.0 billion to our estimated U.S. TAM. IDgenetix now has expanded Medicare coverage, from depression only to seven additional mental health conditions. We are entering the remainder of 2022 with an estimated in-market U.S. TAM of just under $8 billion, for all our franchises combined.

"We believe our progress is only possible through the dedication of our Castle team, who allows us to execute at a high level, further our impact on patient care and position ourselves for continued value creation."

First Quarter Ended March 31, 2022, Financial and Operational Highlights

Revenues were $26.9 million, an 18% increase compared to $22.8 million during the same period in 2021. Included in revenue for the current year was $0.6 million related to tests delivered in prior periods. Revenue for the same quarter last year included $5.3 million related to tests delivered in prior periods.
Adjusted revenues, which exclude the effects of revenue adjustments related to tests delivered in prior periods, were $26.3 million, an 50% increase, compared to $17.5 million for the same period in 2021.
Delivered 8,627 total test reports in the first quarter of 2022, an increase of 68% compared to 5,142 in the same period of 2021:
DecisionDx-Melanoma test reports delivered in the quarter were 6,023, compared to 4,060 in the first quarter of 2021, an increase of 48%.
DecisionDx-SCC test reports delivered in the quarter were 1,142, compared to 527 in the first quarter of 2021, an increase of 117%.
myPath Melanoma and DecisionDx DiffDx-Melanoma (Castle’s comprehensive diagnostic offering) aggregate test reports delivered in the quarter were 950, compared to 218 in the first quarter of 2021, an increase of 336%.
DecisionDx-UM test reports delivered in the quarter were 456, compared to 337 in the first quarter of 2021, an increase of 35%.
TissueCypher Barrett’s Esophagus test reports delivered in the quarter were 56.
Gross margin for the quarter ended March 31, 2022, was 72%, and adjusted gross margin was 77%.
Operating cash flow was $(21.4) million, compared to $(3.6) million for the same period in 2021, and adjusted operating cash flow was $(21.4) million, compared to $(5.5) million for the same period in 2021.
Net loss for the first quarter, inclusive of non-cash stock-based compensation expense of $8.4 million, was $(24.6) million, compared to $(4.3) million for the same period in 2021.
Adjusted EBITDA for the first quarter was $(11.4) million, compared to $0.9 million for the same period in 2021.
Cash and Cash Equivalents

As of March 31, 2022, the Company’s cash and cash equivalents totaled $309 million.

2022 Revenue Guidance

Castle Biosciences is increasing its previously issued guidance for anticipated total revenue in 2022. The Company now anticipates generating $118-123 million in total revenue in 2022, compared to the previously provided guidance of $115-120 million. This includes expected revenue from the TissueCypher Barrett’s Esophagus test, acquired in December 2021, and the IDgenetix pharmacogenomics test for mental health conditions, acquired in April 2022.

First Quarter and Recent Accomplishments and Highlights

Dermatology

In April, the Company announced new real-world data from its ongoing collaborative study with the National Cancer Institute (NCI). This new data showed that patients who received DecisionDx-Melanoma test results in addition to traditional clinicopathologic factors, as part of their clinical care, had improved survival compared to patients who were not tested (that is, their clinician could only rely upon available traditional clinicopathologic factors), with a 27% (hazard ratio (HR)=0.73, p=0.028) and 21% (HR=0.79, p=0.006) MSS (melanoma specific survival) and OS (overall survival) survival benefit compared to matched patients who were not tested, respectively. The data was shared in a poster presentation at the 18th European Association of Dermato-Oncology (EADO) Congress. See the Company’s news release from April 21, 2022, for more information.
Castle’s U.S. Federal Supply Schedule (FSS) contract with the Veterans Health Administration (VHA) was expanded to include coverage for the Company’s entire skin cancer test portfolio, effective April 15, 2022. Castle’s expanded U.S. FSS contract now includes DecisionDx-SCC, DecisionDx DiffDx-Melanoma, myPath Melanoma and DecisionDx-CMSeq, in addition to DecisionDx-Melanoma. Castle was awarded its first U.S. FSS contract in August 2021 for DecisionDx-Melanoma. See the Company’s news release from April 29, 2022, for more information.
In April, the Company gave a poster presentation highlighting data and concluding that its non-invasive skin scraping technique produces sufficient ribonucleic acid (RNA) to assess reproducible gene expression for its inflammatory skin disease pipeline test. The poster was presented at the 4th Annual Revolutionizing Atopic Dermatitis Conference. The Company expects to launch this pipeline test by the end of 2025. See the Company’s news release from April 18, 2022, for more information.
In March, the Company announced new data further demonstrating the performance of DecisionDx-Melanoma and i31-SLNB to provide improved risk prediction of sentinel lymph node (SLN) positivity, compared to using T-stage factors alone, in patients with cutaneous melanoma. In the study, the DecisionDx-Melanoma test outperformed T-stage in identifying patients with low-risk tumors who could forgo SLN biopsy, with an Area Under the Curve of 0.89 versus 0.78 for T-stage in patients with T1-T2 tumors, indicating that DecisionDx-Melanoma provides improved predictions compared to those of the T-stage system. See the Company’s news release from March 11, 2022, for more information.
Uveal Melanoma

In January, the Company announced the publication of a study in Ocular Oncology and Pathology demonstrating that the combined application of DecisionDx-UM, DecisionDx-PRAME and DecisionDx-UMSeq allows for highly accurate analysis of RNA and DNA from a single biopsy sample for patients with uveal melanoma (UM). DecisionDx-UMSeq is Castle’s 7-gene test that uses next-generation sequencing to identify somatic mutations relevant to UM. This information, together with results from the DecisionDx-UM gene expression profile test, is designed to help build a comprehensive genomic profile of an individual UM tumor from a single biopsy, which can then be used to inform patient care. See the Company’s news release from Jan. 12, 2022, for more information.
Gastroenterology

In March, the Company announced that CMS granted ADLT status for the TissueCypher Barrett’s Esophagus test, effective March 24, 2022. TissueCypher is Castle’s prognostic test designed to predict future development of high-grade dysplasia and/or esophageal cancer in patients with BE. ADLT status requires that a clinical diagnostic laboratory test provide new clinical diagnostic information that cannot be obtained from any other test or combination of tests, among other criteria.1 The announcement of ADLT status for TissueCypher confirms that the test meets these criteria established by CMS for laboratory tests under the Protecting Access to Medicare Act of 2014 (PAMA). See the Company’s news release from March 29, 2022, for more information.
In April, the company announced an independent, peer-reviewed article published in Clinical Gastroenterology and Hepatology. The study reinforces the ability of TissueCypher to significantly improve predictions of progression to esophageal cancer in patients with BE, compared to predictions based on traditional clinicopathologic variables alone, allowing for more informed disease management decisions. See the Company’s news release from April 27, 2022, for more information.
Mental Health

In April, the Company diversified and expanded its portfolio into the mental health market with the acquisition of AltheaDx and the IDgenetix PGx test for mental health conditions. IDgenetix has been reimbursed by Medicare for depression since the fall of 2020, and in a randomized, controlled clinical-use trial demonstrated clinical utility over standard of care, when physicians used the test prior to prescribing a medication. The acquisition adds approximately $5.0 billion to the Company’s estimated U.S. TAM. See the Company’s news release from April 26, 2022, for more information.
In May, the Company announced a collaboration with Camille Schrier, Miss America 2020, as part of Mental Health Awareness Month, to promote the potential of genetic testing and the IDgenetix test to help improve treatment for mental health conditions. See the Company’s news release from May 6, 2022, for more information.
Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, May 9, 2022, at 4:30 p.m. Eastern time to discuss its first quarter 2022 results and provide a corporate update.

A live webcast of the conference call can be accessed here: or via the webcast link on the Investor Relations page of the Company’s website, View Source Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until May 30, 2022.

To access the live conference call via phone, please dial 844 200 6205 from the United States, or +1 929 526 1599 internationally, at least 10 minutes prior to the start of the call, using the conference ID 355837.

There will be a brief Question & Answer session following management commentary.

Use of Non-GAAP Financial Measures (UNAUDITED)

In this release, we use the metrics of Adjusted Revenue, Adjusted Gross Margin, Adjusted Operating Cash Flow and Adjusted EBITDA, which are non-GAAP financial measures and are not calculated in accordance with generally accepted accounting principles in the United States (GAAP). Adjusted Revenue and Adjusted Gross Margin reflect adjustments to net revenues to exclude changes in variable consideration related to test reports delivered in previous periods. Adjusted Gross Margin further excludes acquisition-related intangible asset amortization. Adjusted Operating Cash Flow excludes the effects of repayments to Medicare of COVID-19 government relief advancements to healthcare providers. Adjusted EBITDA excludes from net loss interest expense, depreciation and amortization expense, income tax expense, stock compensation expense, and change in fair value of contingent consideration.

We use Adjusted Revenue, Adjusted Gross Margin, Adjusted Operating Cash Flow and Adjusted EBTIDA internally because we believe these metrics provide useful supplemental information in assessing our revenue and cash flow performance reported in accordance with GAAP, respectively. We believe Adjusted Revenue and Adjusted Gross Margin are also useful to investors because they provide additional information on current-period performance by removing the effects of revenue adjustments related to tests delivered in previous periods and acquisition-related intangible asset amortization, which we believe may facilitate revenue and gross margin comparisons to historical periods. We believe Adjusted Operating Cash Flow is also useful to investors as a supplement to GAAP measures in the assessment of our cash flow performance by removing the effects of COVID-19 government relief payments, which we believe are not indicative of our ongoing operations. We believe Adjusted EBITDA may enhance an evaluation of our operating performance based on recent revenue generation and product/overhead cost control because it excludes the impact of prior decisions made about capital investment, financing and other expenses. However, these non-GAAP financial measures may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes.

These non-GAAP financial measures are not meant to be considered in isolation or used as substitutes for net revenues, gross margin, net cash (used in) provided by operating activities or net loss reported in accordance with GAAP; should be considered in conjunction with our financial information presented on GAAP basis; have no standardized meaning prescribed by GAAP; and are not prepared under any comprehensive set of accounting rules or principles. In addition, from time to time in the future, there may be other items that we may exclude for purposes of these non-GAAP financial measures, and we may in the future cease to exclude items that we have historically excluded for purposes of these non-GAAP financial measures. Likewise, we may determine to modify the nature of adjustments to arrive at these non-GAAP financial measures. Because of the non-standardized definitions of non-GAAP financial measures, the non-GAAP financial measure as used by us in this press release and the accompanying reconciliation tables have limits in their usefulness to investors and may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. Accordingly, investors should not place undue reliance on non-GAAP financial measures. Reconciliations of these non-GAAP financial measures to the most directly comparable GAAP financial measures are presented in the tables at the end of this release.

1Centers for Medicare & Medicaid Services: View Source

On May 9, 2022 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing IgM antibodies, reported its financial results for the first quarter ended March 31, 2022 and provided an update on recent developments (Press release, IGM Biosciences, MAY 9, 2022, View Source [SID1234614001]).

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"IGM took a major step in expanding the scope and support of our research pipeline of IgM antibodies with the recent closing of an exclusive worldwide collaboration agreement with Sanofi for the creation, research and development of agonist IgM antibodies against three oncology targets and three autoimmune/inflammation targets," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "We have also made significant progress in the clinical development of our wholly-owned pipeline of IgM antibodies with the start of our two Phase 2 studies for our T cell bispecific antibody, IGM-2323, and the continuation of our Phase 1 trial of IGM-8444. In addition to continued progress in these ongoing clinical trials, we also expect to file INDs for our targeted IL-15 IgM antibody, IGM-7354, and our CD38 x CD3 bispecific IgM antibody, IGM-2644, this year."

Corporate Updates

Closing of collaboration agreement with Sanofi. IGM announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended (the HSR Act), has expired in connection with the Company’s previously announced exclusive worldwide collaboration agreement with Sanofi. In connection with the closing of the collaboration agreement, Sanofi will pay IGM a $150 million upfront payment. The agreement is for the creation, development, manufacture, and commercialization of IgM antibody agonists against three oncology targets and three autoimmune/inflammation targets. In addition to the $150 million upfront payment, IGM is eligible for over $6 billion in aggregate development, regulatory and commercial milestones, a 50:50 profit share in certain major market countries and tiered royalties in the rest of world for oncology targets, and tiered royalties for autoimmune/inflammation targets.

Completed underwritten public offering of common stock. In April 2022, IGM closed a public offering of its common stock with gross proceeds of $230 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by IGM.
Pipeline Updates

IGM-2323 (CD20 x CD3)

Phase 2 studies. IGM previously announced the initiation of two Phase 2 studies to assess the safety and efficacy of two doses of IGM-2323, 100 mg and 300 mg, in patients with diffuse large B cell lymphoma (DLBCL) and follicular lymphoma (FL).
IGM-8444 (DR5)

Clinical development of IGM-8444. IGM continues to advance the clinical development of IGM-8444, the Company’s IgM DR5 agonist, in an open-label, multicenter, Phase I study of IGM-8444 in multiple combinations in subjects with relapsed and/or refractory solid and hematologic cancers.
IGM-7354 (IL-15 x PD-L1)

IND application expected to be filed this year. IGM expects to file an Investigational New Drug Application (IND) for IGM-7354, the Company’s targeted IL-15 IgM antibody, in solid tumors this year.
IGM-2644 (CD38 x CD3)

IND application expected to be filed this year. IGM expects to file an IND for IGM-2644, the Company’s CD38 x CD3 bispecific IgM antibody, in multiple myeloma this year.
First Quarter 2022 Financial Results

Cash and Investments: Cash and investments as of March 31, 2022 were $187.5 million, compared to $229.5 million as of December 31, 2021.
The March 31, 2022 cash and investments balance does not include the $230.0 million in gross proceeds, before deducting the underwriting discounts and commissions and other offering expenses payable by IGM, received in connection with IGM’s 2022 public offering, which closed in April.
The March 31, 2022 cash and investments balance also does not include the $150.0 million upfront payment that IGM is expected to receive from Sanofi during the second quarter of 2022 under the terms of the collaboration agreement announced in March 2022.
Research and Development (R&D) Expenses: For the first quarter of 2022, R&D expenses were $38.9 million, compared to $23.6 million for the same period in 2021.
General and Administrative (G&A) Expenses: For the first quarter of 2022, G&A expenses were $13.1 million, compared to $8.1 million for the same period in 2021.
Net Loss: For the first quarter of 2022, net loss was $51.9 million, or a loss of $1.53 per share, compared to a net loss of $31.6 million, or a loss of $0.95 per share, for the same period in 2021. The net loss included non-cash stock-based compensation expense of $11.5 million and $5.5 million for the first quarter of 2022 and 2021, respectively.
2022 Financial Guidance

IGM reiterates its previously issued financial guidance expecting full year GAAP operating expenses of $250 million to $260 million including estimated non-cash stock-based compensation expense of approximately $50 million. IGM expects to end 2022 with a balance of over $390 million in cash and investments.

On May 9, 2022 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a developer of next-generation biopharmaceuticals and pioneer of the sustainable FastPharming Manufacturing System, reported a poster presentation on IBIO–101, the Company’s monoclonal antibody candidate for the treatment of solid tumors (Press release, iBioPharma, MAY 9, 2022, View Source [SID1234614063]). The presentation will take place at Frontiers in Cancer Immunotherapy 2022 by the New York Academy of Sciences from May 9-11.

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Preclinical evaluation of IBIO-101, produced with iBio’s FastPharming and GlycaneeringSM Systems, showed equivalent efficacy and potency compared with this IL-2 sparing anti-CD25 antibody made using traditional mammalian cell culture methods (RTX-003 from RubrYc Therapeutics, Inc.). Additionally, iBio’s proprietary afucosylation technology enabled the engineering of a more potent version without incremental intellectual property access costs. The Company now plans to advance its Glycaneered anti-CD25 monoclonal antibody for the depletion of regulatory T cells ("Tregs") to the clinic next year.

Dillon Phan, PhD, iBio’s Vice President and Head of Early Research and Development, will present the poster, titled "Plant-Based Expression and Glyco-Engineering of Novel IL-2 Signaling Permissive Anti-CD25 Antibodies for Effective Treg Depletion in Cancer", which highlights:

A CD25 epitope-survey using a novel artificial intelligence/machine learning platform to identify one epitope and corresponding antibodies with particularly high antibody-dependent cellular cytotoxicity ("ADCC") activity.
The production of a Glycaneered version of IBIO-101, which significantly increased effector function resulting from afucosylation using a deltaXT/FT N. benthamiana host compared with a fucoslyated form of the molecule.
Binding of IBIO-101 specifically to CD25+ cancerous cells with high affinity.
Preserved IL-2 signaling to effector T cells via pSTAT5.
Potent ADCC activity in killing cancer cells.

On May 9, 2022 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported that it has entered into a definitive agreement with a single leading healthcare investor for the purchase and sale of 10,563,380 of the Company’s American Depositary Shares ("ADSs") (or ADS equivalents), each ADS representing ten (10) ordinary shares, at a purchase price of $1.42 per ADS (or ADS equivalent), in a registered direct offering (Press release, RedHill Biopharma, MAY 9, 2022, View Source [SID1234613885]). RedHill has also agreed to issue to the investor unregistered private warrants to purchase up to an aggregate of 13,204,225 ADSs in a concurrent private placement. The warrants have an exercise price of $1.48 per ADS, are exercisable six months after the issuance date and have a term of five and one-half years. The closing of the offering is expected to occur on or about May 11, 2022, subject to the satisfaction of customary closing conditions.

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Cantor Fitzgerald & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds to the Company from this offering are expected to be approximately $15 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. The Company intends to use the net proceeds from this offering for working capital, acquisitions, and general corporate purposes.

The securities described above (but not the warrants or the ADSs underlying the warrants) are being offered by the Company pursuant to a "shelf" registration statement on Form F-3 (File No 333-258259) previously filed with the Securities and Exchange Commission (the "SEC") on July 29, 2021, and declared effective by the SEC on August 9, 2021. The offering of the securities is made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the securities being offered will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting Cantor Fitzgerald & Co., 499 Park Avenue, 4th Floor, New York, New York 10022, Attn: Capital Markets Department, or by email at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the ADSs underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying ADSs may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On May 9, 2022 bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") reported financial results and business highlights for the first quarter ended March 31, 2022, and shared recent operational progress (Press release, bluebird bio, MAY 9, 2022, View Source [SID1234613918]).

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"For more than a decade, bluebird bio has helped chart the path for the field of gene therapy and today, we are closer than ever before to realizing our mission of bringing potentially curative gene therapies to patients and their families," said Andrew Obenshain, chief executive officer, bluebird bio. "We have taken action to sharpen our focus and our finances, as the FDA completes its review of the beti-cel and eli-cel BLAs, and we prepare for the launch of two gene therapies in the second half of the year. In parallel, we are actively collecting the manufacturing comparability data required for lovo-cel and remain focused on submitting our BLA for sickle cell disease to the FDA in Q1 2023."

RECENT HIGHLIGHTS

BETI-CEL AND ELI-CEL

ADVISORY COMMITTEE MEETING ON JUNE 9-10, 2022 – On April 13, the U.S Food and Drug Administration (FDA) confirmed its plans to review betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel) in an FDA Cellular, Tissue and Gene Therapies Advisory Committee Meeting that will take place over two days on June 9-10, 2022. The advisory committee will discuss the efficacy and safety data supporting the benefit/risk of beti-cel and eli-cel respectively, as well as safety information relevant to both therapies. If approved, beti-cel and eli-cel would be the Company’s first FDA approved therapies as an independent severe genetic disease company. An overview of the agenda was included in the Federal Register announcement posted on April 13. The full agenda will be determined by the FDA and included in briefing materials that will be posted by the FDA closer to the date of the advisory committee meeting.
LOVO-CEL

CONTINUED PROGRESS TOWARD BLA SUBMISSION – bluebird bio has enrolled more than half of the patients in the HGB-210 study that will be needed to support manufacturing data requirements for the lovotibeglogene autotemcel (lovo-cel) biologics licensing application (BLA) and the Company is on track to submit a BLA to the FDA for sickle cell disease (SCD) in the first quarter of 2023. As previously communicated, the Company has treated all patients in HGB-206 Group C who will form the primary basis of efficacy for BLA submission, with the demonstration of analytical comparability and validation of the commercial manufacturing process as the key remaining actions prior to submission of the planned BLA. Enrollment and dosing for patients 18 and older are continuing in the HGB-210 study and the Company remains in active dialogue with the FDA about the resolution of the partial clinical hold for patients under 18.
COMPANY

RESTRUCTURING UPDATE – On April 5, 2022, bluebird bio announced a comprehensive restructuring intended to deliver up to $160 million in cost savings over the next two years, reduce its workforce by approximately 30% and extend its cash runway into the first half of 2023. Near-term cost savings were realized in April as bluebird began implementing the restructuring. The Company continues to evaluate additional financing options, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
NEW HEADQUARTERS MOVE –bluebird bio has begun transitioning to its new headquarters in Assembly Row. The Company’s new HQ is designed to reflect modern ways of working and estimated to result in more than $120 million in cost savings over the next six years. bluebird will maintain laboratory space and operations at 60 Binney St. in Cambridge through 2023.
UPCOMING INVESTOR EVENTS

Members of the management team will participate in the following upcoming investor conferences:

BofA Securities 2022 Health Care Conference, Wednesday, May 11, at 3:20 p.m. PT at the Encore Hotel, Las Vegas, NV
2022 RBC Capital Markets Global Healthcare Conference, Tuesday, May 17, at 8:00 a.m. ET at the Intercontinental NY Barclay, New York, NY
Goldman Sachs 43rd Annual Global Healthcare Conference, Wednesday, June 15, at 4:00 p.m. PT at the Terranea Resort, Rancho Palos Verdes, CA
To access the live webcast of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcasts will be available on the bluebird bio website for 90 days following the event.

UPCOMING ANTICIPATED MILESTONES

LOVO-CEL

The Company is in active communication with the FDA to resolve the partial clinical hold and resume enrollment and treatment of patients under the age of 18.
The Company plans to complete manufacturing of commercial drug product validation lots by mid-2022.
The Company expects to confirm vector and drug product analytical comparability by Q4 2022.
The Company plans to submit its BLA for lovo-cel in Q1 2023.
BETI-CEL

The FDA has set a PDUFA goal date of August 19, 2022, for a decision on the approval of beti-cel in patients with β-thalassemia with commercial launch expected to follow in the beginning of Q4 2022 if approved.
An FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
ELI-CEL

The FDA has set a PDUFA goal date of September 16, 2022, for a decision on the approval of eli-cel in patients with cerebral adrenoleukodystrophy with therapy availability expected in Q4 2022 if approved.
An FDA advisory committee meeting for beti-cel and eli-cel will be held over the course of two days on June 9-10, 2022.
bluebird bio is in active communication with the FDA to resolve the clinical hold and anticipates the FDA’s questions may be resolved concurrent with the agency’s ongoing review of the Company’s BLA submission.
FIRST QUARTER 2022 FINANCIAL RESULTS

Cash Position: The Company’s restricted cash, cash and cash equivalents and marketable securities balance was approximately $312 million, including restricted cash of approximately $45 million, as of March 31, 2022. The full-year 2022 cash burn is expected to be less than $340 million with a 35 to 40 percent reduction in operating costs anticipated by year-end 2022.

The Company is exploring multiple financing opportunities, including public or private equity financings and monetizing any priority review vouchers that may be issued upon approval of beti-cel or eli-cel.
Revenues: Total revenue was $1.9 million for the three months ended March 31, 2022, compared to $0.9 million for the three months ended March 31, 2021.
R&D Expenses: Research and development expenses from continuing operations were $77.9 million for the three months ended March 31, 2022, compared to $82.8 million for the three months ended March 31, 2021. The decrease of $4.9 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses, offset by increased manufacturing costs.
SG&A Expenses: Selling, general and administrative expenses from continuing operations were $36.1 million for the three months ended March 31, 2022, compared to $63.6 million for the three months ended March 31, 2021. The decrease of $27.5 million was primarily due to decreased employee compensation, benefit, and other head-count related expenses and decreased commercial readiness activities due to the Company’s decision to focus its efforts on the U.S. market for beti-cel, eli-cel, and lovo-cel.
Net Loss: Net loss from continuing operations was $122.2 million for the three months ended March 31, 2022, compared to $121.5 million for the three months ended March 31, 2021.