On August 9, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2021 and provided operational updates, including the announcement that the U.S. Food and Drug Administration (FDA) placed a clinical hold on clinical studies of elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy (licensed as SKYSONA in Europe) for cerebral adrenoleukodystrophy (CALD) (Press release, bluebird bio, AUG 9, 2021, View Source [SID1234586078]).

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"I’m tremendously proud of what bluebird has accomplished this quarter both operationally and strategically to ready ourselves to launch both bluebird bio and 2seventy bio," said Nick Leschly, chief bluebird. "Seven months after announcing our intent to split, and thanks to the incredibly hard work by our teams, we have created a solid foundation for both organizations. The ABECMA launch is exceeding expectations, the oncology INDs and severe genetic disease (SGD) biologics license application (BLA) filings are tracking for later this year, and we have established clear visions and leadership teams for each business. Importantly, we have made tough strategic decisions to reshape the overall cost structure to allow both companies to launch in a strong position to execute through important value-creating milestones."

BUSINESS SEPARATION

In January 2021, bluebird announced its intent to separate into two independent, publicly traded companies (bluebird bio and 2seventy bio). The company expects the separation to be completed by the end of 2021 and to be tax-free to bluebird shareholders.

Key members of the executive teams of both companies have been announced, effective upon completion of the planned separation. This includes Andrew Obenshain as CEO of bluebird and Nick Leschly as CEO of 2seventy.
The full board of directors for both companies will be announced closer to the separation date.
2seventy has confidentially filed its Form 10 Registration Statement with the U.S. Securities and Exchange Commission (SEC), in which it describes the planned tax-free spin-off of 2seventy as a publicly traded company.
bluebird plans to distribute 100% of the outstanding shares of 2seventy common stock to bluebird’s stockholders on a pro-rata basis.
Based on current cash position and the expected $110M upfront payment upon closing of the National Resilience, Inc. strategic collaboration, the Company anticipates having a cash balance of approximately $900M at the time of separation. Together with existing and emerging sources of revenue, we expect our cash balance will be sufficient to fund approximately 24 months of operations for bluebird and 2seventy under current business plans.
ELI-CEL SAFETY UPDATE

The company received a reported Suspected Unexpected Serious Adverse Reaction (SUSAR) of myelodysplastic syndrome (MDS), that is likely mediated by Lenti-D lentiviral vector (LVV) insertion, in a patient who was treated with eli-cel, or Lenti-D drug product for CALD over one year ago in the Phase 3 ALD-104 study. Evidence currently available suggests that specific design features of Lenti-D LVV likely contributed to this event. The company has shared this information with the independent data monitoring committee of the study and the FDA has placed the eli-cel program on a clinical hold. The company does not anticipate the clinical hold to impact its programs in sickle cell disease (SCD), β-thalassemia or oncology. Subject to resolution of the clinical hold, the company anticipates completing the submission of the rolling BLA for eli-cel in 2021.

"Our hearts go out to this patient and his family, who are dealing with a challenging diagnosis," said Nick Leschly, chief bluebird. "Given what we know, we remain confident that eli-cel can offer hope for patients and families impacted by this devastating disease who have very few treatment options. We are committed to working with regulators and physicians in order to resolve this hold as soon as possible and bring this important therapy to patients in need."

BLUEBIRD BIO BUSINESS UPDATE

Today, bluebird bio is announcing that the company intends to focus its SGD business on the U.S. market and on further investments in research and development to optimize its core three programs in SCD, β-thalassemia and CALD, as well as on the development of a pipeline exploring new disease indications using in vivo LVV technology. The company remains focused and is on track to complete the rolling submissions of the U.S. BLAs in β-thalassemia in 3Q 2021 and CALD in 2021, pending resolution of the eli-cel clinical hold.

In connection with the planned completion of the business separation in the fourth quarter of 2021 and pivot to U.S.-centric efforts for SGD, bluebird plans an orderly wind down of its operations in Europe and to explore how to give patients in Europe access to its gene therapies, including potentially out-licensing the ex-U.S. rights to its three lead products to a company with European experience and capabilities.

"bluebird’s decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for ZYNTEGLO in Europe, which makes bringing its transformative gene therapies like ZYNTEGLO and SKYSONA to patients and physicians in Europe untenable for a small innovative company at this time," said Andrew Obenshain, president, severe genetic diseases, bluebird bio. "While European regulators have been innovative partners in supporting accelerated regulatory paths for these therapies, European payers have not yet evolved their approach to gene therapy in a way that can recognize the innovation and the expected life-long benefit of these products. We are committed to and hope to find a potential partner who can help us carry forward our therapies in Europe."

BLUEBIRD BIO RECENT HIGHLIGHTS

BB1111 AND ZYNTEGLO CLINICAL HOLD LIFT – On June 7, 2021, bluebird announced that the FDA has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for SCD gene therapy (bb1111) for adult and pediatric patients with SCD, and the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies of betibeglogene autotemcel gene therapy (beti-cel; licensed as ZYNTEGLO in the EU and the UK) for adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT). The company is working closely with study investigators and clinical trial sites to resume all study activities as soon as possible.
β-THALASSEMIA

EHA DATA – On June 11, 2021, bluebird presented data from several studies of beti-cel in adult, adolescent and pediatric patients with TDT. These data were presented during EHA (Free EHA Whitepaper)2021 Virtual, the 26th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). With 51 patients enrolled, data from the long-term follow-up study (LTF-303) show that all patients treated with beti-cel who achieve transfusion independence (TI) remain free from transfusions, with the longest follow-up of seven years. Across Phase 3 studies, 89% (32/36) of evaluable patients across ages and genotypes achieved TI and remain transfusion free, including 91% (20/22) of evaluable pediatric patients under the age of 18. Data from bluebird bio’s Phase 1/2 and Phase 3 clinical studies represent more than 220 patient-years of experience with beti-cel.
EU MARKETING AUTHORIZATION – On July 9, 2021, bluebird announced that the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has concluded based on the review of all available data that the benefit-risk balance of ZYNTEGLO (beti-cel) remains favorable. bluebird bio informed the EMA that the company has lifted the voluntary marketing suspension. Today, bluebird is announcing that on the basis of the PRAC decision, the EMA CHMP (Committee for Medicinal Products for Human Use) has endorsed the positive recommendation for ZYNTEGLO by the PRAC. A confirmatory decision by the European Commission (EC) is expected in 3Q 2021.
CEREBRAL ADRENOLEUKODYSTROPHY

SKYSONA EC DECISION – On July 21, 2021, bluebird announced that the EC granted marketing authorization of SKYSONA, a one-time gene therapy for the treatment of early CALD in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available.
2SEVENTY BIO RECENT HIGHLIGHTS

ABECMA LAUNCH – This quarter, bluebird and Bristol-Myers Squibb (BMS) launched ABECMA (idecabtagene vicleucel; ide-cel) in the U.S. ABECMA generated total U.S. revenues of $24 million in 2Q 2021 which bluebird shares equally with BMS. As of June 30, 2021, over 65 sites in the U.S. had been qualified to treat patients. bluebird and BMS have reported robust demand for ABECMA and are working to continue to increase manufacturing capacity over time.
STRATEGIC MANUFACTURING ALLIANCE – On July 28, 2021, bluebird and National Resilience, Inc. (Resilience) announced a strategic alliance aimed to accelerate the early research, development and delivery of cell therapies. As part of the agreement, Resilience will acquire bluebird’s Research Triangle (bRT) manufacturing facility located in North Carolina for $110 million and will retain all of the more than 100 highly skilled technical staff and administrators currently employed at the site. Resilience will continue to support vector supply for both bluebird and 2seventy. The two companies are also finalizing a definitive agreement to establish partner programs that will share expense and revenue for successful commercialized oncology products and in parallel establish a next-generation manufacturing R&D collaboration. Additionally, 2seventy plans to invest in internal drug product manufacturing capability and capacity to support its future clinical studies.
KARMMA ASCO (Free ASCO Whitepaper) DATA – On May 19, 2021, bluebird and BMS announced new data and analyses from the pivotal KarMMa study evaluating ABECMA. These data showed a 24.8-month median overall survival in triple-class exposed relapsed/refractory multiple myeloma. With more than 24-month median follow-up, results represent longest follow-up to date from a global clinical trial of a CAR T cell therapy in multiple myeloma with 73% overall response rate and responses ongoing with a median duration of response in patients achieving a ≥CR of 21.5 months. Analysis of characteristics of neurotoxicity (NT) observed in KarMMa study reinforce well-understood safety profile of ABECMA with mostly Grade 1/2 occurrences of NT having early onset and resolution.
UPCOMING ANTICIPATED MILESTONES

BLUEBIRD BIO

bluebird anticipates the separation of its SGD and oncology businesses into two independent, publicly traded companies (bluebird bio and 2seventy bio) to be completed by the end of 2021.
TDT: The company is on track to complete its rolling BLA submission to the FDA for beti-cel in 3Q 2021. This submission is anticipated to include adult, adolescent and pediatric patients with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes).
CALD: Subject to resolution of the clinical hold, the company plans to complete its rolling BLA submission to the FDA for eli-cel in 2021.
SCD: The company is continuing to evaluate the impact of the recently-lifted clinical hold on bb1111 and plans to continue to work closely with the FDA in their review of these events to provide an update on the company’s development plan and timeline for submission for regulatory approval of bb1111 by year end.
SCD: The company plans to present clinical data from its ongoing HGB-206 clinical study of bb1111 by the end of 2021.
2SEVENTY BIO

Continued commercial launch of ABECMA in the U.S.
Submission of 1-2 investigational new drug (IND) applications by the end of 2021.
Presentation of clinical data from the ongoing CRB-402 study of bb21217 by the end of 2021.
SECOND QUARTER 2021 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2021 and December 31, 2020 were $941.6 million and $1.27 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating activities.
Revenues: Total revenues were $7.5 million for the three months ended June 30, 2021 compared to $198.9 million for the three months ended June 30, 2020. Total revenues were $20.3 million for the six months ended June 30, 2021 compared to $220.8 million for the six months ended June 30, 2020. The decrease for both periods was primarily driven by a cumulative catch-up adjustment to revenue recorded in connection with the May 2020 BMS contract modification in the second quarter of 2020.
R&D Expenses: Research and development expenses were $144.3 million for the three months ended June 30, 2021 compared to $156.3 million for the three months ended June 30, 2020. Research and development expenses were $298.8 million for the six months ended June 30, 2021 compared to $310.4 million for the six months ended June 30, 2020. The decrease for both periods was primarily driven by decreased manufacturing expenses, a decrease in license and milestone fees, and a decrease in clinical costs in light of safety events in the HGB-206 study of LentiGlobin for SCD gene therapy.
SG&A Expenses: Selling, general and administrative expenses were $78.6 million for the three months ended June 30, 2021 compared to $68.6 million for the three months ended June 30, 2020. Selling, general and administrative expenses were $165.5 million for the six months ended June 30, 2021 compared to $141.9 million for the six months ended June 30, 2020. The increase for both periods was primarily driven by an increase in consulting fees associated with the ongoing project to separate the company’s severe genetic disease and oncology businesses into two independently traded companies as well as an increased employee compensation, benefit, and other headcount related expenses.
Net Loss: Net loss was $241.7 million for the three months ended June 30, 2021 compared to $21.5 million for the three months ended June 30, 2020. Net loss was $447.5 million for the six months ended June 30, 2021 compared to $224.1 million for the six months ended June 30, 2020.
Investor Conference Call Information

bluebird will hold a conference call to discuss this update on Monday, August 9 at 8:00 a.m. ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 9698691.

To access the live webcast of bluebird’s presentation, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird website at View Source A replay of the webcast will be available on the bluebird website for 90 days following the event.

On August 9, 2021 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, immunologic diseases (e.g., complement-mediated diseases and cancers) and central nervous system disorders, reported recent highlights and developments as well as financial results for the second quarter ended June 30, 2021, which include (Press release, Omeros, AUG 9, 2021, View Source [SID1234586114]):

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OMIDRIA revenues for the second quarter of 2021 were $28.8 million compared to $21.1 million in the first quarter. The 37 percent increase over the prior quarter primarily reflects growth in sales of OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3% in ambulatory surgery centers (ASCs).
Net loss in the second quarter of 2021 was $28.6 million, or $0.46 per share, including non-cash expenses of $3.9 million, or $0.06 per share. This compares to a net loss of $35.1 million, or $0.57 per share, which included non-cash expenses of $4.1 million, or $0.07 per share, for the previous quarter.
At June 30, 2021, Omeros had cash, cash equivalents and short-term investments available for operations of $73.7 million.
Omeros’ Biologics License Application (BLA) for narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangipathy (HSCT-TMA or TA-TMA) is under priority review by the U.S. FDA with an action date of October 17, 2021 under the Prescription Drug User Fee Act (PDUFA).
Omeros announced preliminary results from the Phase 1 clinical trial of OMS906, the company’s MASP-3 inhibitor, which showed that (i) OMS906 was well tolerated at all doses tested and (ii) human pharmacokinetic and pharmacodynamic data are consistent with once-monthly or less frequent subcutaneous dosing.
"In the second quarter of 2021, Omeros achieved a number of important milestones," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "As OMIDRIA sales continue to grow, CMS in its recent OPPS Proposed Rule reaffirmed its determination that OMIDRIA receive separate payment when used in ASCs, and our MASP-3 inhibitor OMS906 is successfully advancing through its Phase 1 clinical trial. Several other important milestones should reach resolution in the near term – FDA’s decision on our pending BLA for narsoplimab in the treatment of patients with TA-TMA, results from narsoplimab in the COVID-19 I-SPY platform clinical trial, and the outcome of the NOPAIN Act, which, if enacted by Congress, would mandate Medicare separate payment for non-opioid surgical pain management drugs like OMDRIA not only in ASCs but also in hospital outpatient departments. The remainder of our pipeline also pressed ahead, including the GPR174 program, the core of our immuno-oncology platform – a platform that has expanded beyond GPR174 with new CAR-T and adoptive cellular therapy programs. Omeros’ momentum is building, and we look forward to seeing what the rest of the year brings."

Second Quarter and Recent Developments

Recent developments regarding OMIDRIA include the following:
In July 2021, the Centers for Medicare and Medicaid Services (CMS) released its Outpatient Prospective Payment System (OPPS) and ASC Payment System proposed rule for calendar year 2022, which reaffirmed its earlier decision that OMIDRIA, when used in the ASC setting, qualifies for separate payment under CMS’ policy regarding non-opioid pain management surgical drugs. This policy has been in effect since 2019.
Recent developments regarding narsoplimab, Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) in advanced clinical programs for the treatment of TA-TMA, immunoglobulin A (IgA) nephropathy, atypical hemolytic uremic syndrome (aHUS) and severely ill COVID-19 patients, include the following:
Omeros announced data from the second cohort of 10 critically ill COVID-19 patients treated with narsoplimab under compassionate use in Bergamo, Italy.
All of the patients had comorbidities and/or risk factors for poor outcomes, were mechanically ventilated, and had failed other therapies, including steroids.
90 percent of the patients were intubated prior to initiation of narsoplimab treatment.
80 percent of the patients recovered, survived and were discharged. The two deaths involved a 76-year-old who died of complications related to his pre-existing cardiomyopathy and a 68-year-old who began narsoplimab treatment after 13 days of intubation.
A manuscript from the Omeros-Cambridge Center for Complement and Inflammation Research (OC3IR) on the inhibition of the lectin pathway and MASP-2 as a potential treatment for severe COVID-19 was published in the peer-reviewed journal Frontiers in Immunology.
An abstract on the pharmacokinetic and pharmacodynamic modeling of lectin pathway inhibition by narsoplimab was accepted for presentation at the 16th International Symposium on IgA nephropathy.
Updates regarding Omeros’ other development programs and platforms include the following:
Omeros announced preliminary results from the Phase 1 clinical trial evaluating the pharmacokinetics/pharmacodynamics (PK/PD) and safety of OMS906 in healthy subjects. OMS906 inhibits MASP-3, the key activator of the alternative pathway of complement. MASP-3 is responsible for the conversion of pro-factor D to mature factor D. Data from the first five cohorts of the trial’s single-ascending dose stage show that (i) OMS906 was well tolerated at all doses tested (up to 5 mg/kg) and (ii) a single 3 mg/kg intravenous dose of OMS906 and a single dose of the lowest subcutaneous concentration tested each suppressed mature complement factor D below minimum detectable levels. The human PK/PD data were consistent with once-monthly or less frequent subcutaneous dosing. Following completion of the single- and multiple-ascending dose stages of the Phase 1 trial, Omeros plans to initiate a Phase 2 clinical trial.
A paper detailing the mechanism of action of PDE7 inhibition in nicotine addiction was published in the peer-reviewed Journal of Neuroscience and was selected for inclusion in the journal’s Featured Research page. Omeros has completed a successful Phase 1 trial with the lead compound in its PDE7 inhibitor program.
Financial Results

For the second quarter of 2021, OMIDRIA revenues were $28.8 million compared to $21.1 million for the first quarter.

Total costs and expenses for the second quarter of 2021 were $52.8 million compared to $51.7 million for the first quarter. The increase was primarily due to increased selling, general and administrative expenses in preparation for the anticipated U.S. launch of narsoplimab and additional employee-related costs. Research and development costs decreased quarter over quarter due to the timing of narsoplimab manufacturing-related costs, which are expensed rather than included as inventory until the initial marketing approval for narsoplimab is certain.

For the three months ended June 30, 2021, Omeros reported a net loss of $28.6 million, or $0.46 per share, which included non-cash expenses of $3.9 million, or $0.06 per share. This compares to a net loss in the previous quarter of $35.1 million, or $0.57 per share, which included non-cash expenses of $4.1 million, or $0.07 per share.

As of June 30, 2021, the company had $73.7 million of cash, cash equivalents and short-term investments. The company also has a line of credit, which permits borrowing up to the lesser of $50.0 million and 85 percent of eligible accounts receivable less certain reserves. Omeros also has an "at the market" program in place that allows the company to sell, from time to time, up to $150.0 million of its common stock.

Conference Call Details

Omeros’ management will host a conference call to discuss the financial results and to provide an update on business activities. The call will be held today at 1:30 p.m. Pacific Time; 4:30 p.m. Eastern Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 4195376. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 4195376.

To access the live or subsequently archived webcast of the conference call on the internet, go to the company’s website at View Source

On August 9, 2021 Celcuity Inc. (NASDAQ:CELC), a clinical-stage biotechnology company pursuing an integrated companion diagnostic and therapeutic strategy for treating patients with cancer, reported financial results for the second quarter ended June 30, 2021 and summarized recent business progress (Press release, Celcuity, AUG 9, 2021, View Source [SID1234586130]).

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"Celcuity’s recent follow-on equity offering strengthens our balance sheet and provides additional funding to support clinical development activities for the advancement of gedatolisib, a potential first-in-class PI3K/mTOR inhibitor," said Brian Sullivan, CEO and co-founder of Celcuity. "We are also very excited about the progress we made expanding our clinical development and clinical operations teams. This positions us well to initiate, subject to feedback from the FDA, a Phase 2/3 clinical trial evaluating gedatolisib in combination with palbociclib and an endocrine therapy in patients with ER+/HER2- advanced or metastatic breast cancer in the first half of 2022."

Second Quarter 2021 Business Highlights and Other Recent Developments

In early April, Celcuity entered into a worldwide licensing agreement with Pfizer for the exclusive right to develop and commercialize gedatolisib.
Celcuity closed two significant financings to support development of gedatolisib. In early April, Celcuity entered into a debt financing agreement with Innovatus Life Sciences Lending Fund I, LP to provide up to $25.0 million in term loans with the first tranche of $15.0 million funded at closing. In early July, Celcuity completed a follow-on public offering that raised gross proceeds of approximately $56.3 million.
In April, Celcuity presented results of studies evaluating gedatolisib, inavolisib (a PI3K-α inhibitor), and navitoclax (a BCL inhibitor) in breast and ovarian patient tumors in two posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The results showed that gedatolisib inhibited nine times more signaling test activity in tumors with hyperactive RAS network signaling, on average, than inavolisib, when evaluated at equal concentrations with the CELsignia test.
In June, Celcuity announced the addition of Igor Gorbatchevsky, MD, as VP of Clinical Development and Jill Krause as VP of Clinical Operations to Celcuity’s leadership team. Dr. Gorbatchevsky has over two decades of hands-on oncology drug development experience, including successful regulatory IND and NDA/BLA filings across several drug classes. Ms. Krause has deep clinical operations expertise, including over nine years of experience managing and executing trials in breast cancer. Since joining Celcuity, Dr. Gorbatchevsky and Ms. Krause have each added key members to their respective teams.
In July, results from a 17-patient Phase 1 study that evaluated the safety and preliminary activity of gedatolisib combined with carboplatin and paclitaxel were published in the journal, Clinical Cancer Research1 . The study was designed to explore the hypothesis that inhibition of the PI3K/mTOR pathway can promote sensitivity to platinum-based treatment. The objective response rate (ORR) was 65% (11/17) patients: eight partial responses (PR) and three complete responses (CR) were reported. The stable disease rate was 17% (3/17). Promising results were observed in patients with advanced clear cell ovarian carcinoma (CCOC), a tumor type with poor prognosis generally considered to be chemo resistant. The ORR in patients with CCOC was 80% (8/10), of which 3/10 (30%) reported a CR. Among nine previously platinum-treated patients, 45% (4/9) had a PR (two patients with CCOC, one low grade serous ovarian cancer and one NSCLC). The drug combination was found to be tolerable with a manageable safety profile.
Second Quarter 2021 Financial Results

Unless otherwise stated, all comparisons are for the second quarter ended June 30, 2021, compared to the second quarter ended June 30, 2020.

The following discussion should be read in conjunction with our unaudited condensed consolidated financial statements and related notes on Form 10-Q for the second quarter ended June 30, 2021.

Total operating expenses were $13.6 million for the second quarter of 2021, compared to $2.2 million for the second quarter of 2020.

Research and development (R&D) expenses were $13.1 million for the second quarter of 2021, compared to $1.8 million for the second quarter of 2020. The increase during the second quarter of 2021 compared to the prior year, primarily resulted from a $10.0 million upfront license fee related to the execution of the Pfizer license agreement, which included $5.0 million of non-cash expense for the issuance of common stock. The remaining increase in expenses related to compensation, clinical validation and laboratory studies and legal expenses.

General and administrative (G&A) expenses were $0.6 million for the second quarter of 2021, compared to $0.4 million for the second quarter of 2020. The increase in the second quarter of 2021 arose primarily from higher professional fees associated with being a public company, director and officer insurance and non-cash stock-based compensation.

Net loss for the second quarter of 2021 was $14.0 million, or $1.11 loss per share, compared to a net loss of $2.2 million, or $0.21 loss per share, for the second quarter of 2020. Non-GAAP adjusted net loss for the second quarter of 2021 was $8.3 million, or $0.66 loss per share, compared to non-GAAP adjusted net loss of $1.8 million, or $0.17 loss per share, for the second quarter of 2020. Non-GAAP adjusted net loss excludes stock-based compensation expense, issuance of common stock and non-cash interest. Because these items have no impact on Celcuity’s cash position, management believes non-GAAP adjusted net loss better enables Celcuity to focus on cash used in operations. For a reconciliation of financial measures calculated in accordance with generally accepted accounting principles in the United States (GAAP) to non-GAAP financial measures, please see the financial tables at the end of this press release.

Net cash used in operating activities for the second quarter of 2021 was $7.6 million, compared to $1.6 million for the second quarter of 2020.

At June 30, 2021, Celcuity had cash and cash equivalents of $41.6 million, compared to cash and cash equivalents of $11.6 million at December 31, 2020. On April 8, 2021, Celcuity paid an upfront license fee of $5.0 million in conjunction with the Pfizer gedatolisib license agreement and received $14.4 million of net proceeds from a debt financing agreement. On July 1, 2021, Celcuity completed a follow-on equity offering of common stock that resulted in gross proceeds of approximately $56.3 million. After the follow-on offering, Celcuity had approximately $94.4 million of cash on hand.

Anticipated Milestones

Celcuity expects to achieve the following potential milestones over the next twelve months:

Announce an additional clinical trial collaboration utilizing the CELsignia platform in the second half of 2021.
Initiate a Phase 2/3 clinical trial for gedatolisib in patients with ER+/HER2- metastatic breast cancer in the first half of 2022, subject to feedback from the FDA.
Provide an update on the lifecycle development plan for gedatolisib in the first half of 2022.
Provide interim results from the FACT-1 and FACT-2 trials in late 2021 or early 2022.
Webcast and Conference Call Information

The Celcuity management team will host a webcast/conference call at 4:30 p.m. ET today to discuss the second quarter financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-844-369-8770 and international callers should dial 862-298-0840. A live webcast presentation can also be accessed using this weblink: View Source . A replay of the webcast will be available on the Celcuity website following the live event.

On August 9, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Cellectar Biosciences, AUG 9, 2021, View Source [SID1234586147]).

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Second Quarter and Recent Corporate Highlights

Announced the expansion of the ongoing collaboration with biotechnology company IntoCell Inc., combining their novel linker chemistry with Cellectar’s validated targeting platform to create novel next generation phospholipid drug conjugate (PDC) therapeutics.

Announced a co-development and commercialization collaboration with LegoChemBio, a clinical stage biotechnology company to utilize their proprietary drug conjugate linker-toxin platform to further enhance the company’s portfolio of next generation PDC therapeutics.

Presented a poster entitled "Treatment Free Remission (TFR) and Overall Response Rate (ORR) Results in Patients with Relapsed/Refractory Waldenstrom’s Macroglobulinemia (WM) Treated with CLR 131" at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual meeting.

The poster provided an update of six patients from the company’s Phase 2a study of CLR 131 in Waldenstrom’s macroglobulinemia demonstrating encouraging data.
A 100% (6/6) overall response rate, 83.3% (5/6) major response rate and a 16.7% (1/6) complete response rate.
A median time to initial response of 22 days after first infusion with a median time to major response, as defined as at least a 50% reduction in IgM, of 44 days after first infusion.
A mean treatment free remission, as defined as the time from the last CLR 131 infusion to progression of disease, of 1.1 years and ongoing.
Median duration of response had not been reached, with 100% of the MYD88 wild type and high-risk patients exceeding 8.5 months.
Progression free survival (PFS) for both MYD88 wild type patients as well as the high-risk subgroup had not been reached after 18 months; PFS for the multi-drug refractory patients subgroup was 11 months.

Hosted Key Thought Leader event with Dr. Sikander Ailawadhi, M.D., of the Mayo Clinic, the lead investigator for the company’s pivotal study of CLR 131 in patients with Waldenstrom’s macroglobulinemia.

Added extensive hematology and oncology expertise to the company’s board of directors with the addition of Dr. Asher Alban Chanan-Khan as an independent director.
"During the second quarter, we remained focused on advancing both our preclinical and clinical objectives. The high level of interest and participation in our WM pivotal study by international thought leadership and academic centers from around the globe is extremely exciting," said James Caruso, president and CEO of Cellectar. "We presented compelling CLR 131 data at ASCO (Free ASCO Whitepaper) and announced two new collaborations with biotechnology companies specializing in proprietary drug conjugate linker chemistry to diversify our PDC pipeline. With over $46 million in cash and cash equivalents as of June 30, 2021, we are well capitalized with the cash runway to execute on our anticipated value enhancing milestones into 2023."

Second Quarter Financial Highlights

Cash and Cash Equivalents: As of June 30, 2021, the company had cash and cash equivalents of $46.8 million compared to $57.2 million at December 31, 2020. Cash used in operating activities was approximately $11.6 million during the six months ended June 30, 2021 as compared to $6.6 million during the six months ended June 30, 2020.

Research and Development Expense: R&D expense for the three months ended June 30, 2021 was $4.6 million, compared to $2.5 million for the three months ended June 30, 2020. The cumulative R&D spending for the first six months of 2021 was $9.3 million as compared to $5.1 million for the first six months of 2020. The increase in R&D expense year-to-date in 2021 was primarily a result of an increase related to start-up costs for our WM pivotal study and other clinical project costs and general research and development costs offset by lower manufacturing and related costs.
General and Administrative Expense: G&A expense for the three months ended June 30, 2021 was $1.4 million compared to $1.2 million for the three months ended June 30, 2020. The cumulative G&A spending for the first six months of 2021 were of $3.1 million as compared to $2.5 million for the first six months of 2020. The increase in G&A expense year-to-date in 2021 was primarily a result of an increase in professional fees, insurance and personnel costs.

Net Loss: The net loss attributable to common stockholders for the three months ended June 30, 2021 was ($6.0) million, or ($0.11) per share, compared to ($3.6) million, or ($0.26) per share, in 2020. Net loss attributable to common stockholders for the six months ended June 30, 2021 was ($12.4) million, or ($0.25) per share, compared to ($7.6) million, or ($0.65) per share, in 2020.

On August 9, 2021 Cytocom Inc. (Nasdaq: CBLI), a leading biopharmaceutical company creating next-generation immune therapies that focus on immune restoration and homeostasis, reported an update regarding its portfolio of clinical programs (Press release, Cleveland BioLabs, AUG 9, 2021, View Source [SID1234586175]).

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"We are excited and believe that we are well positioned to further the development of our clinical-stage pipeline and showcase the power of our expanded post-merger drug development capabilities," stated Michael K. Handley, President and CEO of Cytocom. "We are strategically focused on immune-modulating treatments designed to address anemia and neutropenia, emergent viruses, cancer, and autoimmune diseases."

Mr. Handley continued, "The coming months will be busy. We are completing the necessary steps to begin enrolling patients by year-end 2021 in a Phase 3 clinical trial for our lead drug candidate, CYTO-201, for the treatment of Crohn’s disease. Building on our legacy Cytocom pipeline are the Cleveland BioLabs assets, specifically entolimod, an immune-stimulatory agent. We are eager to explore new indications for entolimod and remain excited about the potential for toll-like receptor 5 agonists in treating neutropenia and anemia in cancer patients. Our team is working to put together development plans in hematology and we are in talks with a renowned medical facility to begin a clinical study using entolimod in the coming months."

Mr. Handley concluded, "In addition, we plan to follow in the first half of 2022 with a clinical trial exploring CYTO-401 as an adjunct to standard of care therapy to extend the duration of disease remission in patients with pancreatic cancer. We are also completing the necessary steps to activate clinical trial sites and begin enrolling patients in clinical trials exploring CYTO-205 in patients with acute and post-acute COVID-19. This could be a particularly compelling opportunity given the pernicious nature of the SARS-CoV-2 virus, which, despite growing vaccination rates, continues to impact millions of people worldwide. What’s more, the medical community has specifically expressed interest on a "long haulers" study."

CYTO-201 and Crohn’s Disease

Cytocom’s lead investigational drug candidate, CYTO-201, is being studied for the treatment of pediatric patients with Crohn’s disease, an inflammatory bowel disease that causes chronic inflammation of the gastrointestinal (or digestive) tract, causing symptoms such as persistent diarrhea, abdominal pain and rectal bleeding. Studies show that because the signs and symptoms of the disease are unpredictable, patients living with the disease endure significant burdens, not only physical, but also emotional and economic.

On July 27, 2021, Cytocom completed a successful end of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) regarding a clinical development plan for a Phase 3 clinical trial evaluating CYTO-201 in pediatric Crohn’s patients. Cytocom is partnering with ICON plc (NASDAQ: ICLR), a global contract research organization (CRO), to manage the trial, and expects to begin enrolling patients by year-end 2021.

Entolimod and Hematology

Cytocom’s new management team is now reviewing information regarding the past development work for entolimod, as well as previous clinical trial data. The company plans to address the clinical hold imposed by the U.S. Food and Drug Administrations (FDA) in 2019 in order to initiate clinical trials for new indications. In addition to continuing work on the Acute Radiation Syndrome or ARS, Cytocom plans to explore new indications for entolimod based on the potential of toll-like receptor 5 agonists in hematology indications, specifically the treatment of neutropenia and anemia in cancer patients. Cytocom is developing a hematology program and discussions are underway with a renowned academic institution to potentially initiate the first study later this year using entolimod in a hematology indication.

CYTO-401 and Pancreatic Cancer

Cytocom is developing CYTO-401 as an adjunct to standard of care therapy to extend the duration of disease remission in patients with pancreatic cancer. In August 2021, the company received FDA feedback regarding the clinical development program. Having feedback on the development program and establishing an advisory panel of oncology experts, Cytocom is now evaluating contract research organizations (CROs) and is planning to initiate the clinical trial in the first half of 2022.
CYTO-205 and Acute and Post-Acute COVID-19

With the FDA having cleared an Investigational New Drug (IND) application, Cytocom expects to begin a Phase 2 clinical trial in the third quarter of 2021 to evaluate the safety and efficacy of CYTO-205 to slow or halt the progression of the SARS-CoV-2, the virus that causes COVID-19.

Cytocom is finalizing protocols for a study evaluating CYTO-205 in patients with post-acute COVID-19 syndrome (PACS). Also known as "long haulers," these patients represent a high unmet medical need with roughly 30% of all COVID-19 infections developing into long-haul syndrome. Cytocom plans to conduct the trial under the existing IND and expects to begin enrolling patients by year-end 2021. CYTO-205 is designed to modulate immune system function by decreasing elevated inflammatory responses associated with viral infection.