On May 5, 2022 Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, reported financial results for the quarter ended March 31, 2022, and highlighted recent progress (Press release, Elevation Oncology, MAY 5, 2022, https://elevationoncology.com/elevation-oncology-reports-first-quarter-2022-financial-results-and-highlights-recent-company-progress/?utm_source=rss&utm_medium=rss&utm_campaign=elevation-oncology-reports-first-quarter-2022-financial-results-and-highlights-recent-company-progress [SID1234613642]).

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"CRESTONE is an important study for both Elevation Oncology and for patients with NRG1 fusions, a genomically defined patient population with no approved therapies addressing the unmet medical need. We are honored for the opportunity to share the initial and first ever clinical data of seribantumab in this patient population through an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting in June 2022." said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "In addition to executing on CRESTONE, we are building an industry-leading team, led by people like Dr. David Dornan, our newly appointed Chief Scientific Officer, who brings a wealth of cancer drug development experience to advance our mission of maximizing the potential of seribantumab and building out an industry-leading precision oncology pipeline. Together with our diagnostic and academic partners, we look forward to advancing the field of precision oncology by expanding upon our operational platform and continuing to make genomic testing results therapeutically actionable."

Recent Progress and Highlights
Seribantumab

Initial CRESTONE data selected for oral presentation at ASCO (Free ASCO Whitepaper) 2022. ASCO (Free ASCO Whitepaper) abstracts to be published on May 26, 2022 at 5:00PM ET. The presentation, scheduled for June 7, 2022, from 9:45AM-12:45PM CT, will highlight initial clinical data from approximately 10 patients from Cohort 1 in the ongoing Phase 2 CRESTONE study evaluating seribantumab at 3 grams weekly in patients with solid tumors harboring NRG1 gene fusions.
Corporate

David Dornan, PhD appointed Chief Scientific Officer. In March, Elevation Oncology announced the appointment of Dr. Dornan, PhD, as the Company’s first Chief Scientific Officer, bringing deep oncology R&D and drug development expertise and further strengthening the leadership team.
Expected Upcoming Milestones and Operational Objectives
Initial Phase 2 CRESTONE data to be presented in an oral presentation at ASCO (Free ASCO Whitepaper) 2022
Complete enrollment of the first 20 patients in Cohort 1 of the CRESTONE study in mid-2022
Ongoing target evaluation and continued execution of our strategy for future pipeline expansion
First Quarter 2022 Financial Results
As of March 31, 2022, the Company had cash, cash equivalents and marketable securities totaling $132.1 million, compared to $146.3 million as of December 31, 2021.

Research and development expenses for the first quarter 2022 were $13.6 million, compared to $4.1 million for the first quarter 2021. The increase in R&D expense was primarily related to an increase in manufacturing, personnel costs and other expenses associated with the CRESTONE study.

General and administrative expenses for the first quarter 2022 were $3.8 million, compared to $1.0 million for the first quarter 2021. The increase in G&A expense was primarily related to personnel costs, professional services and other administrative costs.

Net loss for the first quarter 2022 was $17.3 million, compared to $5.1 million for the first quarter 2021.

Financial Outlook
Elevation Oncology expects its existing cash, cash equivalents and marketable securities as of March 31, 2022 will be sufficient to fund its current operations into the fourth quarter of 2023.

About Seribantumab and NRG1 Gene Fusions
Seribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins." The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are predominantly mutually exclusive of other known genomic driver mutations and are considered a unique oncogenic driver event associated with tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, cholangiocarcinomas, and sarcomas. In preclinical experiments, seribantumab prevented the activation of HER3 signaling in cells that harbor an NRG1 gene fusion and destabilized the entire ERBB family signaling pathway, including the activation of HER2, EGFR, and HER4. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to more than 800 patients across twelve Phase 1 and 2 studies, both as a monotherapy and in combination with various anti-cancer therapies. Seribantumab is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the Phase 2 CRESTONE Study
Clinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions (CRESTONE) is a Phase 2 tumor-agnostic "basket trial" evaluating the safety and efficacy of seribantumab in patients with solid tumors that harbor an NRG1 fusion and have progressed after at least one prior line of standard therapy. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab as a monotherapy specifically in patients whose solid tumor is uniquely driven by an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling patients in the United States, Australia, and Canada. For more information visit www.NRG1fusion.com.

On May 5, 2022 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported financial results for the first quarter ended March 31, 2022 and provided a clinical update on its Phase 3 clinical program of simufilam in Alzheimer’s disease (Press release, Pain Therapeutics, MAY 5, 2022, View Source [SID1234613641]). Simufilam is Cassava Sciences’ lead drug candidate for the proposed treatment of Alzheimer’s disease.

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Net loss was $17.5 million, or $0.44 per share, compared to a net loss of $3.5 million, or $0.09 per share, for the same period in 2021. Net cash used in operations was $23.5 million during the first quarter of 2022, including over $10 million of contractual pre-payments. Pre-payments are amounts paid for in advance of future research and development (R&D) services.

"We’re seeing an uptick in the rate of patient enrollment," said Remi Barbier, President & CEO. "In part, the pandemic challenged some clinical sites, from staffing shortages to operational gaps. We hope these challenges are in the rearview mirror as we continue to move forward with our Phase 3 studies of simufilam in Alzheimer’s disease, while keeping an eye on the balance sheet."

Financial Results for First Quarter 2022

At March 31, 2022, cash and cash equivalents were $209.7 million, with no debt.

Net loss was $17.5 million, or $0.44 per share. This compares to a net loss of $3.5 million, or $0.09 per share, for the same period in 2021. Net loss increased compared to the prior period due primarily to a significant increase in our R&D activities and G&A expenses.

Net cash used in operations was $23.5 million during the first quarter of 2022.

Net cash use in operations for the first half 2022 is now expected to be approximately $35 to $40 million, including significant (over $10 million) contractual pre-payments made to R&D vendors for future services, such as our contract research organization for the Phase 3 clinical program of simufilam in Alzheimer’s disease.

Research and development (R&D) expenses were $14.9 million. This compared to $2.5 million for the same period in 2021. R&D expenses increased compared to the prior period due primarily to increased activities and expenses related to clinical studies, clinical trial supplies and support functions and personnel expenses.

General and administrative (G&A) expenses were $2.9 million. This compared to $1.0 million for the same period in 2021. G&A expenses increased compared to the prior period due primarily to increased activities and expenses related to legal services and depreciation and amortization.
Overview of On-going Phase 3 Clinical Program
Our Phase 3 program consists of two double-blind, randomized, placebo-controlled studies of simufilam in patients with mild-to-moderate Alzheimer’s disease. Both Phase 3 studies have Special Protocol Assessments (SPA) from the U.S. Food and Drug Administration.

A total of over 120 subjects have now been enrolled in our Phase 3 studies. Studies are being conducted in over 115 clinical trial sites across the U.S., Canada and Puerto Rico.

Overview of Each On-going Phase 3 Study – RETHINK-ALZ and REFOCUS-ALZ
Our Phase 3 study called "RETHINK-ALZ" is designed to evaluate the safety and efficacy of oral simufilam 100 mg in enhancing cognition and slowing functional decline over 52 weeks. This randomized, double-blind, placebo-controlled study plans to enroll approximately 750 patients with mild-to-moderate Alzheimer’s disease.

Details of the RETHINK-ALZ Phase 3 study include:

Subjects are randomized (1:1) to simufilam 100 mg or placebo twice daily.
The co-primary efficacy endpoints are ADAS-Cog12 (a cognitive scale) and ADCS-ADL (a functional scale). A secondary efficacy endpoint is iADRS, a clinical tool that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
Our Phase 3 study called "REFOCUS-ALZ" is designed to evaluate the safety and efficacy of oral simufilam 100 mg and 50 mg over 76 weeks. This randomized, double-blind, placebo-controlled study plans to enroll approximately 1,000 patients with mild-to-moderate Alzheimer’s disease.

Details of the REFOCUS-ALZ Phase 3 study, include:

Subjects are randomized (1:1:1) to simufilam 100 mg, 50 mg, or placebo twice daily.
The co-primary efficacy endpoints are ADAS-Cog12 (a cognitive scale) and ADCS-ADL (a functional scale). A secondary efficacy endpoint is iADRS, a clinical tool that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
Open-label Study – closed enrollment

In March 2020, we initiated a long-term, open-label study to evaluate simufilam, our lead drug candidate, in patients with mild-to-moderate Alzheimer’s disease. The study is intended to monitor the long-term safety and tolerability of simufilam 100 mg twice daily for 12 or more months. The open-label study has reached its final target enrollment of approximately 200 subjects with Alzheimer’s disease. We expect to announce full study results second half 2022.

Cognition Maintenance Study (CMS) – on-going
In May 2021, we initiated a Cognition Maintenance Study (CMS). This is a double-blind, randomized, placebo-controlled study of simufilam in patients with mild-to-moderate Alzheimer’s disease. Study participants are randomized (1:1) to simufilam or placebo for six months. To enroll in the CMS, patients must have previously completed 12 months or more of open-label treatment with simufilam. The CMS is designed to evaluate simufilam’s effects on cognition and health outcomes in Alzheimer’s patients who continue with drug treatment versus patients who discontinue drug treatment. The target enrollment for the CMS is approximately 100 subjects. Over 75 subjects have been enrolled in the CMS and 35 have completed the study.

SavaDx – on-going
SavaDx, is an early-stage program focused on detecting the presence of Alzheimer’s disease from a small sample of blood. For business, technical and personnel reasons, we continue to prioritize the development of simufilam, our lead drug candidate, over SavaDx.

About Simufilam
Simufilam (sim-uh-FILL-am) is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA) protein in the brain. Cassava Sciences owns worldwide development and commercial rights to its research programs in Alzheimer’s disease, and related technologies, without royalty obligations to any third party.

On May 5, 2022 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq: RDUS), reported its financial results for the first quarter ended March 31, 2022. The Company also provided a general business update (Press release, Radius, MAY 5, 2022, View Source [SID1234613638]).

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Radius CEO Kelly Martin provided the following commentary covering a variety of topics:

"Failure to meet the prespecified primary endpoint in the long awaited abaloparatide transdermal system pivotal trial resulted in a swift and precipitous 50+% drop in the Radius share price during the month of December. Conversely, our convertible notes, as an indicator for the credit market assessment of the Radius balance sheet, cash flows and business fundamentals, were basically unchanged in price and value during that same period."

Martin added, "against this dislocation and backdrop, our approach is to ‘control what we can control’ and focus on advancing the Radius business on behalf of shareholders, creditors, patients and employees."

"To that end, we are working closely with our partner – the Menarini Group – on all aspects of elacestrant. Recent competitor trial failures in the SERD space may expand the potential commercial opportunity – and subsequent value – for the asset if approved. Our financial participation is realized through achievement of both milestones and royalties and, importantly, incorporates all indications, geographies and any combination therapies following the monotherapy data and potential approval. As announced in February, we will return 100% of the value generated by elacestrant to our capital providers – both creditors as well as shareholders."

"For RAD011, we are progressing this molecule in a deliberate and disciplined manner with a focus on specific genetically based and orphan neurological/behavioral diseases. Interest in the program – post the April R&D webcast – has been, and continues to be, noteworthy. Clear and specific patient need, little to no treatment options, pre-existing data and a concise regulatory path leading to single pivotal trial readouts in 2H 2024 underscore the characteristics of the opportunity."

"Lastly, for TYMLOS we are preparing for a potential US launch for the male indication, assisting our partners Paladin Labs in Canada and Teijin Pharma in Japan for launches in their respective countries. As per our previously stated goal to ‘expand the global footprint of the asset’ we expect to finalize up to three additional geographic/partner agreements over the coming months, and plan for up to four ex-US market launches in 2023. We have also completed all necessary steps for the EU re-submission and await that decision in 2H 2022."

In summary, Martin concluded, "as mentioned in our FY 2021 earnings in February, the recent market dislocation in biotechnology has been significant. We expect these challenges to continue throughout 2022. Within this environment, Radius continues to have the opportunity to differentiate itself by remaining active with the balance sheet and monitoring cash flow, managing the risk/reward dynamics of the three assets and, lastly, incorporating overall corporate timelines and potential business inflection points."

Q1 2022 FINANCIAL HIGHLIGHTS

Revenues

Total net revenues for the quarter were $43 million compared to $56 million in Q1 2021, which included $11 million in one-time payments received for abaloparatide approval in Japan and licensing in Canada
TYMLOS net product revenue was $43 million compared to $45 million in Q1 2021, down 5%. This modest decrease was expected in Q1 and was driven primarily by inventory destocking and seasonal payer dynamics
Patient Growth: average active patients on therapy increased by 4% compared to Q1 2021
Reiterate full year 2022 guidance of $232 million with 42% of net revenue estimated to come in 1H ($97 million) and 58% ($135 million) in 2H 2022. The Q1 net revenue is on track to meet the 1H guidance
Productivity and Headcount

Total headcount for the quarter was 258 compared to 294 in Q1 2021, down 12%; and down 31% compared to 376 in Q1 2020
Dramatic Productivity Improvements: TYMLOS net revenue per commercial employee was $374k in Q1 2022 compared to $283k in Q1 2021, up 32%; compared to Q1 2020, commercial productivity has improved by 66%
The Company continues to employ a ‘hybrid work environment’. Real estate footprint vs. 2020 has been reduced by nearly 85% and those associated infrastructure costs are down by ~65%
P&L

Net Loss of ($18.3) million in Q1 2022 vs ($15.7) million in Q1 2021
Adjusted EBITDA (Non-GAAP) loss of ($9.1) million Q1 2022 vs. ($4.6) million Q1 20211
Diluted EPS (GAAP): ($0.39) in Q1 2022 vs. ($0.34) in Q1 2021
Balance Sheet

$72 million of cash, cash equivalents and marketable securities as of March 31, 2022
Decrease in cash driven by change in net working capital ($19 million), annual bonus payouts, one-time reduction in force (RIF) payouts, and other one-time items
Mark Conley, Chief Financial Officer, commented, "Our first quarter TYMLOS net revenue is in line with our 1H 2022 net revenue guidance of $97 million and today we are reiterating our financial objectives for 2022, including TYMLOS net revenue of $232 million. Our total headcount has reduced further this quarter and our productivity has increased significantly, demonstrating efficient management of our cost structure, and increased operational efficiency."

Conley added, "We ended the quarter with $72 million in cash, driven by several one-time items. We expect cash on the balance sheet to increase as the year progresses."

Key Financial Objectives for 2022

Radius reiterates its financial objectives for 2022, as set out in its press release dated February 24, 2022, which included:

$232 million TYMLOS Net Revenue (42% estimated in 1H and 58% in 2H 2022)
($5) to $5 million Net Loss
$35 to $45 million Company Adjusted EBITDA (Non-GAAP)
ASSETS UPDATE

Elacestrant

Elacestrant is the first and currently only investigational oral SERD to show positive topline results in a pivotal trial as a monotherapy vs. standard of care (SoC) for the treatment of ER+HER2-advanced or metastatic breast cancer (mBC).

Recent competitor trial failures may broaden the opportunity for elacestrant
Differentiator of EMERALD from other SERD trials: two primary endpoints, all-comers and ESR1 mutant subgroup
Current focus: robust clinician engagement, life cycle and ‘go to market’ activity
Regulatory submissions: on track to file in the US in Q2 2022 with EU filing to follow the US filing
Life cycle management: Menarini Group is working to further develop elacestrant in the adjuvant setting, combination therapy, and metastatic breast cancer that has metastasized to the brain
RAD011

RAD011 is the Company’s investigational synthetic cannabidiol oral solution which has potential utilization in multiple neuro-endocrine, neurodevelopmental, or neuropsychiatric disease areas.

Initiating two single pivotal trials in Angelman syndrome (AS) (Q3) and Prader-Willi syndrome (PWS) (Q2)
SCOUT-015 study evaluating hyperphagia in PWS is open for recruitment and has begun patient screening in the US
Plans to follow AS and PWS trials with a Phase 2 infantile spasms (IS) trial evaluating spasm resolution
Dup15q syndrome: first company to receive Orphan Drug Designation; possible life cycle opportunity
Orphan Drug Designations have been granted for all four of the above indications in the US
On April 5, 2022, Radius hosted an R&D webcast dedicated to RAD011; a replay of the webcast and presentation is available on the Company’s website (View Source)
ABALOPARATIDE

TYMLOS in Male Indication

As previously reported on March 1, 2022, the Company filed a Supplemental New Drug Application (sNDA) with the US Food and Drug Administration (FDA) for TYMLOS (abaloparatide) subcutaneous injection in men with osteoporosis at high risk for fracture.

10-month review process and, subject to approval, plan to launch in early Q1 2023
Estimated that 30% of all hip fractures occur in men and approximately 20% of men over the age of 50 will experience an osteoporosis-related fracture in their lifetime
The full data set from the ATOM Phase 3 study will be presented at the upcoming American Association of Clinical Endocrinology (AACE) Annual Meeting on May 12-14, 2022
TYMLOS Intellectual Property

On March 8, 2022, Radius announced the United States Patent and Trademark Office (USPTO) granted patent 11,255,842, which extends TYMLOS exclusivity to January 10, 2040
This adds to the Company’s four issued patents covering TYMLOS, which provide significant depth and breadth in protecting the commercial runway of the asset
Financial Results

Three Months Ended March 31, 2022

Net Loss
For the three months ended March 31, 2022, Radius reported a net loss of $18.3 million, or $0.39 per share, compared to a net loss of $15.7 million, or $0.34 per share, for the three months ended March 31, 2021.

For the three months ended March 31, 2022, Adjusted EBITDA (Non-GAAP), was ($9.1) million, or $0.19 per share, compared to Adjusted EBITDA (Non-GAAP) of ($4.6) million, or $0.10 per share, for the three months ended March 31, 2021.

Revenue
For the three months ended March 31, 2022, TYMLOS net product revenues were $43.0 million compared to $45.3 million for the three months ended March 31, 2021.

For the three months ended March 31, 2022, $0.2M in license revenue was recognized compared to $11.0 million recognized for the three months ended March 31, 2021.

Costs and Expenses
For the three months ended March 31, 2022, research and development expense was $22.7 million compared to $31.4 million for the three months ended March 31, 2021, a decrease of $8.7 million, or 28%. This decrease was primarily driven by a decrease of $7.3 million in abaloparatide-TD program costs, a $5.5 million decrease in abaloparatide-SC program costs, a $0.8 million decrease in professional fees driven by billed reimbursable consultant costs, and a $2.1 million decrease in elacestrant program costs, which is comprised of a $7.3 million decrease in gross program expenses as well as a $5.2 million increase in billed reimbursable expenses. These decreases were offset by a $6.1 million increase in RAD011 program costs, a $0.4 million increase in occupancy and depreciation costs, and a $0.4 million increase in compensation expense, which is comprised of a $1.0 million increase in compensation expense related to headcount offset by $0.5 million of billed reimbursable expenses.

For the three months ended March 31, 2022, selling, general and administrative expenses were $30.0 million compared to $34.1 million for the three months ended March 31, 2021, a decrease of $4.1 million, or 12%. This decrease was primarily the result of a $3.5 million decrease in professional support costs and a $1.2 million decrease in wages and employee benefit costs due to a decrease in headcount. These decreases were offset by a $1.4 million increase in occupancy and depreciation costs and other operating costs.

Webcast and Conference Call

In connection with today’s reporting of First Quarter 2022 Financial Results, Radius will host a conference call and live audio webcast at 8:30 a.m. ET today, May 5, 2022, to review financial results and provide a Company update.

Conference Call Information:
Date: May 5, 2022
Time: 8:30 a.m. ET
Domestic Dial-In Number: 1 (866) 323-7965
International Dial-In Number: 1 (346) 406-0961
Conference ID: 5844208
Webcast Link: View Source

A live audio webcast of the call can be accessed from the Investors section of the Company’s website, www.radiuspharm.com. The full text of the announcement and financial results will also be available on the Company’s website.

A replay of the conference call will be available on May 5th at 11:30 a.m. ET. A live audio webcast of the call will be archived on the Company’s website for 12 months. To access the replay, dial (855) 859-2056 or (404) 537-3406 for International, using conference ID number 5844208. The live audio webcast of the call can be accessed from the Investors section of the Company’s website, View Source. The full text of the announcement and financial results will also be available on the Company’s website.

Use of Non-GAAP Financial Measures
To supplement our condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), we use the following non-GAAP financial measures in this press release: Adjusted EBITDA and Adjusted EBITDA per share. The Company defines adjusted EBITDA as net income before interest, taxes, depreciation and amortization, adjusted for the impact of certain additional non-cash and other items that management does not consider in its evaluation of ongoing performance of the Company’s core operations. These items include stock-based compensation expense and other one-time expenses. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with Radius’ GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to Radius’ operating performance and can enhance investors’ ability to identify operating trends in our business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of Radius’ operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2022 and 2021 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

This release includes forward-looking guidance for adjusted EBITDA. The Company is not able to provide, without unreasonable effort, a reconciliation of the guidance for adjusted EBITDA to the most directly comparable GAAP measure because the Company does not currently have sufficient data to accurately estimate the variables and individual adjustments included in the most directly comparable GAAP measure that would be necessary for such reconciliations, including (a) one-time items or other expenses that we do not believe are indicative of our ongoing operations. These adjustments are inherently variable and uncertain and depend on various factors that are beyond our control and as a result we are also unable to predict their probable significance. Therefore, because management cannot estimate on a forward-looking basis without unreasonable effort the impact these variables and individual adjustments will have on its reported results in accordance with GAAP, it is unable to provide a reconciliation of the non-GAAP measures included in its 2022 guidance.

On May 5, 2022 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, reported financial results for the first quarter ended March 31, 2022, as well as recent business highlights (Press release, C4 Therapeutics, MAY 5, 2022, View Source [SID1234613637]).

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"We recently shared our first clinical data for single agent CFT7455 that demonstrated its unique properties, including differentiated pharmacokinetics and potency compared to approved and investigational IKZF1/3 degraders and we are now focused on optimizing dose and schedule to improve the therapeutic index and advance the program in multiple myeloma and non-Hodgkin’s lymphomas," said Andrew Hirsch, chief executive officer of C4 Therapeutics. "At AACR (Free AACR Whitepaper), we also presented data highlighting the capability of our TORPEDO platform to develop highly potent and selective degraders against three distinct oncology target classes that we are progressing to the clinic. Our strong balance sheet provides runway to enable us to execute our strategy of optimizing the dosing regimen for CFT7455 and advance CFT8634, CFT1946 and CFT8919."

FIRST QUARTER 2022 AND RECENT HIGHLIGHTS

CFT7455: CFT7455 is a novel degrader candidate targeting IKZF1/3 for the treatment of multiple myeloma (MM) and non-Hodgkin’s lymphomas (NHL), including peripheral T-cell lymphoma and mantle cell lymphoma.

Presented Clinical Data from Cohort A at AACR (Free AACR Whitepaper): In April 2022, C4T presented clinical data from Cohort A of its ongoing CFT7455 Phase 1/2 clinical trial. Data demonstrated that single agent CFT7455 resulted in deep and durable degradation of IKZF1/3, as quantified by mass spectrometry, and meaningful decreases in serum free light chain. Neutropenia, a known on-target toxicity associated with IKZF1/3 degraders, was dose-limiting at the 50 μg daily 21 day on/7 day off starting dose and schedule. No serious adverse events were reported.
Progressed Ongoing Phase 1/2 Clinical Trial: Enrollment is ongoing in Cohort B1, exploring CFT7455 as a monotherapy for relapsed or refractory MM, and Cohort C, exploring CFT7455 as a monotherapy for NHL.
Presented Pre-clinical Data at AACR (Free AACR Whitepaper): In April 2022, C4T characterized the chemical structure of CFT7455, the resulting improvements in potency and optimized pharmacokinetic properties. In vitro data suggested that CFT7455 resulted in a high cereblon binding affinity (KD = 0.9 nM) along with rapid, selective and deep degradation of IKZF1/3 that is associated with apoptosis, leading to broad and potent antiproliferative activity in a panel of MM cell lines. In vivo MM models treated with CFT7455 indicated regression in the treatment-naïve H929 MM tumor models at doses ≥10 µg/kg/day, as well as durable antitumor responses consistent with long-lived pharmacodynamic activity. Moreover, CFT7455 was observed to be efficacious in MM models resistant or insensitive to currently approved IMiD treatments.
CFT8634: CFT8634 is a degrader candidate targeting BRD9 for the treatment of synovial sarcoma and SMARCB1-null solid tumors.

Activated Sites for Phase 1/2 Clinical Trial: With sites now active, C4T remains on track to dose the first patient in the Phase 1/2 clinical trial of CFT8634 in synovial sarcoma and SMARCB1-null solid tumors in 1H 2022. The Phase 1/2 trial will primarily investigate safety, tolerability and anti-tumor activity, with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT8634. The Phase 1 portion of the study will evaluate CFT8634 as an oral, single agent therapy for patients with synovial sarcoma and SMARCB1-null solid tumors to identify a recommended Phase 2 dose.
Presented Pre-clinical Data at AACR (Free AACR Whitepaper): In April 2022, C4T presented pre-clinical data on the discovery and characterization of CFT8634, a BiDAC degrader of BRD9. The pre-clinical data demonstrated that CFT8634 selectively inhibits the growth of BAF-perturbed cell lines and demonstrates robust efficacy in clinically relevant patient-derived xenograft models of synovial sarcoma. This pre-clinical data suggests that CFT8634 is a potent and selective degrader of BRD9 in vitro.
Received Orphan Drug Designation: In March 2022, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to CFT8634 for the treatment of soft tissue sarcoma.
CFT1946: CFT1946 is a mutant-selective degrader of BRAF V600X for the treatment of V600 mutant solid tumors.

Presented Pre-clinical Data at AACR (Free AACR Whitepaper): In April 2022, C4T presented its pre-clinical evaluation of CFT1946. Data demonstrated that CFT1946 induces on-mechanism BRAF-V600E degradation, potent inhibition of MAPK signaling and loss of viability in BRAF-V600E cells without impacting wild type-BRAF. In addition, CFT1946 is active in vitro and in vivo in models with BRAF-V600E-driven disease and in the escape mutant BRAF-V600E/NRAS-Q61K-driven model, which is a model of clinical resistance to BRAF inhibitors.
Corporate

Named Bruce Downey as Lead Independent Director: In January 2022, C4T named Bruce Downey as lead independent director. Mr. Downey chairs C4T’s Organization, Leadership and Compensation Committee and also serves on the Audit Committee and Nominating and Corporate Governance Committee. Mr. Downey previously served as chairman and chief executive officer of Barr Pharmaceuticals.
Appointed Utpal Koppikar to Board of Directors: In March 2022, C4T appointed Utpal Koppikar, MBA, to its board of directors, where Mr. Koppikar will also serve as chair of the Audit Committee and a member of the Organization, Leadership and Compensation Committee. Mr. Koppikar is currently the chief financial officer of Atara Biotherapeutics.
KEY UPCOMING MILESTONES

The company anticipates the following milestones:

CFT7455: Continued enrollment of the Phase 1/2 trial throughout 2022. These efforts will inform the identification of a recommended Phase 2 dose(s) and schedule(s) for MM and NHL.
CFT8634: Dose the first patient in CFT8634 Phase 1/2 clinical trial in 1H 2022 and continue to enroll the trial throughout 2022. These efforts will inform the identification of a recommended Phase 2 dose for synovial sarcoma and SMARCB1-null solid tumors.
CFT1946: Submit an IND application and initiate a Phase 1 trial of CFT1946 in BRAF V600X-driven cancers including melanoma, colorectal and non-small cell lung cancer in 2H 2022.
CFT8919: Complete IND-enabling activities for CFT8919 by year-end 2022.
FIRST QUARTER 2022 FINANCIAL RESULTS

Revenue: Total revenue for the first quarter of 2022 was $7.7 million, compared to $7.4 million for the first quarter of 2021. Total revenue reflects revenue recognized under collaboration agreements with Roche, Biogen and Calico.

Research and Development (R&D) Expense: R&D expense for the first quarter of 2022 was $26.2 million, compared to $20.5 million for the first quarter of 2021. The increase in R&D expense was primarily attributable to increased personnel expenses, including an increase in stock compensation expenses of $2.4 million.

General and Administrative (G&A) Expense: G&A expense for the first quarter of 2022 was $12.8 million, compared to $7.4 million for the first quarter of 2021. The increase in G&A expense was primarily attributable to increased personnel expenses, including an increase in stock compensation expense of $2.6 million, and higher facilities costs resulting from additional leased space.

Net Loss and Net Loss per Share: Net loss for the first quarter of 2022 was $31.6 million, compared to $21.0 million for the first quarter of 2021. Net loss per share for the first quarter of 2022 was $0.65, compared to $0.49 for the first quarter of 2021.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of March 31, 2022, were $421.7 million, compared to $451.5 million as of December 31, 2021. The decrease in cash was primarily driven by expenditures to fund operations. C4T expects that its cash, cash equivalents and marketable securities as of March 31, 2022, together with future payments expected to be received under existing collaboration agreements, will be sufficient to fund planned operating expenses and capital expenditures to the end of 2024.

On May 5, 2022 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases, enhance gene therapies and mitigate unwanted immune responses to biologics, reported financial results for the first quarter ended March 31, 2022 and provided a business update (Press release, Selecta Biosciences, MAY 5, 2022, View Source [SID1234613636]).

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"In the first quarter of 2022, we executed across our pipeline and proactively took measures to extend our cash runway into mid-2024," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "In early April, we successfully completed an underwritten offering, raising an additional $38.7 million in gross proceeds, further strengthening our financial resources which we believe will carry us through key value-driving milestones and help Selecta realize the full potential of our leading precision immune tolerance platform. The synergistic effects of ImmTOR in combination with a Treg-selective IL-2 (ImmTOR-IL) has the potential to be a first-in-class, antigen-specific immunotherapy, with broad applications across all three pillars of our pipeline. Furthermore, we believe we are well-positioned to deliver on our clinical development strategy across our wholly owned pipeline while continuing to pursue strategic partnerships in gene therapy and biologics. We remain on track to deliver on multiple upcoming catalysts in 2022, including the initiation of a Phase 1 trial of SEL-302, our wholly owned gene therapy in combination with ImmTOR for the treatment MMA, the completion of the Phase 3 DISSOLVE clinical program in chronic refractory gout and several IND-enabling studies that collectively advance our strategy for re-imagining immunotherapy for autoimmune disease, unlocking the potential of AAV gene therapy and amplifying the efficacy of biologics."

Recent Highlights and Anticipated Upcoming Milestones:

Tolerogenic Therapies for Autoimmune Disease

ImmTOR with proprietary IL-2 protein agonist (ImmTOR-IL): Pre-clinically, Selecta has observed synergistic activity when ImmTOR is combined with engineered IL-2 molecules that are selective for Tregs. When ImmTOR-IL was co-administered with an antigen of interest in a preclinical study, the resulting preclinical data suggest that ImmTOR may have profound synergistic effects in further expanding antigen-specific Tregs when compared to ImmTOR alone, positioning it to be a potential first-in-class antigen-specific therapy for the treatment of autoimmune disease.
Selecta is working with its partner, Cyrus Biotechnology, to develop a next generation IL-2 molecule to combine with ImmTOR.
Selecta continues work toward identifying suitable target indications and accelerating the development of ImmTOR-IL to the clinic.
Primary biliary cholangitis (PBC): Selecta continues IND-enabling work on an ImmTOR platform approach to treating PBC.
Gene Therapies:

SEL-302 for methylmalonic acidemia (MMA): On March 9, 2022, the FDA removed the clinical hold on SEL-302 for the treatment of patients with MMA.
Selecta expects initiation of the Phase 1 clinical trial of SEL-302 in the second half of 2022.
SEL-018 IgG Protease (Xork): In collaboration with Genovis, Selecta continues to advance Xork, a next-generation IgG protease, to help address disease in those patients who are ineligible for gene therapies due to pre-existing anti-AAV antibodies. Selecta believes the novel combination of Xork and ImmTOR has the potential to simultaneously address two of the key hurdles in gene therapy today: pre-existing immunity and the inability to re-dose AAV gene therapies.
IND-enabling studies are expected to commence in 2022.
Biologic Therapies:

SEL-212 for chronic refractory gout: Selecta continues to advance DISSOLVE, the Phase 3 development program of SEL-212, which has been licensed to Swedish Orphan Biovitrum AB (publ.) ("Sobi").
On December 1, 2021, Selecta announced complete enrollment for DISSOLVE I, currently being run in the United States.
DISSOLVE II continues to enroll, with trial sites in the United States and four Eastern European countries. Screening and randomization in both Russia and Ukraine have been temporarily closed to preserve study supplies in these countries for those already enrolled. Moreover, 11 additional sites in the United States have been activated to speed enrollment and help mitigate any potential disruptions from the closure of screening and randomization in Russia and Ukraine, and DISSOLVE II enrollment has been increased to approximately 140 study participants.
DISSOLVE I & II studies are on track for completion in Q4 2022 with joint topline readout expected in Q1 2023.
ImmTOR with IgA1 protease for IgA nephropathy: Selecta is working with both Ginkgo Bioworks and IGAN Biosciences to identify and develop a next generation IgA protease to combine with ImmTOR.
Selecta anticipates enzyme candidate selection to be completed in 2022.
Corporate Updates:

Completed underwritten offering of common stock and warrants, raising approximately $38.7 million in gross proceeds.
Amended outstanding term loan to defer principal amortization period to April 1, 2023.
Performed strategic review and portfolio prioritization, pausing SEL-313, OTC-D gene therapy development and increasing focus on providing modality-enabling solutions to AAV gene therapy companies.
First Quarter 2022 Financial Results

Cash Position: Selecta had $118.8 million in cash, cash equivalents, marketable securities, and restricted cash as of March 31, 2022, as compared to cash, cash equivalents, marketable securities, and restricted cash of $129.4 million as of December 31, 2021. With the approximately $36.0 million in net proceeds raised from the April underwritten offering, Selecta believes its available cash, cash equivalents, restricted cash, and marketable securities will be sufficient to meet its operating requirements into mid-2024. Net cash used in operating activities was $11.9 million for the quarter ended March 31, 2022, as compared to $12.1 million of cash used in operating activities for the same period in 2021.

Collaboration and License Revenue: Collaboration and license revenue for the first quarter of 2022 was $34.0 million, as compared to $11.1 million for the same period in 2021. Revenue was primarily driven by the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program under the license agreement with Sobi.

Research and Development Expenses: Research and development expenses for the first quarter of 2022 were $17.7 million, as compared to $13.0 million for the same period in 2021. The increase in cost was primarily the result of expenses incurred for the preclinical programs, salaries and contract license and milestone payments.

General and Administrative Expenses: General and administrative expenses for the first quarter of 2022 were $5.5 million, as compared to $5.2 million for the same period in 2021. The increase in costs was primarily the result of stock compensation expenses.

Net Income (loss): For the first quarter of 2022, Selecta reported net income of $28.8 million, or basic net income per share of $0.23, compared to net loss of $(24.6) million, or $(0.22) per share, for the same period in 2021.

Conference Call and Webcast Reminder
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s first quarter 2022 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10157872. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.