On May 10, 2022 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company reported that the Company’s product portfolio of FDA-approved brands delivered combined revenues of $11.2 million during the first quarter of 2022, – which is a 6% increase over the prior year period (Press release, Cumberland Pharmaceuticals, MAY 10, 2022, View Source [SID1234614088]). Adjusted earnings for the quarter were $0.5 million, or $0.03 a share. The company ended the first quarter with $96.7 million in total assets, $55.9 million in total liabilities, and $41.0 million of shareholders’ equity. Cumberland will report its first quarter 2022 financial results and provide a company update via a conference call and live internet webcast today at 4:30 p.m. Eastern Time.

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At the start of the first quarter, Cumberland announced its acquisition of the oncology-supportive drug Sancuso from the U.S. subsidiary of Kyowa Kirin, Inc., a Japan-based specialty pharmaceutical company. Sancuso is the first and only FDA-approved prescription patch that prevents nausea and vomiting in cancer patients receiving certain types of chemotherapy treatment. Through the acquisition, Cumberland obtained full commercial responsibility for Sancuso in the U.S., including its marketing, promotion, distribution and manufacturing. Cumberland formed a new sales division, Cumberland Oncology, to support the brand.

On Monday, May 9, 2022, Cumberland announced that it has entered into a co-promotion agreement with Verity Pharmaceuticals to bring Sancuso to more patients across the U.S. Verity will feature Sancuso through its national oncology sales organization, covering a majority of the U.S. market for an initial three-year term.

Cumberland also announced several new partnerships for its Vibativ product during the first quarter. Vibativ is an antibiotic used to treat patients with pneumonia and serious skin infections resulting from gram-positive bacteria:

In March of 2022, the company announced a new partnership with Saudi Arabia-based Tabuk Pharmaceutical Manufacturing Company to introduce Vibativ in the Middle East. The arrangement provided Tabuk exclusive rights to distribute Vibativ in Saudi Arabia and Jordan, with the option to expand into other countries in the region. Tabuk is a fully owned subsidiary of publicly traded Astra Industrial Group, a leading industrial conglomerate in Saudi Arabia. Tabuk is a well-respected pharmaceutical company with a strong distribution network throughout the Middle East.
Cumberland also announced the launch of Vibativ in Puerto Rico, again through a partnership with Verity Pharmaceuticals, which has a particular strength and experience in the Puerto Rican market.
The company continues to enroll patients in the three Phase II clinical programs that it is sponsoring. These studies are evaluating its ifetroban product candidate for:

Duchenne Muscular Dystrophy, a fatal, genetic neuromuscular disease;
Systemic Sclerosis, a debilitating autoimmune disorder; and
Aspirin-Exacerbated Respiratory Disease, a severe form of asthma.
Cumberland is also designing a Phase II study to evaluate the use of ifetroban to treat patients with Progressive Fibrosing Interstitial Lung Diseases. The company is currently preparing an application to the FDA to support the new program.

"We continue to seek opportunities to expand the use of our existing brands, while also building our portfolio of differentiated products," said A.J. Kazimi, CEO of Cumberland Pharmaceuticals. "We made significant progress toward that goal during the first quarter and are particularly encouraged by the addition of Sancuso. We believe in the product’s potential to help more cancer patients and expect it to be a meaningful contributor to our business."

To participate in today’s call, dial (877) 303-1298 (for U.S. callers) or (253) 237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing (855) 859-2056 (for U.S. callers) or (404) 537-3406 (for international callers). The Conference ID for the rebroadcast is 7574078. Both the live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

FINANCIAL RESULTS:

Net Revenue: For the three months ended March 31, 2022, net revenues from continuing operations were $11.2 million.

Net revenue by product for the first quarter of 2022, included $3.9 million for Kristalose, $3.4 million for Sancuso, $2.5 million for Vibativ and $1.0 million for Caldolor.

Operating Expenses: Total operating expenses for the first quarter were $12.5 million.

Adjusted Earnings: Adjusted earnings for the first quarter of 2022 was $0.5 million, or $0.03 per share.

The adjusted earnings calculation does not include the benefit of the $0.9 million of Vibativ cost of goods, which were received with the product acquisition. It also does not include the benefit of the $0.4 million of Sancuso cost of goods, which were received with that product’s acquisition.

Cash Flow: Cash flow from operations for the quarter ended March 31, 2022 was $(0.15) million.

Balance Sheet: At March 31, 2022, Cumberland had $96.7 million in total assets including $17.3 million in cash and cash equivalents.

Total liabilities were $55.9 million, including $20 million outstanding on the Company’s revolving line of credit. Total shareholders’ equity was $41 million.

On May 10, 2022 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is biologically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune diseases, reported first quarter 2022 financial results and provided a business update (Press release, Rubius Therapeutics, MAY 10, 2022, View Source [SID1234614104]).

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"With the updated results showing single-agent activity and encouraging tolerability of monotherapy RTX-240 in patients whose disease progressed on PD-(L)1 inhibitors, we believe we have the opportunity to develop RTX-240 as a combination therapy with immune checkpoint inhibitors in earlier lines of therapy, where the greatest need exists for patients with NSCLC and RCC," said Pablo J. Cagnoni, M.D., president and chief executive officer. "We have expanded our Phase 1 arm of RTX-240 in combination with pembrolizumab to focus on less heavily pretreated patients with NSCLC and RCC. We plan to report initial clinical results from the combination arm in advanced solid tumors and initial data from the NSCLC and RCC patients in the second half of 2022."

Dr. Cagnoni continued, "We believe RTX-321 has shown promising pharmacodynamic effects with dramatic expansion of CD4+ T cells, which is one of the key cells involved in the mechanism by which IL-12 stimulates a broad anti-tumor response. Importantly, we continue to see no dose-limiting toxicities and no treatment-related adverse events, giving us confidence that we may be able to safely exploit IL-12’s potent antitumor activity with RTX-224, our second broad immune agonism program. RTX-224 expresses higher copy numbers of IL-12 on the cell surface than does RTX-321. Given the additional investment required to dose escalate and the eventual need for a companion diagnostic for patient selection for RTX-321, we are focusing our resources at this time on advancing our broad agonism approach with RTX-240 and RTX-224."

Recent Highlights

Broad Immune Stimulation

RTX-240

RTX-240 is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to simultaneously present hundreds of thousands of copies of the costimulatory molecule 4-1BB ligand (4-1BBL) and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in their native forms. RTX-240 is designed to broadly stimulate the immune system by activating and expanding both NK and CD8+ memory T cells to generate an anti-tumor response.

Monotherapy RTX-240 in Advanced Solid Tumors

Reported updated clinical data from the monotherapy Phase 1 arm of RTX-240 in relapsed/refractory or locally advanced solid tumors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022
Results included updated safety (n=34) and efficacy (n=27) data from 9 completed dose cohorts at the time of the March 4, 2022, data cutoff
There were three partial responses (PR) in NSCLC, anal cancer and uveal melanoma patients:
an unconfirmed PR (uPR) with 41% decrease of all target lesions and a notable decrease of an external protruding chest wall mass in a patient with NSCLC whose disease had progressed on prior anti-PD-L1 therapy;

a confirmed PR with a 54% reduction in the target lesions in a patient with metastatic anal cancer whose disease had progressed on anti-PD-L1 therapy; and

a uPR with 100% decrease of the target hepatic lesion and resolution of multiple non-target hepatic lesions in a patient with metastatic uveal melanoma whose disease had progressed on anti-PD-1 therapy

The uPR in NSCLC and 5 cases of stable disease (SD) were observed across the 3e10 cohorts, including 3 SDs in patients with metastatic NSCLC and 2 with RCC supporting the Company’s decision to expand the Phase 1 arm of RTX-240 plus pembrolizumab to NSCLC and RCC patients
One patient each with NSCLC and RCC remained on monotherapy treatment with SD greater than 6 months as of the cutoff date
All of these patients had experienced disease progression on prior anti-PD-(L)1 therapy
RTX-240 was shown to have been generally well tolerated with no treatment-related or investigator-identified immune-related Grade 3/4 adverse events (AE’s) and no dose-limiting toxicities.
Based on the totality of clinical, tolerability and pharmacodynamic data, a recommended monotherapy Phase 2 dose of 5e10 cells administered every 3 weeks was selected
This dose is being further explored in the combination expansion cohort of NSCLC and RCC patients
Enrollment continues in the monotherapy arm of the trial at the recommended Phase 2 dose of 5e10 cells administered every 3 weeks
RTX-240 + Pembrolizumab in Advanced Solid Tumors

Advanced enrollment in the Phase 1 combination arm of RTX-240 plus pembrolizumab in patients with advanced solid tumors
Expanded ongoing Phase 1 arm to enroll up to 20 patients each with NSCLC and RCC who are less heavily pretreated in preparation for a future Phase 2 clinical trial of RTX-240 in combination with pembrolizumab in an earlier line of therapy
RTX-224

RTX-224 is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to express hundreds of thousands of copies of 4-1BBL and IL-12 on the cell surface. In contrast to RTX-240, RTX-224 is designed as a broad immune agonist of both adaptive and innate responses, activating CD8+ and CD4+ T cells, promoting antigen presentation and activating and expanding NK cells. It is expected to produce a broad and potent anti-tumor T cell response, an innate immune response and have anti-tumor activity in those tumor types with known sensitivity to T cell killing, including tumor types with high mutational burden, PD-L1 expression and prior activity of checkpoint inhibitors.

Continuing dose escalation in the Phase 1/2 clinical trial of RTX-224 in selected relapsed/refractory or locally advanced solid tumors that include non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer
Initial clinical results are expected by year-end or during the first quarter of 2023
Antigen-Specific Immune Stimulation

RTX-321 Artificial Antigen-Presenting Cell (aAPC) Development Program for HPV 16-Positive Cancers

RTX-321 is an allogeneic, off-the-shelf aAPC therapy product candidate that is engineered to induce a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface and is designed to mimic human T cell-APC interactions.

Three dose cohorts were completed (n=9) with one patient with anal squamous cell carcinoma with SD ongoing at 16 weeks at the highest dose cohort to date of 1e10 administered every three weeks. Prior to enrollment, the patient experienced disease progression on anti-PD-1 therapy. RTX-321 was generally well tolerated with no DLTs or Grade 3/4 treatment-related AE’s. Consistent with the combined mechanism of action of IL-12 and 4-1BBL, increases in activated CD4+ T cells, activated CD8+ T cells and activated NK cells were observed at the higher dose levels.

Manufacturing

Scaled manufacturing to 200L bioreactors in support of a potential future pivotal trial for RTX-240 and potential commercialization
This scaleup represents 4 times more cells than what was produced using the 50L bioreactor
Anticipated 2022 Catalysts and Operational Objectives

To evaluate the full potential of RTX-240, Rubius’ other oncology programs and the RED PLATFORM, Rubius plans to execute several critical milestones within the next 12 months and has sufficient cash runway into the second half of 2023:

Report initial Phase 1 clinical results for RTX-240 in combination with pembrolizumab in advanced solid tumors and data from the initial enrolled NSCLC and RCC patients in the second half of 2022;
Select a clinical candidate for the first autoimmune program in type 1 diabetes during the second half of 2022; and
Report initial Phase 1 clinical results for RTX-224 for the treatment of advanced solid tumors by year-end or during the first quarter of 2023.
First Quarter 2022 Financial Results

Net loss for the first quarter of 2022 was $52.4 million or $0.58 per common share, compared to $42.3 million or $0.51 per common share in the first quarter of 2021.

In the first quarter of 2022, Rubius invested $38.3 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, compared to $27.7 million in the first quarter of 2021. This year-over-year increase was principally due to a $5.3 million increase in costs related to our lead cancer programs, RTX-240, RTX-321 and RTX-224, primarily from clinical research organization (CRO) and internal manufacturing costs incurred in connection with all three programs. Additionally, platform development, early-stage research and other unallocated expenses increased by $5.6 million due principally to increases of $2.4 million in personnel-related costs and $0.9 million in stock-based compensation related to the increase in headcount to support our expanded operations. Contract research and development and laboratory supplies also increased to support drug discovery and platform development activities.

G&A expenses were $12.6 million during the first quarter of 2022, compared to $13.2 million for the first quarter of 2021. The lower costs were primarily driven by a reduction in stock-based compensation related to stock option awards that fully vested during the third quarter of 2021.

Cash Position

As of March 31, 2022, cash, cash equivalents and investments were $176.5 million, compared to $225.8 million as of December 31, 2021, providing Rubius with a cash runway into the second half of 2023. During the quarter, the Company used $47.1 million of cash to fund operations and $2.4 million to fund capital expenditures, consisting mostly of renovation costs incurred at our manufacturing facility.

On May 10, 2022 EDAP TMS SA (Nasdaq: EDAP), the global leader in robotic energy-based therapies, reported that it will release its financial results for the first quarter ended March 31st, 2022 after the markets close on Tuesday, May 17th, 2022 (Press release, EDAP TMS, MAY 10, 2022, View Source [SID1234614121]).

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An accompanying conference call and webcast will be hosted by Marc Oczachowski, Chairman of the Board and Chief Executive Officer, Ryan Rhodes, CEO of EDAP U.S., and François Dietsch, Chief Financial Officer. The call will be held at 8:30am EDT on Wednesday, May 18th, 2022. Please refer to the information below for conference call dial-in information and webcast registration.

On May 10, 2022 Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, reported its financial results for the first quarter ended March 31, 2022, and recent business highlights (Press release, Achilles Therapeutics, MAY 10, 2022, View Source [SID1234614168]).

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"We recently dosed the first patient with our higher-dose cNeT therapy in the CHIRON clinical trial for the treatment of advanced non-small cell lung cancer (NSCLC) and began enrollment in Cohort B of the THETIS clinical trial evaluating cNeT in combination with a PD-1 checkpoint inhibitor for the treatment of metastatic malignant melanoma. We expect to report initial higher-dose monotherapy data from both CHIRON and THETIS as well as initial combination data from THETIS Cohort B in the second half of 2022," said Dr Iraj Ali, Chief Executive Officer of Achilles Therapeutics. "We also expanded our global footprint, including increased clinical manufacturing capacity in the United Kingdom and the United States, where we have established a US headquarters in Philadelphia that will house our first US R&D facility, to further support our clinical programs. Overall, our unique approach to targeting clonal neoantigens, differentiated ability to detect, quantify and track our cNeT products, and strong balance sheet continue to position us well to execute across our upcoming value-creating catalysts."

Business Highlights

Dosed the first patient with higher-dose (Process 2) cNeT monotherapy in the CHIRON clinical trial for advanced NSCLC.
Following a positive Independent Data Safety Monitoring Committee review, initiated enrollment of THETIS Cohort B to evaluate cNeT therapy in combination with a PD-1 checkpoint inhibitor for the treatment of metastatic malignant melanoma.
Strengthened the Board of Directors with the addition of independent member Bernhard Ehmer, MD.
Expanded global manufacturing by increasing capacity in the United Kingdom with a GMP license obtained for the Cell & Gene Therapy Catapult facility, and entered into a partnership agreement for clinical manufacturing in the United States with the Center for Breakthrough Medicines, a contract development and manufacturing organization in King of Prussia, Pennsylvania.
Hosted a key opinion leader webcast highlighting important data selected from the 31 posters and presentations on the TRACERx study presented at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
Financial Highlights

Cash and cash equivalents: Cash and cash equivalents were $236.9 million as of March 31, 2022, as compared to $266.3 million as of December 31, 2021. The Company anticipates that its cash and cash equivalents are sufficient to fund its planned operations into the second half of 2024, including full funding of the ongoing Phase I/IIa CHIRON and THETIS clinical trials.
Research and development (R&D) expenses: R&D expenses were $13.0 million for the first quarter ended March 31, 2022, as compared to $8.9 million for the first quarter ended March 31, 2021. The increase was primarily driven by increased activity related to our ongoing clinical trials and overall R&D.
General and administrative (G&A) expenses: G&A expenses were $6.0 million for the first quarter ended March 31, 2022, as compared to $4.8 million for the first quarter ended March 31, 2021. The increase was primarily driven by fees associated with the Company’s public company obligations, and an increase in headcount and related personnel costs.
Net loss: Net loss for the first quarter ended March 31, 2022, was $17.4 million or $0.45 per share compared to $13.8 million, or $8.38 per share for the first quarter ended March 31, 2021.
2022 Milestones and Upcoming Events

Higher-dose Monotherapy: Report initial data from the higher-dose cohort of patients undergoing cNeT monotherapy for the treatment of NSCLC and melanoma in the second half of 2022.
cNeT Combination: Dose first melanoma patient with cNeT in combination with a PD-1 checkpoint inhibitor in Cohort B of the THETIS clinical trial and report initial data in the second half of 2022.
Manufacturing: Begin clinical cNeT production at the Cell & Gene Therapy Catapult facility in the second half of 2022.
Tumor Archiving Program: Initiate program in the second quarter of 2022.
Achilles will present at the following medical and investor conferences in May 2022. Additional details will be available in the Events & Presentations section of the Company’s website:

BofA Securities 2022 Healthcare Conference: May 9-13, 2022
H.C. Wainwright Global Investment Conference: May 23-26, 2022

On May 10, 2022 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of injectable biologics to selectively engage and modulate targeted T cells directly within the patient’s body, reported first quarter 2022 financial results (Press release, Cue Biopharma, MAY 10, 2022, View Source [SID1234614231]). The Company will not host a business update call in conjunction with its financial results press release.

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Recent Business Updates

Extended cash runway with an aggregate of $23.6 million from the sale of 3,593,407 shares of our common stock pursuant to our at-the-market (ATM) equity offering sales agreement with Jefferies LLC. As of March 31, 2022, the Company sold 2,396,013 shares of common stock under the October 2021 ATM Agreement for proceeds of approximately $17.6 million net of commissions paid, excluding transaction expenses, and in April 2022 sold an additional 1,197,394 shares of common stock for proceeds of approximately $6.0 million, net of commissions paid, excluding transaction expenses.
Completed enrollment of 20 patients at the recommended Phase 2 dose of 4mg/kg of the Phase 1b monotherapy trial of CUE-101 for the treatment of HPV16-driven recurrent/metastatic head and neck squamous cell carcinoma (R/M HNSCC) in second line and beyond treatment-refractory patients. To date, CUE-101 has demonstrated a favorable tolerability profile as well as encouraging anti-tumor clinical activity and what appears to be an emerging trend of enhanced overall survival (OS).
Completed dose escalation phase of CUE-101 in combination with Merck’s KEYTRUDA, an anti-PD-1 biologic agent, as first-line therapy in patients with advanced HPV16+ HNSCC. Patient enrollment has begun in the expansion phase at the recommended Phase 2 dose of 4mg/kg of CUE-101.
"Through a focused and strategic deployment of resources during the first quarter of 2022, we have made significant progress advancing the IL-2-based CUE-100 series for oncology, as well as strengthening our financial position," said Daniel Passeri, chief executive officer of Cue Biopharma. "As reported, we recently completed the patient expansion phase of the monotherapy Phase 1b clinical trial of CUE-101 and the dose escalation phase of the CUE-101 Phase 1 combination trial with KEYTRUDA. We are very pleased with our clinical trial progress to date and have enhanced confidence in the emerging data supporting the potential of our CUE-100 series pipeline in immuno-oncology through targeted and selective stimulation of the patient’s immune system against cancer."

Kerri-Ann Millar, chief financial officer of Cue Biopharma, added, "We continue to be in a solid financial position as we remain disciplined in our resource deployment and adroit in our response to the challenges of the current biotech capital markets. Accessing our ATM common stock facility during the first four months of 2022 allowed us to extend the anticipated operational runway further into the third quarter of 2023 which we anticipate will enable us to assess the data readouts from both our Phase 1 monotherapy and combination clinical trials of CUE-101."

First-Quarter 2022 Financial Results
The Company reported collaboration revenue of approximately $1.0 million and $1.6 million for the three months ended March 31, 2022 and 2021, respectively.

Research and development expenses were $10.1 million and $9.8 million for the three months ended March 31, 2022 and 2021, respectively. The increase in research and development expenses of $0.3 million was primarily due to an increase in laboratory and drug substance manufacturing costs, employee and scientific and clinical advisory board compensation, other professional fees, licensing fees, and rent.

General and administrative expenses were $5.2 million and $4.3 million for the three months ended March 31, 2022 and 2021, respectively. The increase in general and administrative expense of $0.9 million was primarily due to an increase in stock-based compensation expense, professional and consulting fees, and employee and board compensation incurred in the first quarter of 2022 as compared to the same period in 2021.