On May 11, 2021 Immatics N.V. (NASDAQ: IMTX, "Immatics"), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, reported data from its second T cell receptor (TCR) Bispecifics program, IMA402, supporting preclinical proof-of-concept for the program and further validating this proprietary therapeutic modality (Press release, Immatics, MAY 11, 2021, View Source [SID1234579679]). IMA402 is directed against the cancer target PRAME, a protein that is frequently expressed in many solid cancers, thereby supporting the program’s potential to address a broad cancer patient population. IMA402 is the second program originating from Immatics’ TCR Bispecifics pipeline, called TCER (T Cell Engaging Receptor). The lead candidate showed anti-tumor activity against PRAME-positive cancer cells leading to consistent reduction of the engrafted tumors, including complete responses in an in vivo mouse model. The preclinical data will be presented at the virtual 17th Annual PEGS Boston Protein Engineering and Cell Therapy Summit, on May 11-13, 2021.

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Preclinical data highlights:

The IMA402 TCER candidate targets an HLA-A*02-bound peptide derived from preferentially expressed antigen in melanoma (PRAME).
The target peptide was selected and validated based on quantitative mass spectrometry data from Immatics’ proprietary XPRESIDENT platform and is prevalent in many solid tumor indications including lung, ovarian and breast cancer as well as other solid cancer types.
Over 50 different human wild-type TCRs recognizing the PRAME target peptide were systematically evaluated using Immatics’ XCEPTOR platform. Two TCRs with high avidity and specificity were selected and affinity-enhanced by at least 1,000-fold while retaining specificity through the XPRESIDENT-guided screening for off-target toxicity and cross-reactivity. Different engineered TCR variants were then incorporated into the bispecific TCER scaffold and the best candidate was selected.
The IMA402 TCER candidate induces killing of tumor cells in vitro with PRAME target peptide levels similar to levels found in cancer patients.
Administration of IMA402 TCER candidate leads to consistent tumor regression including complete responses in an in vivo mouse model.
The IMA402 TCER candidate demonstrates selective PRAME recognition leading to an at least 1,000-fold therapeutic window between tumor and normal cell reactivity in vitro.
Preclinical data support antibody-like profiles for manufacturability and pharmacokinetics of the IMA402 TCER candidate.

Carsten Reinhardt, M.D., Ph.D., Chief Development Officer at Immatics commented: "Having generated a strong preclinical proof-of-concept data package for our second TCR Bispecifics program is a significant milestone for Immatics. Together with our Adoptive Cell Therapy (ACT) program IMA203, which also targets PRAME, we are attacking this ubiquitous cancer cell protein from two different angles using our distinct therapeutic modalities. Based on the demonstrated preclinical data supporting significant single-agent activity of both of our TCER programs against established tumors, we are looking forward to advancing our TCER candidates, IMA401 and IMA402, into the clinic with the aim to treat cancer patients who have an urgent need for new treatment options."

For the IMA402 TCER program, Immatics has initiated GMP process development activities to advance this program towards the Investigational New Drug (IND) stage and clinical development. The company’s first TCER program, IMA401 remains on track for submission of a clinical trial application (CTA) by year end 2021. The company had previously announced preclinical proof-of-concept data for IMA401 in last quarter of 2020.

The full presentation of preclinical data from the IMA402 program is available on Immatics’ website using this link.

About TCER
Immatics’ TCER molecules are antibody-like "off-the-shelf" biologics that leverage the body’s immune system by redirecting and activating T cells towards cancer cells expressing a specific tumor target. To do so, the proprietary biologics are engineered to have two binding regions. The first region contains an affinity- and stability-improved TCR that binds specifically to the cancer target on the cell surface presented by a human leukocyte antigen (HLA) molecule. The second region is derived from an antibody domain that recruits endogenous T cells to the tumor to become activated. The design of the TCER molecules enables the activation of any T cell in the body to attack the tumor, regardless of the T cells’ intrinsic specificity. In addition, the TCER molecule has a Fc-part conferring stability, half-life extension and enhanced manufacturability.

On May 11, 2021 VBL Therapeutics (Nasdaq: VBLT) reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, VBL Therapeutics, MAY 11, 2021, View Source [SID1234579678]).

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"Our OVAL Phase 3 clinical trial remains on track and passed its most recent safety review, with the next one coming in the third quarter," said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. "During the first quarter and in early April, we strengthened our balance sheet, raising a total of approximately $40 million in additional capital, $12.3 million primarily through the exercise of outstanding warrants and gross proceeds of $28.3 million through a public financing. The additional capital extends our cash runway until year-end 2023. We have a number of important milestones anticipated through the rest of 2021 and look forward to keeping investors apprised of our progress."

First Quarter and Recent Corporate Highlights

In February, a successful pre-planned DSMC review of the OVAL study found no safety issues with the trial and recommended its continuation as planned.
In March, the Company announced the initiation of randomized, controlled and blinded trial of VB-111 in patients with recurrent Glioblastoma Multiforme (rGBM).
In March, the Company announced the publication of positive results in peer-reviewed journal Gynecologic Oncology of the pre-specified interim analysis of the OVAL Phase 3 Registration Enabling Study of VB-111 in Ovarian Cancer. The analysis showed a CA-125 response of at least 58% in the VB-111 treatment arm.
Development of VB-111 in rGBM and colorectal cancer, as well as of pipeline assets, continues as planned.
Corporate

In January, the Company entered into an ordinary share purchase agreement of up to $20 million with Aspire Capital Fund LLC
During the first quarter, the Company raised an additional $12.3 million through a combination of sales under the Aspire share purchase agreement, sales through its At-the-Market (ATM) facility and the exercise of outstanding warrants
In April, the Company closed a public offering raising gross proceeds of $28.3 million
Financial Results for the First Quarter 2021

As of March 31, 2021, VBL had cash, cash equivalents, short-term bank deposits and restricted bank deposits totaling $36.6 million. After the end of the first quarter, VBL raised gross proceeds of $28.3 million in a public offering of shares and pre-funded warrants. VBL expects that its cash and cash equivalents and short-term bank deposits will be sufficient to fund operating expenses and capital expenditure requirements until year-end 2023.
Revenues for the first quarter 2021 were $185 thousand, as compared to $366 thousand in the comparable period in 2020.
R&D expenses, net were $4.8 million for the first quarter compared to $4.5 million in the comparable period in 2020.
G&A expenses were $1.7 million for the fiscal year, compared to $1.3 million in the comparable period in 2020.
VBL reported a net loss for the quarter ended March 31, 2021, of $6.3 million, or ($0.12) per basic share, compared to a net loss of $5.4 million, or ($0.15) per basic share, in the comparable period in 2020.
Conference Call:

The live webcast will be available online and may be accessed from the "Events and Presentation" page of the company website. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.

On May 11, 2021 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, reported that provided a company update and first quarter 2021 financial results (Press release, Cumberland Pharmaceuticals, MAY 11, 2021, View Source [SID1234579677]). Net revenues from continuing operations during the quarter were $10.5 million, a 26.5% increase over the prior year period. The company also recorded an additional $500,000 in revenue during the first quarter associated with divested product rights for two brands it is no longer distributing.

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The Company’s financial position included $93 million in total assets – with $25 million in cash, $46 million of total liabilities, and $47 million of shareholders’ equity at the end of the quarter.

"As the novel coronavirus continues to affect lives over a year after its arrival in the U.S., we remain hopeful, as an overall reopening of our country is now in progress," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "Given all the challenges of operating a business in the wake of a pandemic, Cumberland was able to generate solid financial performance by adjusting our market strategies, retooling our sales communications and reinventing the way in which we operate our business."

RECENT COMPANY DEVELOPMENTS:

Vibativ Case Studies

Cumberland’s Vibativ product has been used across the country to help COVID-19 patients who develop secondary bacterial infections in their lungs. Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia that can result from COVID-19, flu, and other infections.

The Company subsequently compiled a dossier of patient case studies from across the country outlining several real-world instances where Vibativ effectively and safely treated the hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia resulting from MSSA and MRSA infections that developed in patients hospitalized with COVID-19.

RediTrex Launch

The Company is now finalizing plans for the full launch of its FDA-approved RediTrex product line. RediTrex is approved for patients with severe, active rheumatoid arthritis and polyarticular juvenile idiopathic arthritis who have difficulty tolerating or responding to orally delivered methotrexate. It is also approved for symptomatic control of severe, recalcitrant, disabling psoriasis in adults who are not adequately responsive to other forms of therapy.

Cumberland initially implemented a soft launch during the fourth quarter of 2020 and is currently planning a national launch of the product line during the second half of 2021, once additional product supplies are received and market conditions return to normal.

Hyponatremia Publication in Support of Vaprisol

A recent study publication called Health Outcome Predictive Evaluation (HOPE) COVID-19 Registry Analysis, an international study of over 4,000 patients, found that patients hospitalized with COVID-19 had a high risk of developing hyponatremia. These COVID-19 patients also had a higher incidence of mortality due to their hyponatremia. The study results support the use of an intravenous vaptan to treat hyponatremia in critically ill patients afflicted with COVID-19.

Hyponatremia, an imbalance of serum sodium to body water, is the most common electrolyte disorder among hospitalized patients. Cumberland’s Vaprisol product is one of two branded prescription products indicated for the treatment of hyponatremia, and the only intravenously administered branded treatment. Vaprisol has a proven day-1 response to help normalize serum sodium levels in hyponatremic patients and move them out of the ICU as efficiently as possible.

FINANCIAL RESULTS:

Net Revenues: For the three months ended March 31, 2021, net revenues from ongoing operations were $10.5 million, up 26.5% from $8.3 million for the prior year period. The company also recorded an additional $500,000 in revenue during the first quarter associated with divested product rights.

Net revenue by product for the three months ended March 31, 2021, included $5.1 million for Vibativ, $3.0 million for Kristalose, $1.5 million for Caldolor, and $1.1 million for Vaprisol.

Operating Expenses: Total operating expenses for the three months ended March 31, 2021, were $10.9 million, compared to $10.2 million during the prior year period. This increase in expenses resulted primarily from the additional cost of goods associated with our growth in Vibativ sales during the quarter.

Earnings: Net income for the first quarter 2021 was $0.2 million or $0.01 a share, compared to a loss of $1.1 million or $0.07 a share for the prior year period.

Adjusted Earnings for the first quarter were $1.1 million or $0.07 per diluted share, compared to a loss of $0.4 million or $0.03 per diluted share for the prior year period.

Balance Sheet: At March 31, 2021, Cumberland had $93.3 million in total assets including $24.9 million in cash and cash equivalents. Total liabilities were $46.5 million, including $15.0 million outstanding on the Company’s revolving line of credit, resulting in total shareholders’ equity of $47.0 million.

Conference Call and Webcast

A conference call and live internet webcast will be held on Tuesday, May 11, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please 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 7973325. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On May 11, 2021 Anticancer Bioscience (ACB), pioneers in synthetic lethal approaches to precision oncology, reported that progress in its MYC-synthetic lethal (MYC-SL) program (Press release, Anticancer Bioscience, MAY 11, 2021, View Source [SID1234579676]). The Company has identified three classes of novel small molecule compounds that demonstrate robust anti-cancer activity in preclinical studies, acting through interfering with mitosis and blocking cytokinesis. ACB is progressing through optimization and final candidate selection with the goal of progressing at least one of these MYC-SL compounds into clinical trials by 2022.

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The MYC family oncogene, encoding a transcription factor Myc, is deregulated in over 50% of human cancers, and this deregulation is frequently associated with poor prognosis and unfavorable patient survival. Myc has a central role in almost every aspect of the oncogenic process, orchestrating proliferation, apoptosis, differentiation, and metabolism. Direct targeting of Myc has been a challenge for decades owing to its "undruggable" protein structure, with a lack of a drug binding pocket. The synthetic lethality associated with Myc overexpression is being explored for its potential in the development of novel anti-cancer therapies.

Commenting on the progress and pre-clinical data, Dun Yang PhD, Founder, President, and CEO of ACB said:

"We are excited by our early in vitro and in vivo pre-clinical studies and in our molecular biology work to identify the target that our novel compounds hit. In animal models, data consistent with the in vitro studies has confirmed that the MYC-SL compounds act through interfering mitosis and blocking cytokinesis. We have filed patents on our compounds and are progressing rapidly through optimization to candidate selection and IND enabling studies."

The novel target for ACB’s MYC-SL compounds has been identified and will be subject to peer review publication in due course.

ACB has developed an innovative and proprietary general utility new scaffold-drug fragment (GUNS-DF) library approach to small molecule drug discovery. It is constructing 10 types of pilot GUNS-DF libraries, each of which contains a distinct core scaffold. Twenty compounds with excellent drug-like properties have been identified from three types of GUNS-DF libraries with a potency of less than 20 nM in a cell-based assay for MYC-SL agents. This potency was achieved after synthesizing and screening approximately 350 analogs during lead optimization.

At low nM concentrations, these compounds elicited potent cytotoxicity in a panel of 50 cell lines representing a variety of human malignancies. With adequate bioavailability, many of these compounds also suppressed the growth of various cancer cell lines that grew as xenografts in immunocompromised mice.

Having raised CNY131m (~USD21m) in seed finance to fund its discovery research, ACB is currently pursuing a Series A financing to progress at least one of its MYC-SL compounds into clinical trials.

ACB will be showcasing its innovative drug discovery platforms and pipeline at ChinaBio and BioEquity in May, and at BIO in June.

On May 11, 2021 Ipsen (Euronext: IPN; ADR: IPSEY) reported it has exercised its option to collaborate with Exelixis, Inc. (Exelixis) in the pivotal COSMIC-311 Phase III trial (Press release, Ipsen, MAY 11, 2021, View Source [SID1234579675]). COSMIC-311 is evaluating Cabometyx (cabozantinib) 60 mg versus placebo in people living with radioiodine-refractory differentiated thyroid cancer (DTC) who have progressed after up to two prior vascular endothelial growth factor receptor (VEGFR)-targeted therapies.2 Radioactive iodine (RAI) is a treatment option for DTC when patients are at high risk of disease recurrence, have incompletely resected cancer, or distant metastases.3 Patients who develop RAI-refractory DTC, whereby they are resistant to RAI treatment, typically have a poor prognosis with an estimated survival of three to five years on average.4

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Howard Mayer, Executive Vice President and Head of Research and Development at Ipsen, commented:
"A planned interim analysis of the COSMIC-311 Phase III trial has shown promising and clinically meaningful results in the use of Cabometyx in people living with radioiodine-refractory differentiated thyroid cancer who have progressed after prior therapy. We are delighted to build on our strong foundation and join Exelixis to further evaluate, and to work with regulatory authorities on, the potential of Cabometyx in a patient population who currently have limited treatment options."

Results from the planned Phase III interim analysis of COSMIC-311 showed that the trial met the co-primary endpoint of demonstrating significant improvement in progression-free survival.1 The detailed results from the analysis will be presented at the forthcoming ASCO (Free ASCO Whitepaper) Annual Meeting, taking place virtually from 4 to 8 June 2021.

Ipsen has an exclusive collaboration agreement with Exelixis for the commercialization of Cabometyx outside of the U.S. and Japan. Following the decision to opt-in to the COSMIC-311 trial, Ipsen gains access to the results to support potential future regulatory submissions in its territories.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for Cabometyx in February 2021 as a potential treatment for people living with DTC who have progressed following prior therapy and who are RAI-refractory (if RAI is appropriate).