On May 10, 2021 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company engineering a novel class of injectable biologics designed to selectively engage and modulate targeted T cells within the patient’s body, reported that interim clinical data, including a confirmed partial response (PR), from its ongoing Phase 1a/1b monotherapy study of CUE-101 as second-line treatment for patients with HPV+ R/M HNSCC (Press release, Cue Biopharma, MAY 10, 2021, View Source [SID1234608280]). To date, CUE-101 has demonstrated a favorable tolerability profile in a monotherapy dose escalation trial and continues to generate encouraging emerging data pertaining to its pharmacokinetic (PK) and pharmacodynamic (PD) profiles, as well as clinical anti-tumor activity.

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"We are very excited by the partial response observed in this patient, since it demonstrates single-agent activity of CUE-101, so rarely seen in mono-immunotherapy treatments," said Ken Pienta, M.D., acting chief medical officer of Cue Biopharma. "Based on recent clinical observations, we have delivered CUE-101 to patients at tolerated doses, achieving a confirmed PR as well as stable disease (SD) in several patients while still in the dose escalation portion of the trial. This provides evidence that CUE-101 is an active agent with promising potential for HPV+ HNSCC patients. This is bolstered by the supporting pharmacodynamic data in which we have observed activation of disease-specific T cells and NK cells in the blood of our treated patients. We look forward to initiating the expansion phase of the Phase 1 trial which may provide a potential path forward for a registration-directed clinical trial for CUE-101 as a monotherapy for HPV+ R/M HNSCC."

Key interim analysis of patient data from the ongoing open-label Phase 1a/1b study include:

Confirmed PR in one patient and SD in five patients, all confirmed by RECIST criteria, in the dose escalation phase of the study to date, providing early signs of potential single-agent activity of CUE-101.
Demonstrated evidence of both tumor-specific CD8+ T cell expansion as well as dose-dependent increases in NK cells in patients.
Observed immune cell infiltration and tumor cell necrosis in patient tumor biopsies after CUE-101 treatment, supporting CUE-101’s mechanism of action at engaging and modulating targeted T cells within the patient’s body.
Dose-proportional PK profile and comparable drug exposure levels in patients receiving repeated dosing cycles, consistent with a lack of drug-clearing anti-drug antibodies.
No maximum tolerated dose (MTD) observed in patients dosed with up to 8 mg/kg of CUE-101.
Dan Passeri, chief executive officer of Cue Biopharma added, "Receiving early confirmation of clinical activity of CUE-101 as a monotherapy in this challenging patient population is an important validation of the potential of the CUE-101 program for oncology, as well as for our follow-on programs such as CUE-102, from the IL2-based CUE-100 series. This confirmed PR provides valuable proof of concept of the Immuno-STAT platform to activate and expand disease specific T cells and NK cells as a method to treat other cancers. We anticipate a number of important milestones throughout this year with the potential of further data to inform CUE-101’s clinical application. These milestones include, the potential selection of a recommended Phase 2 dose of CUE-101 by mid-2021 for further development as a single-agent treatment for HPV+ 2L+ head and neck squamous cell carcinoma; our plans to report initial Phase 1 results from the combination study of CUE-101 with pembrolizumab in the second half of 2021; and our plans to initiate a Phase 2 neoadjuvant study to evaluate the effects of CUE-101 on the tumor microenvironment, which is also expected to start in the second half of 2021."

Conference Call
Cue Biopharma will discuss these new data points during its first quarter business update call and webcast on May 17 at 4:30 p.m. ET. Please check the Events page of the Investor and Media section of the Company’s website at www.cuebiopharma.com for details. A live and archived version of the webcast will be available for 30 days after the call.

About the CUE-101 Clinical Trial
The trial (NCT03978689) is a multi-center, first-in-human, open-label Phase 1 dose escalation and expansion study evaluating the safety, anti-tumor effect and immunogenicity of CUE-101 as a monotherapy in second-line patients with confirmed HPV16-driven recurrent/metastatic HNSCC and HLA-A*02:01 serotype. Based on translational data from the trial, a maximum tolerated dose (MTD) or recommended Phase 2 dose will be determined. The company has expanded the study to evaluate CUE-101 in combination with KEYTRUDA (pembrolizumab) as first-line treatment in patients with HPV16-driven recurrent/metastatic HNSCC.

About the CUE-100 Series
The CUE-100 series consists of Fc-fusion biologics that incorporate peptide-MHC (pMHC) molecules along with rationally engineered IL-2 molecules. This singular biologic is anticipated to selectively target, activate and expand a robust repertoire of tumor-specific T cells directly in the patient. The binding affinity of IL-2 for its receptor has been deliberately attenuated to achieve preferential selective activation of tumor-specific effector T cells while reducing the potential for effects on regulatory T cells (Tregs) or broad systemic activation, potentially mitigating the dose-limiting toxicities associated with current IL-2-based therapies.

About Immuno-STAT
The company’s Immuno-STAT (Selective Targeting and Alteration of T cells) biologics are designed for targeted modulation of disease-associated T cells in the areas of immuno-oncology and autoimmune disease. Each of our biologic drugs is designed using our proprietary scaffold comprising: 1) a pMHC to provide selectivity through interaction with the T cell receptor (TCR), and 2) a unique co-stimulatory signaling molecule to modulate the activity of the target T cells.

The simultaneous engagement of co-regulatory molecules and pMHC binding mimics the signals delivered by antigen presenting cells (APCs) to T cells during a natural immune response. This design enables Immuno-STAT biologics to engage with the T cell population of interest, resulting in selective T cell modulation. Because our drug candidates are delivered directly in the patient’s body (in vivo), they are fundamentally different from other T cell therapeutic approaches that require the patients’ T cells to be extracted, modified outside the body (ex vivo), and reinfused.

On May 10, 2021 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a biotech innovator and biologics contract manufacturing organization, reported that it will report its fiscal 2021 third quarter financial results before market open on Monday, May 17, 2021 (Press release, iBioPharma, MAY 10, 2021, View Source [SID1234585483]). iBio management will host a webcast and conference call at 8:00 a.m. Eastern Time to discuss the results and provide a corporate update.

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The live and archived webcast may be accessed on the Company’s website at www.ibioinc.com under "News and Events" in the Investors section. The live call can be accessed by dialing (833) 672-0651 (domestic) or (929) 517-0227 (international) and referencing conference code: 3085726.

On May 10, 2021 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (NASDAQ: EGRX) reported that TREAKISYM RI (50ml) liquid formulation has been filed with the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan (Press release, Eagle Pharmaceuticals, MAY 10, 2021, View Source [SID1234584740]).

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The application is based on the results of clinical studies investigating the safety and pharmacokinetics of TREAKISYMRTD administered by 10-minute intravenous infusion.

"We are pleased that the RI application has been submitted ahead of schedule, which will enable patients and providers alike to reap the benefits of this formulation. We believe we can get close to peak income of $20-$25 million as early as next year from the RTD and RI products. This is an important extension of the bendamustine franchise, and we value the relationship with Symbio," stated Scott Tarriff, Chief Executive Officer.

In September 2017, Eagle licensed to SymBio intellectual property necessary to develop, market and sell RTD and RI formulations of bendamustine under the trade name TREAKISYM in Japan utilizing Eagle’s proprietary technology. As part of the agreement, SymBio assumed responsibility for securing regulatory approval of the TREAKISYM RTD and RI products using the licensed technology in Japan.

TREAKISYM RI has the advantage of reducing infusion time to 10 minutes (from the current 60 minutes), eliminating the need for manual reconstitution and significantly reducing preparation time, benefitting both patients and healthcare providers.

Heron Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On May 10, 2021 Celcuity Inc. (NASDAQ:CELC), a clinical-stage biotechnology company pursuing an integrated companion diagnostic (CDx) and therapeutic strategy for treating patients with cancer, reported financial results for the first quarter ended March 31, 2021 and summarized recent business progress (Press release, Celcuity, MAY 10, 2021, View Source [SID1234580661]).

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"Celcuity took a transformational strategic step in April when we entered into a global licensing agreement with Pfizer to obtain exclusive rights to develop and commercialize gedatolisib, a pan-PI3K/mTOR inhibitor, in clinical development to treat patients with ER+/HER2-negative advanced or metastatic breast cancer," said Brian Sullivan, CEO and co-founder of Celcuity. "Celcuity is planning 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 the first half of 2022. We have a highly experienced drug development team and the financial resources in place to advance the gedatolisib program and are excited by the opportunity to utilize our CELsignia cellular analysis platform to support the development of a potential first-in-class targeted cancer therapy like gedatolisib."

First Quarter 2021 Business Highlights and Other Recent Developments

In January, Celcuity entered a collaboration with Sarah Cannon Research Institute and Pfizer Inc. to conduct an open-label Phase 2 clinical trial. This trial will evaluate the efficacy and safety of two Pfizer targeted therapies, VIZIMPRO, a pan-HER inhibitor, and XALKORI, a c-Met inhibitor, in patients with previously treated metastatic HER2-negative breast cancer selected with Celcuity’s CELsignia Multi-Pathway Activity Test. Celcuity believes there is significant clinical interest in finding new diagnostic tests and targeted therapies for patients with metastatic HER2-negative breast cancer whose disease progressed on prior therapies. Patient enrollment is expected to begin in the second or third quarter of 2021 with interim results in the second half of 2022.
Celcuity raised approximately $43.0 million of gross proceeds from financings in the first quarter of 2021 and April 2021.
In late February, Celcuity completed a successful follow-on public offering that raised gross proceeds of approximately $27.6 million.
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. Celcuity will be able to draw on two additional tranches of $5.0 million each upon the achievement of certain clinical trial and financing milestones.
In March, Celcuity entered into a clinical trial collaboration with MD Anderson, Novartis, and Puma Biotechnology to evaluate the efficacy and safety of Novartis’ targeted therapy TABRECTA and Puma’s NERLYNX in patients with metastatic HER2-negative breast cancer selected by Celcuity’s CELsignia Multi-Pathway Activity Test. This is Celcuity’s second clinical trial to treat patients diagnosed with hyperactive HER2 and c-Met signaling breast cancers with matching targeted therapies and Celcuity now has five clinical trial collaborations in place.
In April, Celcuity entered a worldwide licensing agreement with Pfizer for the exclusive right to develop and commercialize gedatolisib. Gedatolisib is in Phase 1b clinical development for the treatment of patients with ER+/HER2-negative advanced or metastatic breast cancer. Celcuity announced preliminary data for the 103 patients enrolled in the expansion portion of the ongoing Phase 1b clinical trial evaluating gedatolisib, plus Ibrance and endocrine therapy. As of the January 11, 2021 data cut-off, 53 of the 88 evaluable patients (60%) had an objective response. Gedatolisib was also generally well tolerated, with the majority of treatment-related adverse events (TRAE) being Grade 1 or 2. The most common Grade 3 or 4 TRAEs related to gedatolisib were stomatitis and rash. Celcuity plans to meet with the FDA later this year to discuss its clinical development plans for gedatolisib.
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. Gedatolisib at one-fifth the concentration of inavolisib (30 nM vs. 150 nM), inhibited five times more signaling activity as quantified by the CELsignia test. Data also showed that synergistic cooperation between PI3K/mTOR and BCL signaling was detected, suggesting potential patient benefit of combining gedatolisib with a BCL inhibitor.
First Quarter 2021 Financial Results
Unless otherwise stated, all comparisons are for the first quarter ended March 31, 2021, compared to the first quarter ended March 31, 2020.

Total operating expenses were $2.79 million for the first quarter of 2021, compared to $2.31 million for the first quarter of 2020.

Research and development (R&D) expenses were $2.24 million for the first quarter of 2021, compared to $1.85 million for the first quarter of 2020. The approximately $0.39 million increase during the first three months of fiscal year 2021, compared to the first three months of fiscal year 2020, resulted from a $0.06 million increase in compensation related expenses, which included a decrease of approximately $0.04 million of non-cash stock-based compensation expense. In addition, other research and development expenses increased $0.33 million due to clinical validation and laboratory studies, and operational and business development activities.

General and administrative (G&A) expenses were $0.56 million for the first quarter of 2021, compared to $0.46 million for the first quarter of 2020. The approximately $0.09 million increase during the first three months of fiscal year 2021, compared to the first three months of fiscal year 2020, resulted primarily from a $0.08 million increase in professional fees associated with being a public company and director and officer insurance.

Net loss for the first quarter of 2021 was $2.79 million, or $0.25 per share, compared to a net loss of $2.25 million, or $0.22 per share, for the first quarter of 2020. Non-GAAP adjusted net loss for the first quarter of 2021 was $2.34 million, or $0.21 per share, compared to non-GAAP adjusted net loss of $1.78 million, or $0.17 per share, for the first quarter of 2020. Non-GAAP adjusted net loss excludes stock-based compensation expense. Because this item has 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 first quarter of 2021 was $2.52 million, compared to $1.83 million for the first quarter of 2020.

At March 31, 2021, Celcuity had cash and cash equivalents of $34.9 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 $14.5 million of net proceeds from a debt financing agreement. Taking into account these two events subsequent to the end of the first quarter, Celcuity has approximately $44.0 million of cash-on-hand.

Anticipated Milestones
Celcuity expects to do the following over the next twelve months:

Announce additional clinical trial collaborations in the first half of 2021 utilizing the CELsignia platform.
Initiate Phase 2/3 clinical trial for gedatolisib in breast cancer in the first half of 2022 pending discussions with the FDA regarding the clinical development pathway.
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 first quarter financial results and provide a corporate update. To participate in the teleconference, domestic callers should dial 1-877-407-8035 and international callers should dial 201-689-8035. 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.