On May 17, 2021 Athenex, Inc. (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, led by its Orascovery platform, reported that investigators from Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of Medicine presented new clinical data from the ongoing GINAKIT2 phase 1 study of Athenex’s cell therapy candidate KUR-501 targeting GD2 in neuroblastoma at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 24th Annual Meeting on Friday, May 14, at 11 am ET (Press release, Athenex, MAY 17, 2021, View Source [SID1234580111]).

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Observed responses to date in 11 evaluable, heavily pretreated patients with neuroblastoma, include one complete response (CR) and one partial response (PR). Four additional patients have exhibited stable disease (SD). The durations of response for the CR and PR were approximately 5 and 3 months, respectively. No patients experienced grade 2 or higher toxicities related to the GD2-CAR NKTs. Post-treatment tumor biopsies showed GD2-CAR NKTs homing to metastatic lesions at all dose levels. The CAR-NKT AUC normalized to disease burden (AUC/Curie score using MIBG scan quantified tumor burden) appears to be associated with response to therapy.

"The results of the ongoing phase I study further validate the clinical activity of CAR-NKT cells," said Dr. Kurt Gunter, Chief Medical Officer of Athenex Cell Therapy. "The data thus far demonstrate clinical responses and tumor homing with an attractive safety profile. We are grateful to the patients and families for their participation in these studies and to the scientists and physicians at Texas Children’s Cancer Center and Baylor College of Medicine for their scientific leadership."

Dr. Andras Heczey, from Baylor College of Medicine, presented updated data from the GINAKIT2 study of autologous CAR-NKT cells. The title of the presentation is "Natural Killer T Cells Expressing a GD2-CAR and IL-15 Are Safe and Can Induce Complete Remission in Children with Relapsed Neuroblastoma – A First-in-Human, Phase 1 Trial". Dr. Heczey is the principal investigator for this study. The abstract and his work were selected for presentation at this year’s Clinical Trials Spotlight Symposium.

"We are encouraged by the reassuring safety profile, expansion/persistence/tumor trafficking parameters and by the evidence of clinical activity with autologous, engineered CAR-NKT cell therapy at relatively low doses," said Dr. Heczey, Associate Professor of Pediatrics – Oncology at Baylor College of Medicine and member of the Dan L Duncan Comprehensive Cancer Center. "We look forward to advancing the program into additional dose levels and treating additional patients."

About KUR-501

KUR-501 is an autologous product in which NKT cells are engineered with a CAR targeting GD2, which is expressed on almost all neuroblastoma tumors, as well as other malignancies. KUR-501 is being tested in the phase 1 GINAKIT2 clinical study (NCT03294954) in patients with R/R high risk neuroblastoma. The single-arm study will evaluate six dose levels of KUR-501 with patients receiving pre-dose lymphodepletion chemotherapy consisting of cyclophosphamide and fludarabine.

Neuroblastoma is a pediatric cancer and patients with R/R high risk neuroblastoma have a poor prognosis and a significant unmet medical need. The KUR-501 development program is also designed to provide autologous proof-of-concept for CAR-NKT cells in solid tumors using a validated target.

The GINAKIT2 study is supported by Athenex, Inc., which acquired Kuur Therapeutics in May 2021, and Alex’s Lemonade Stand Foundation, is conducted by Athenex’s collaborator, BCM, and is currently recruiting patients.

On May 17, 2021 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported financial results for the first quarter of 2021 ended March 31, 2021 and provided an update on the Company’s recent and anticipated future developments (Press release, Pieris Pharmaceuticals, MAY 17, 2021, View Source [SID1234580151]).

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"The last few months were marked by significant accomplishments. We leveraged existing and new alliances to materially bolster our balance sheet with $46 million in capital through recent upfront and milestone payments, focused equity stakes by partners AstraZeneca and Seagen, and ongoing funding support by our partners for several assets, ensuring that we are well positioned to execute on our strategic plans into 2023. These partnerships are helping us advance several therapeutic programs and further validate our Anticalin platform. We are pleased that dosing of asthmatic patients with PRS-060/AZD1402 has begun in the previously announced phase 2a study, and we expect to report data from this study next year. The phase 1 data we presented at AACR (Free AACR Whitepaper) last month for cinrebafusp alfa, our 4-1BB/HER2 bispecific, support entry into a phase 2 study this summer in HER2-high and HER2-low gastric cancer, where we are setting a high bar to govern development beyond the initial cohort in each study arm. The preclinical data we presented at AACR (Free AACR Whitepaper) with our co-development partner, Servier, for PRS-344/S095012, a 4-1BB/PD-L1 bispecific, provide a compelling rationale for clinical development, which will begin later this year. Expanding our roster of 4-1BB-based bispecifics advancing towards clinical development, we also recently announced an immuno-oncology collaboration with Boston Pharmaceuticals to advance PRS-342, a 4-1BB/GPC3 bispecific, into the clinic," said Stephen S. Yoder, President and Chief Executive Officer of Pieris.

PRS-060/AZD1402 and AstraZeneca Collaboration: Dosing has begun in the first part of the global phase 2a study of PRS-060/AZD1402, an inhaled IL-4 receptor alpha inhibitor under development in collaboration with AstraZeneca for the treatment of moderate-to-severe asthma. Pieris and AstraZeneca expect to announce data from the phase 2a study next year. Upon completion of the study, which is being sponsored and funded by AstraZeneca, Pieris will have options to co-develop and, subsequently, co-commercialize PRS-060/AZD1402 in the United States. Pieris and AstraZeneca continue to advance each of the four programs in the collaboration beyond PRS-060/AZD1402.
Cinrebafusp Alfa (PRS-343): Pieris is preparing a two-arm phase 2 study for cinrebafusp alfa, a 4-1BB/HER2 bispecific for the treatment of HER2-expressing solid tumors, in gastric cancer that will begin this summer and will set a high bar for clinical benefit, durability of response, and safety to determine further development beyond the planned initial 20 patients in each study arm. Pieris presented updated data for this program in an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021. In clinical trials to date, cinrebafusp alfa has shown an acceptable safety profile at all doses tested with no dose-limiting toxicities. Single-agent activity has been most pronounced on a Q2W regimen, which includes one confirmed complete response and four confirmed partial responses, and which will be the basis for phase 2 development. The bispecific also showed a clear dose response and a 4-1BB-driven mechanism of action based on clinical benefit and pharmacodynamic correlates. Additionally, clinical activity was observed in patients with "cold" tumors, as well as those with HER2-low expressing tumors. Supported by these data, the first arm of the announced phase 2 study will be in combination with ramucirumab and paclitaxel in HER2-high gastric cancer, while the second arm will be in combination with tucatinib in HER2-low gastric cancer. Collaboration partners Lilly and Seagen will supply ramucirumab and tucatinib, respectively, under previously announced drug supply agreements.
PRS-344/S095012 and Servier Collaboration: Pieris and Servier presented preclinical data for PRS-344/S095012, a 4-1BB/PD-L1 bispecific, as part of a poster session at the AACR (Free AACR Whitepaper) Annual Meeting 2021. PRS-344/S095012 induced a dose-dependent anti-tumor response and significantly extended survival in an anti-PD-L1-resistant mouse model. The data also demonstrated PRS-344/S095012 to be superior to the combination of individual PD-L1- and 4-1BB- targeting molecules. PRS-344/S095012 is expected to enter phase 1 development later this year. Pieris holds exclusive commercialization rights for PRS-344 in the United States and will receive royalties on ex-U.S. sales for this program. As part of our multi-program immuno-oncology collaboration, Servier is also responsible for further development of PRS-352, an undisclosed bispecific.
PRS-342/BOS-342 and Boston Pharmaceuticals Collaboration: Pieris entered into an exclusive product license agreement wherein Boston Pharmaceuticals will develop PRS-342, a 4-1BB/GPC3 immuno-oncology Anticalin-antibody bispecific fusion protein in the IND-enabling phase. Under the terms of the agreement, Boston Pharmaceuticals exclusively licensed worldwide rights to PRS-342. Pieris will receive an upfront payment of $10 million and is further entitled to receive up to approximately $353 million in development, regulatory, and sales-based milestone payments, and tiered royalties. Pieris will also contribute an undisclosed amount and input for manufacturing activities to support IND-readiness.
Preclinical Respiratory Pipeline: Pieris continues to advance several proprietary early-stage respiratory programs and expects to share data and rationale for advancement of one of its lead innovative proprietary programs this year.
First Quarter Financial Update:

Cash Position – Cash and cash equivalents totaled $66.8 million for the quarter ended March 31, 2021, compared to a cash and cash equivalents balance of $70.4 million for the quarter ended December 31, 2020. The increase was due to $13 million received from Seagen in March 2021 offset by operating cash needs. The quarter end cash balance excludes both $23 million received from AstraZeneca in April and $10 million to be received from Boston Pharmaceuticals.

R&D Expense – R&D expenses were $16.6 million for the quarter ended March 31, 2021, compared to $12.8 million for the quarter ended March 31, 2020. The increase reflects higher spending on preclinical activities for our proprietary respiratory program and manufacturing costs for our immuno-oncology programs, while maintaining flat spending on other non-project related R&D costs.

G&A Expense – G&A expenses were $4.1 million for the quarter ended March 31, 2021, compared to $4.4 million for the quarter ended March 31, 2020. The decrease reflects more one-time costs incurred in 2020 related to the move to a new R&D facility in Hallbergmoos, Germany, partially offset by higher consulting expenses in the first quarter of 2021.

Net Loss – Net loss was $4.2 million or $(0.07) per share for the quarter ended March 31, 2021, compared to a net loss of $3.6 million or $(0.07) per share for the quarter ended March 31, 2020.

First Quarter Financial Update Conference Call:

Pieris management will host a conference call beginning at 8:00 AM EDT on Monday, May 17, 2021, to discuss the first quarter of 2021 financial results and provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). Alternatively, a listen-only audio webcast of the call can be accessed here.

For those unable to participate in the conference call or listen to the webcast, a replay will be available on the Investors section of the Company’s website, www.pieris.com.

On May 17, 2021 Hummingbird Bioscience, an innovative clinical-stage biotech company focused on developing precision therapies against hard-to-drug targets to improve treatment outcomes, reported the close of its US$125 million Series C financing round (Press release, Hummingbird Bioscience, MAY 17, 2021, View Source [SID1234580169]). The financing was led by Novo Holdings, with significant participation from new investors including Frazier Healthcare Partners, Octagon Capital, EDBI, AMGEN Ventures, DROIA Ventures, Morningside Ventures, Pureos Bioventures, Polaris Partners, Affinity Asset Advisors, Ally Bridge Group and Altrium Capital Management. Existing investors including SK Inc, Heritas Capital, and Mirae Asset Venture Capital also joined the round.

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"We are delighted to have the support and confidence of leading global healthcare investors to advance the development of our unique pipeline of precision therapies against important, yet hard-to-drug targets. We have made tremendous progress since closing our Series B in 2019 and this financing reflects strong support for our platform, people, and development strategy," said Piers Ingram, Chief Executive Officer and co-founder, Hummingbird Bioscience. "With our world-class team of researchers and proprietary technologies, Hummingbird is at the cutting-edge of scientific discovery. These new funds give us further resources to invest in our early stage pipeline, as well as supporting the clinical development of our two lead programs that we believe can deliver very meaningful benefit for patients."

Proceeds from the financing will be used to advance the clinical development of Hummingbird’s lead assets including HMBD-001, a best-in-class HER3 antibody for NRG1-fusion and HER3-driven tumors, and HMBD-002, a first-in-class anti-VISTA neutralizing antibody for advanced solid tumors. The funds will also be used to expand the capabilities of Hummingbird’s proprietary Rational Antibody Discovery platform and progress the development of its next-generation pipeline of precision therapeutics including HMBD-009, a BCMA-TACI dual-specific T cell engager.

"We are delighted to lead this financing in Hummingbird and pleased to have attracted a syndicate of sophisticated investors to enable the acceleration of the company’s clinical development activities. We believe that Hummingbird’s novel data-driven, systems biology approach brings new precision to the field of antibody drug discovery and development, and we are proud to support the team in realizing their vision," said Kenneth Harrison, Partner, Novo Ventures.

Kenneth Harrison along with Dan Estes, General Partner, Frazier Healthcare Partners, and Kiel Kim, Vice President, SK Inc., will join Hummingbird’s board of directors.

"Hummingbird is at an exciting point, on the cusp of starting clinical studies for both HMBD-001 and HMBD-002. I look forward to working with the Hummingbird team in developing and building out its portfolio of drug candidates that have transformative potential for patients with cancer and autoimmune diseases," said Dan Estes.

Kiel Kim said: "There is significant potential for novel antibody-based therapeutics and through Hummingbird’s Rational Antibody Discovery platform, we can now discover high value antibodies for challenging targets. We look forward to continuing our partnership with Hummingbird to solve complex challenges in antibody development, and deliver highly differentiated therapies to patients in need."

On May 17, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported an exclusive license and collaboration agreement with Ji Xing Pharmaceuticals (Ji Xing) to develop and, if approved, commercialize the investigational drug etripamil in patients with paroxysmal supraventricular tachycardia (PSVT) and additional cardiovascular conditions in Greater China (Press release, Milestone Pharmaceuticals, MAY 17, 2021, View Source [SID1234580243]). Ji Xing is a biotechnology company headquartered in Shanghai and backed by RTW Investments, LP (RTW) focused on advancing innovative medicines in China.

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"This agreement marks an important step toward realizing our vision for etripamil to benefit patients living with PSVT globally while strengthening our balance sheet and executional capabilities through partnership," said Joseph Oliveto, President and Chief Executive Officer of Milestone Pharmaceuticals. "We look forward to partnering with the talented team at Ji Xing to develop and commercialize this promising therapy in the licensed regions."

"Etripamil has the potential to change the treatment paradigm for PSVT and could serve as a meaningful new therapeutic option for patients," said Peter Fong, Chief Executive Officer of Ji Xing and Head of Company Creation at RTW. "We are delighted to expand Ji Xing’s cardiovascular focus by partnering with Milestone and look forward to unlocking the full therapeutic potential of etripamil for patients with PSVT in China."

Under the terms of the agreement, Milestone will grant Ji Xing an exclusive license to develop and, if regulatory approval is obtained, commercialize etripamil in patients with PSVT in Greater China. Milestone will receive an upfront cash payment consisting of $15 million and a $5 million equity investment by RTW. In addition, Milestone is eligible to receive up to $107.5 million in milestone payments and royalties on future sales of etripamil in Greater China. Milestone will supply etripamil and delivery devices to Ji Xing. Ji Xing will be responsible for development and commercialization costs in Greater China.

About Paroxysmal Supraventricular Tachycardia

Paroxysmal supraventricular tachycardia (PSVT) is a rapid heart rate condition characterized by intermittent episodes of supraventricular tachycardia (SVT) that start and stop suddenly and without warning that affects approximately two million Americans. Episodes of SVT are often associated with symptoms including palpitations, sweating, chest pressure or pain, shortness of breath, sudden onset of fatigue, lightheadedness or dizziness, fainting, and anxiety. Certain calcium channel blockers have long been approved for the treatment of PSVT as well as other cardiac conditions. However, calcium channel blockers approved for the termination of SVT episodes must be administered intravenously under medical supervision, usually in an emergency department or other acute care setting.

About Etripamil

Etripamil, Milestone’s lead investigational product, is a novel calcium channel blocker designed to be a rapid-response therapy for episodic cardiovascular conditions. As a nasal spray that is self-administered by the patient, etripamil has the potential to shift the current treatment experience for many patients from the emergency department to the at-home setting. Milestone is conducting a comprehensive development program for etripamil, with Phase 3 trials ongoing in paroxysmal supraventricular tachycardia (PSVT) and a Phase 2 proof-of-concept trial is now underway in patients with atrial fibrillation and rapid ventricular rate (AFib-RVR).

On May 17, 2021 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company engineering a novel class of injectable biologics to selectively engage and modulate targeted T cells within the patient’s body, reported a business and clinical progress update for the first quarter 2021 (Press release, Cue Biopharma, MAY 17, 2021, View Source [SID1234608279]).

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"During the first quarter of 2021 and early second quarter, we continued to make significant progress advancing the Phase 1a/1b monotherapy trial of CUE-101 and continued development of our expanding pipeline and technology platforms, as well as enhancing our capital resources," said Daniel Passeri, chief executive officer of Cue Biopharma. "Importantly, we recently reported a confirmed partial response (PR) in a patient from our ongoing Phase 1 monotherapy dose escalation trial of CUE-101 and look forward to providing further details as well as describing the development implications for CUE-101 and potential of the CUE-100 series and Immuno-STAT platform, during the quarterly update call."

Kerri-Ann Millar, chief financial officer of Cue Biopharma, added, "We finished the first quarter of 2021 in a solid financial position which was further strengthened by the deployment of our at-the-market (ATM) common stock facility in April that enabled us to boost our cash position by an additional $10.4 million giving us operational runway into the fourth quarter of 2022."

Recent News & Business Updates

Reported PR in one patient and stable disease (SD) in five patients, confirmed by RECIST criteria, providing evidence of single-agent clinical activity of CUE-101 in the ongoing Phase 1 monotherapy dose escalation trial in late stage second-line and beyond patients with HPV+ recurrent/metastatic head and neck cancer, as well as evidence of both tumor-specific CD8+ T cell expansion and dose-dependent increases in NK cells.
Extended cash runway with sales in April of an aggregate of $10.4 million shares of our common stock pursuant to our ATM equity offering sales agreement with Stifel. As of April 30, 2021, we sold a cumulative total of 2,099,700 shares of common stock for aggregate net proceeds of $32.7M, net of commissions paid, under the sales agreement.
Initiated Phase 1 dose escalation clinical trial of CUE-101 in combination with Merck’s KEYTRUDA, an anti-PD-1 biologic agent, as first-line therapy in patients with advanced HPV16+ head and neck cancer.
Appointed renowned experts Abul K. Abbas, M.D., distinguished professor in pathology and former chair of the department of pathology at the University of California, San Francisco (UCSF) and Michael Kalos, Ph.D., managing director of Next Pillar Consulting, LLC and former executive vice president and head of research and development at ArsenalBio, to our Scientific Advisory Board (SAB).
First-Quarter 2021 Financial Results

The Company reported collaboration revenue of approximately $1.6 million and $0.9 million for the three months ended March 31, 2021 and 2020, respectively.

Research and development expenses were $9.8 million and $9.9 million for the three months ended March 31, 2021 and 2020, respectively. The decrease in research and development expenses of $0.9 million was primarily due to a decrease in laboratory and drug substance manufacturing costs as the clinical supply for our lead drug candidate, CUE-101, was produced during 2020, as well as a reduction in clinical and travel related expenses.

General and administrative expenses were $4.3 million and $4.0 million for the three months ended March 31, 2021 and 2020, respectively. The increase in general and administrative expense of $0.3 million was primarily due to an increase in stock-based compensation expense and legal fees incurred in the first quarter of 2021 as compared to the same period in 2020.