On August 3, 2022 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results and highlights for the second quarter ended June 30, 2022 (Press release, GlycoMimetics, AUG 3, 2022, View Source [SID1234617373]). Cash and cash equivalents as of the end of the quarter were $60.2 million.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During second quarter, we made strides in advancing our transformation from a research company to a commercially focused organization and are encouraged by the continued progress of our pivotal Phase 3 trial of uproleselan in relapsed/refractory AML," said Harout Semerjian, Chief Executive Officer. "Clearance by the FDA of the IND for GMI-1687 demonstrates our ability to create and advance innovative drug candidates for clinical development. GMI-1687 is now ideally suited for partnership and we are actively pursuing a licensing agreement for continued development of this novel molecule in sickle cell disease."

Operational Highlights

Uproleselan

GlycoMimetics continued efforts to clean the data received from the 70 sites in the U.S., Europe, Canada, and Australia that enrolled a total of 388 patients in the Company’s pivotal Phase 3 trial in relapsed/refractory AML. Progress to date now enables the Company to share a comparison of the demographics of those 388 patients against the patient demographics from the Company’s completed phase 2 study with respect to age, severity of AML, prior stem cell transplantation rate, and distribution of relapsed and refractory patients (Table 1). The Company has previously disclosed and will continue to update its projection of mid-year 2023 for the overall survival events trigger, with disclosure of top-line data results shortly thereafter.
The National Cancer Institute (NCI) continues to prepare for its planned interim analysis of event free survival of the 267 patients in its Phase 2/3 clinical trial evaluating uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy. The Company intends to publicly share the outcome of the NCI’s analysis of the Phase 2 data.
Investigator-sponsored clinical trials to evaluate expanded indications for uproleselan continue to progress at the University of California-Davis, Washington University at St. Louis, MD Anderson Cancer Center, and the University of Michigan.
GMI-1687

In June, GlycoMimetics received clearance from the FDA of an IND application for clinical development of GMI-1687 in SCD.
GMI-1687 is a highly potent E-selectin antagonist initially targeted for development to treat acute vaso-occlusive crises (VOCs) in SCD with potential to address a high unmet medical need.
E-selectin is believed to play a major role in the cascade of events leading to clots and blockages that cause patients’ VOCs. The administration of GMI-1687 via subcutaneous injection may have the potential to offer a treatment option at the onset of pain crisis.
The Company is actively seeking a licensing partner to continue clinical development of this drug candidate.
Second Quarter 2022 Financial Results:

Cash position: As of June 30, 2022, GlycoMimetics had cash and cash equivalents of $60.2 million as compared to $90.3 million as of December 31, 2021.

Revenue: There was minimal revenue recognized during the three months ended June 30, 2022 and 2021.

R&D Expenses: The Company’s research and development expenses decreased to $8.0 million for the quarter ended June 30, 2022, as compared to $10.2 million for the same period in 2021. The decreased expenses were primarily due to lower clinical trial and development costs related to our ongoing global Phase 3 clinical trial of uproleselan in individuals with relapsed/refractory AML as patient enrollment ended in November 2021.

G&A Expenses: The Company’s general and administrative expenses increased to $5.5 million for the quarter ended June 30, 2022, as compared to $4.2 million for the first quarter of 2021 primarily due to commercial start-up expenses for uproleselan.

Shares Outstanding: Shares of common stock outstanding as of June 30, 2022, were 52,423,944.

The Company will host a conference call and webcast today at 8:30 a.m. ET. To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time.

A live webcast of the call will be available on the "Investors" tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call.

About Uproleselan

Discovered and developed by GlycoMimetics, uproleselan is an investigational, first-in-class, targeted inhibitor of E-selectin. Uproleselan (yoo’ pro le’sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy designation from the U.S. FDA and from the Chinese National Medical Products Administration for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment.

About GMI-1687

Discovered and developed by GlycoMimetics, GMI-1687 is a potent E-selectin antagonist that has been shown in animal models to be fully bioavailable following subcutaneous administration. It is a second-generation compound that may be able to be developed to address certain challenges of IV therapies for SCD. E-selectin is believed to play a major role in the cascade of events leading to clots and blockages that cause pain crises in people living with SCD. The administration of GMI-1687 via subcutaneous injection may have the potential to offer a treatment option at the onset of pain crisis.

On August 3, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported it has entered into a broad strategic collaboration and license agreement with Roche, focused on developing allogeneic CAR-T therapies directed to hematologic malignancies (Press release, Poseida Therapeutics, AUG 3, 2022, View Source [SID1234617389]). The global collaboration covers the research and development of multiple existing and novel "off-the-shelf" cell therapies against targets in multiple myeloma, B-cell lymphomas and other hematologic indications.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited to partner and collaborate with Roche, one of the world’s largest biotechnology companies, which has a successful track record in the discovery, development and commercialization of innovative medicines," said Mark Gergen, Chief Executive Officer of Poseida. "Roche is an ideal strategic partner for Poseida with its industry-leading R&D capabilities in oncology, complementary technologies and expertise, and global regulatory and commercial capabilities. Working together, we look forward to advancing novel allogeneic cell therapies based upon Poseida’s technologies for patients battling cancer."

Under the agreement, Roche will receive from Poseida either exclusive rights or options to develop and commercialize a number of allogeneic CAR-T programs in Poseida’s portfolio that are directed to hematologic malignancies, including P-BCMA-ALLO1, an allogeneic CAR-T for the treatment of multiple myeloma and for which a Phase 1 study is underway, and P-CD19CD20-ALLO1, an allogeneic dual CAR-T for the treatment of B-cell malignancies with an IND expected in 2023. Building on complementary expertise and capabilities, the parties will also collaborate in a research program to create and develop next-generation features and improvements for allogeneic CAR-T therapies, from which they would jointly develop additional allogeneic CAR-T product candidates directed to existing and new hematologic targets. For a subset of both the Poseida portfolio programs licensed or optioned to Roche and the parties’ future collaboration programs, Poseida will conduct the Phase 1 studies and manufacture clinical materials before transitioning the programs to Roche for further development and commercialization. Roche will be solely responsible for the late-stage clinical development and global commercialization of all products that are subject to the collaboration.

"We are excited to partner with Poseida to further explore the potential of allogeneic cell therapies to transform cancer care by developing off-the-shelf products that can address high unmet medical needs for a broad patient population," said James Sabry, Global Head of Pharma Partnering at Roche. "Poseida’s differentiated platform technologies complement our ongoing internal efforts and partnerships to discover and develop cell therapies as a next generation of medicines for patients."

Under the agreement, Poseida will receive $110 million upfront and could receive up to $110 million in near-term milestones and other payments in the next several years. In addition, Poseida is eligible to receive research, development, launch, and net sales milestones and other payments potentially up to $6 billion in aggregate value, as well as tiered net sales royalties into the low double digits, across multiple programs.

"We are thrilled that Roche has embraced the opportunity to partner with us and use Poseida’s unique allogeneic approach to develop CAR-T product candidates," said Devon J. Shedlock, Ph.D., Chief Scientific Officer, Cell Therapy at Poseida. "Using our proprietary technologies and manufacturing process including our booster molecule, we have the potential to develop and manufacture a product with high levels of stem cell memory T cells, which are correlated with potent antitumor efficacy in the clinic, at a scale that can potentially reach more patients and enable broad commercial use."

The effectiveness of the agreement is subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act (HSR Act).

Poseida Therapeutics Conference Call and Webcast Information

Wednesday, August 3, 2022 at 8:30 a.m. ET

Poseida’s management team will host a conference call and webcast today at 8:30 a.m. ET to discuss the collaboration and Poseida’s novel approach to allogeneic cell therapy. The dial-in numbers for domestic and international callers are 800-267-6316 and 203-518-9814, respectively. The conference ID number for the call is PSTX0803.

Participants may access the live webcast on the Investors & Media Section of the Poseida website, www.poseida.com. An archived replay of the webcast will be available for approximately 30 days following the event.

On August 3, 2022 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated oncology therapeutics, reported that Sean McCarthy, D.Phil., chief executive officer and chairman, will virtually participate in the following investor conferences in August (Press release, CytomX Therapeutics, AUG 3, 2022, View Source [SID1234617407]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

BTIG Biotechnology Conference 2022
Date: Tuesday, August 9, 2022

2022 Wedbush PacGrow Healthcare Virtual Conference
Date: Wednesday, August 10, 2022
Panel Discussion: 2:20 p.m. ET

A live webcast of the Wedbush panel discussion will be available on the Events and Presentations page of CytomX’s website at www.cytomx.com. In addition, management will be available for one-on-one meetings with investors who are registered to attend the conferences.

On August 3, 2022 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the second quarter ended June 30, 2022 (Press release, Vanda Pharmaceuticals, AUG 3, 2022, View Source [SID1234617426]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"During the second quarter we made significant progress towards commercializing our products and improving access to HETLIOZ for patients with Non-24," said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board. "We advanced our clinical pipeline, with significant clinical milestones expected in the coming quarters, and we are preparing our NDA for tradipitant in gastroparesis and our sNDA for HETLIOZ in insomnia."

Financial Highlights

Second Quarter of 2022

Total net product sales from HETLIOZ and Fanapt were $64.4 million in the second quarter of 2022, a 5% decrease compared to $67.9 million in the second quarter of 2021.

HETLIOZ net product sales were $41.2 million in the second quarter of 2022, a 7% decrease compared to $44.5 million in the second quarter of 2021, due in part to continued reimbursement challenges for prescriptions for patients with Non-24.

Fanapt net product sales were $23.2 million in the second quarter of 2022, a 1% decrease compared to $23.4 million in the second quarter of 2021.

Net income was $2.6 million in the second quarter of 2022 compared to $9.7 million in the second quarter of 2021.
First Six Months of 2022

Total net product sales from HETLIOZ and Fanapt were $124.6 million in the first six months of 2022, a 5% decrease compared to $130.6 million in the first six months of 2021.

HETLIOZ net product sales were $78.2 million in the first six months of 2022, a 7% decrease compared to $83.9 million in the first six months of 2021, due in part to continued reimbursement challenges for prescriptions for patients with Non-24.

Fanapt net product sales were $46.4 million in the first six months of 2022, a 1% decrease compared to $46.7 million in the first six months of 2021.

Net loss was $3.9 million in the first six months of 2022 compared to net income of $18.3 million in the first six months of 2021.

Cash, cash equivalents and marketable securities (Cash) was $440.9 million as of June 30, 2022, representing an increase to Cash of $44.4 million compared to June 30, 2021.
Key Operational Highlights

HETLIOZ (tasimelteon)

Clinical trials for HETLIOZ in delayed sleep phase disorder (DSPD) and sleep disturbances in autism spectrum disorder (ASD) are currently enrolling patients.

Vanda is preparing for the submission of a supplemental New Drug Application (sNDA) for HETLIOZ in the treatment of insomnia.

Since November 2021, more than 15 states have revised or agreed to revise their Medicaid prior authorization criteria to broaden access to HETLIOZ for patients with Non-24 and patients with nighttime sleep disturbances in Smith-Magenis Syndrome (SMS).

In July 2022, an Administrative Law Judge struck down a Medicare Part D plan policy that blocked HETLIOZ coverage for sighted Non-24 patients. Vanda intends to advocate with other Part D plans to challenge similar policies and improve HETLIOZ access for Non-24 patients.

In January 2022, Vanda settled its HETLIOZ patent litigation against one of the Abbreviated New Drug Application (ANDA) defendants. The trial for the consolidated lawsuit against the remaining defendants was held in March 2022. A decision is expected from the court by the end of 2022.
Tradipitant

Vanda is continuing to conduct an open-label safety study for tradipitant in gastroparesis and continues to receive requests from patients seeking access to tradipitant through the Expanded Access program that has multiple patients who have taken tradipitant for more than a year.

Vanda recently held a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) to discuss the planned New Drug Application (NDA) submission for tradipitant in the short-term treatment of nausea in gastroparesis. Vanda is preparing for the submission of the NDA for this indication.

The Phase III study of tradipitant in the treatment of motion sickness is approximately 30% enrolled.
Fanapt (iloperidone)

Enrollment of the Phase III clinical study of Fanapt in acute manic episodes in patients with bipolar disorder is close to being fully enrolled. The study is a placebo controlled four-week evaluation of approximately 400 patients at sites in the U.S. and Europe. Results are expected by the end of 2022.
VQW-765

The Phase II clinical study of a single-dose treatment of VQW-765 to alleviate social/performance anxiety is fully enrolled. Results are expected by the end of 2022.
GAAP Financial Results

Net income was $2.6 million in the second quarter of 2022 compared to net income of $9.7 million in the second quarter of 2021. Diluted net income per share was $0.05 in the second quarter of 2022 compared to diluted net income per share of $0.17 in the second quarter of 2021.

Net loss was $3.9 million in the first six months of 2022 compared to net income of $18.3 million in the first six months of 2021. Diluted net loss per share was $0.07 in the first six months of 2022 compared to diluted net income per share of $0.32 in the first six months of 2021.

2022 Financial Guidance

Conference Call

Vanda has scheduled a conference call for today, Wednesday, August 3, 2022, at 4:30 PM ET. During the call, Vanda’s management will discuss the second quarter 2022 financial results and other corporate activities. Investors can call 1-800-715-9871 (domestic) or 1-646-307-1963 (international) and use passcode number 4304469. A replay of the call will be available on Wednesday, August 3, 2022, beginning at 8:30 PM ET and will be accessible until Wednesday, August 10, 2022 at 8:30 PM ET. The replay call-in number is 1-800-770-2030 for domestic callers and 1-609-800-9909 for international callers. The passcode number is 4304469.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

On August 3, 2022 Advanced Proteome Therapeutics Corporation (TSXV: APC) (FSE: 0E81) ("APC" or the "Company") reported that the United States Patent and Trademark Office has issued a patent to its US subsidiary, Advanced Proteome Therapeutics Inc. ("APTI") (Press release, Advanced Proteome Therapeutics, AUG 3, 2022, View Source [SID1234622591]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On August 2, 2022, APTI was issued U.S. Patent No. 11,400,165 titled ‘Composition and method for modifying polypeptides’. The patent covers highly site-selective antibody conjugate compositions produced with APTI’s two-step conjugation technology including the half-product of the first step and final antibody-drug conjugate (ADC) product of the second step. The claims broadly cover antibodies as a class and different classes of payloads including toxins, drugs and chelators. The patent term, including patent term adjustment, is expected to extend into November 2039. This is the first patent issued under US patent application 16/180,960. APTI is continuing to pursue expanded claims and additional patents within the initial application in order to create additional layers of intellectual property protection.

Dr. Benjamin Krantz, President and CEO of APTI commented, "The issuance of this patent is a critical milestone for APTI. The patent provides market exclusivity to highly site-selective ADC compositions that are created with our two-step antibody conjugation approach. This protection greatly enhances our ability to monetize our invention and increases our attractiveness to potential collaborators. This is another step forward in our 2022 plan and I look forward to other successes in the near future."