On November 10, 2021 Sapience Therapeutics, Inc., a biotechnology company focused on the discovery and development of peptide therapeutics to address difficult-to-treat cancers, reported that the company will present at the Piper Sandler 33rd Annual Healthcare Conference, taking place virtually on November 29, 2021 (Press release, Sapience Therapeutics, NOV 10, 2021, View Source [SID1234595152]).

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Members of Sapience’s management team will present a company overview and will also participate in one-on-one meetings with life science investors during the event.

On November 10, 2021 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapeutics targeting fundamental biological pathways of cancers, reported financial results for the third quarter ended September 30, 2021 and highlighted recent corporate accomplishments (Press release, Zentalis Pharmaceuticals, NOV 10, 2021, View Source [SID1234595222]).

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"This past quarter, Zentalis continued to execute on its clinical development strategy, and we are pleased with the progress we have made to advance our lead program, ZN-c3, a potentially best-in-class WEE1 inhibitor," commented Dr. Anthony Sun, Chairman and Chief Executive Officer of Zentalis. "Most recently, we initiated a Phase 1/2 trial with ZN-c3 in combination with gemcitabine in patients with osteosarcoma, further supporting the broad potential of ZN-c3 in combination therapy settings. Before year-end, we plan to have a total of six ongoing trials investigating our WEE1 inhibitor and look forward to initiating our potentially registrational biomarker-driven Phase 2 trial shortly. Additionally, the foundational research on the discovery of ZN-c3 was published in the peer-reviewed Journal of Medicinal Chemistry, which reviews our objectives for designing ZN-c3 as a potentially safer and more selective WEE1 inhibitor and in turn, overcoming limitations seen with other candidates in development."

Continued Dr. Sun, "We remain on track with our anticipated clinical milestones and are eager to provide future updates on these efforts. Looking ahead, we are excited to host a virtual R&D Day on December 16, during which our management team and key opinion leaders will provide a review of new preclinical science across multiple programs and share clinical updates on ZN-d5 and ZN-e4."

Program Highlights:

In September 2021, Zentalis dosed the first patient in the Phase 1/2 study of ZN-c3 in combination with gemcitabine, a chemotherapy used to treat certain malignant tumors, in patients with relapsed or refractory osteosarcoma. Zentalis recently received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for pediatric osteosarcoma. Zentalis expects to report initial results from this trial in the second half of 2022.
Enrollment is ongoing in the potentially registrational Phase 2 trial of ZN-c3 in women with recurrent or persistent USC. Following an end-of-Phase 1 meeting in July 2021, the FDA agreed in principle that ZN-c3 has the potential for an accelerated approval pathway based on the Phase 2 study design in USC.
In September 2021, research on the discovery of ZN-c3 was published in the Journal of Medicinal Chemistry. The paper reviews Zentalis’ objectives in designing ZN-c3 as a potentially safer and more selective WEE1 inhibitor, overcoming limitations seen with other WEE1 inhibitors.
In September 2021, Zentalis presented six poster presentations on the candidates: ZN-c3, a WEE1 inhibitor; ZN-c5, an oral selective SERD and ZN-d5, a BCL-2 inhibitor at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress.
Zentera Highlights:

In August 2021, Zentera, a Shanghai-based clinical-stage biopharmaceutical company formed by Zentalis, announced the completion of a $75 million Series B financing. Zentera plans to use the proceeds to advance the clinical development in China of three of Zentalis’ product candidates (ZN-c3, ZN-c5 and ZN-d5), as well as expand its pipeline through additional business development opportunities for China and global development.
Zentera received CTA acceptances in China for ZN-c3, ZN-c3 in combination, ZN-c5 and ZN-d5 and four clinical trials are ongoing.
Corporate Highlights:

In July 2021, Zentalis closed an underwritten follow-on offering of 3,565,000 shares of its common stock at a public offering price of $48.50 per share. The total net proceeds were approximately $162.2 million, after deducting underwriting discounts and commissions and offering expenses payable by Zentalis.
Third Quarter 2021 Financial Results

Cash and Marketable Securities Position: As of September 30, 2021, Zentalis had cash, cash equivalents and marketable securities of $366.8 million. We believe that our existing cash, cash equivalents and marketable securities as of September 30, 2021 will be sufficient to fund our operating expenses and capital expenditures requirements into the third quarter of 2023.
Research and Development Expenses: Research and development expenses for the three months ended September 30, 2021 were $54.0 million, compared to $24.7 million for the three months ended September 30, 2020. The increase of $29.3 million was primarily due to increases in external research and development expenses related to our clinical product candidates, as we advanced our clinical pipeline.
General and Administrative Expenses: General and administrative expenses for the three months ended September 30, 2021 were $8.9 million, compared to $10.1 million during the three months ended September 30, 2020. This decrease of $1.2 million was primarily attributable to a decrease of $0.6 million in legal fees and an increase in allocable overhead expenses to research and development expenses of $1.7 million, offset by an increase of $1.1 million of facilities and related permits/fees and licenses expenses.
Net Loss: Net loss was $10.5 million for the three months ended September 30, 2021, compared to $34.7 million for the three months ended September 30, 2020. The $24.2 million decrease in net loss was primarily the result of the gain on deconsolidation of Zentera recognized during the three months ended September 30, 2021, partially offset by increases in research and development expenses discussed above.

On November 10, 2021 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported a business update and financial results for the quarter ended September 30, 2021 (Press release, DiaMedica, NOV 10, 2021, View Source [SID1234595239]). Management will host a conference call Thursday, November 11, 2021, at 7:00AM Central Time/8:00AM Eastern Time to discuss its business update and third quarter financial results.

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Pivotal Phase 2/3 ReMEDy Trial of DM199 for Acute Ischemic Stroke Initiated & First Patient Dosed

The Company previously announced the initiation of the first site and reported dosing of the first patient for its pivotal ReMEDy2 trial. The ReMEDy2 trial is a randomized, double-blind, placebo-controlled Phase 2/3 adaptive trial intended to enroll approximately 350 patients. Patients enrolled in the study will be treated with either DM199 or placebo within 24 hours of the onset of acute ischemic stroke (AIS) symptoms. The trial is studying AIS in a patient population for whom thrombolysis and/or a catheter-based procedure, mechanical thrombectomy, are not medically appropriate or available due to constraints of clot location, comorbidity risks or time from estimated onset of stroke, which represents approximately 80% of all AIS patients.

Also as previously announced, the U.S. Food and Drug Administration (FDA) has accepted the Company’s protocol amendment to evaluate the effects of DM119 on the rate of recurrent AIS as a second, independent, primary endpoint. The FDA’s acceptance of the amendment allows the Company to evaluate the effects of DM199 on both stroke recoveries post AIS, as measured by the well-established modified Rankin Scale (mRS), and the incidence of AIS recurrence at day 90 as two separate, independent, primary endpoints, with each statistically powered for success. Recurrent strokes represent 25% of all ischemic strokes, often occur in the first few weeks after an initial stroke, and are typically more disabling, costly, and fatal than initial strokes. The Company further notes that there are no changes in treatment, duration, or study population of the trial as part of this protocol amendment.

Secondary endpoints for the study will evaluate participant deaths, mRS shift (which shows the treatment effect on participants across the full spectrum of stroke severity) and additional standard stroke scores (NIHSS and Barthel Index scores).

Fast Track Designation Granted to DM199 for Treatment of Acute Ischemic Stroke

The FDA granted Fast Track Designation to the Company’s lead candidate DM199 for the treatment of AIS. Fast Track is a process intended to facilitate the development and expedite the review of investigational drugs for the treatment of serious or life-threatening conditions where there is a significant unmet medical need. Drugs that receive Fast Track Designation may be eligible for more frequent communications and meetings with the FDA to review the drug’s development plan, including the design of the proposed clinical trials, use of biomarkers, and the extent of data needed for approval. Drugs with Fast Track Designation may also qualify for accelerated and priority review of new drug applications if relevant criteria are met.

Additional Interim Data From REDUX Phase 2 CKD Study for IgAN Presented at the ASN Kidney Week 2021 Virtual Conference

Additional data from the Company’s Phase 2 REDUX trial of DM199 in chronic kidney disease (CKD) was presented at the American Society of Nephrology’s (ASN) annual Kidney Week meeting taking place during the first week of November 2021. In the IgA Nephropathy (IgAN) cohort, in addition to continuing to show statistically significant reductions (over 30% decrease) in albuminuria in participants with moderate to severe baseline albuminuria, also demonstrated early signals of potential disease modification with the APRIL and IgA1 biomarkers decreasing 35% and 22%, respectively.

Balance Sheet Strengthened with $30 Million Private Placement

In September 2021, the Company issued and sold in a private placement an aggregate 7,653,060 common shares at a purchase price of $3.92 per share to ten accredited investors, resulting in gross proceeds of $30.0 million and net proceeds of $29.9 million, after deducting offering expenses.

"We are pleased that our regulatory interactions have led to acceptance of the protocol with the FDA and the Fast Track Designation status which will give the continued focus that this trial deserves given the unmet medical need," said Rick Pauls, DiaMedica’s President and Chief Executive Officer. " We believe that we are well positioned to advance toward our goal of offering a treatment option for the millions of patients around the world who suffer from a stroke and need a better chance to recover and avoid recurrent strokes."

Financial Results

Research and development (R&D) expenses increased slightly to $2.3 million for the three months ended September 30, 2021, up from $2.2 million for the three months ended September 30, 2020. R&D expenses increased to $6.9 million for the nine months ended September 30, 2021, compared to $5.1 million for the nine months ended September 30, 2020, an increase of $1.8 million. The increase for the nine month comparison was primarily due to a number of factors including costs incurred for the Company’s pivotal ReMEDy2 clinical study, increased year-over-year costs related to manufacturing process development and increased personnel costs associated with adding staff to support R&D operations. These increases were partially offset by decreased costs incurred for the earlier ReMEDy Phase 2 stroke study, which completed in the prior year, and the REDUX study as the number of enrollments declined in the later stages of the study.

General and administrative (G&A) expenses were $1.1 million for the three months ended September 30, 2021, down from $1.2 million for the three months ended September 30, 2020. G&A expenses increased to $3.5 million for the nine months ended September 30, 2021, up $0.2 million from $3.3 million for the nine months ended September 30, 2020. The increase for the nine month comparison was primarily due to increased professional services costs and increased personnel costs to support the Company’s expanding clinical programs. These increases were partially offset by reduced non-cash, share based compensation costs.

Balance Sheet and Cash Flow

As of September 30, 2021, DiaMedica had cash, cash equivalents and marketable securities of $48.1 million, working capital of $46.9 million and shareholders’ equity of $47.0 million, compared to $27.5 million in cash, cash equivalents and marketable securities, $25.9 million in working capital and shareholders’ equity of $26.0 million as of December 31, 2020. The increases in combined cash, cash equivalents and marketable securities and in working capital are due primarily to the net proceeds from the Company’s September 2021 private placement offering, partially offset by operating costs incurred during the quarter.

Net cash used in operating activities for the nine months ended September 30, 2021, was $9.4 million compared to $6.2 million for the nine months ended September 30, 2020. This increase relates primarily to the increase in the net loss, partially offset by non-cash share-based compensation and the effects of the changes in operating assets and liabilities.

Conference Call and Webcast Information

DiaMedica Management will host a conference call and webcast to discuss its business update and third quarter 2021 financial results on Thursday, November 11, 2021, at 7:00AM Central Time:

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor relations – events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until November 18, 2021, by dialing (800) 770-2030 (US Toll Free) and entering the replay passcode: 4814247.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as stroke, chronic kidney disease, retinopathy, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and South Korea for decades. DM199 is currently being studied in patients with acute ischemic stroke and patients with chronic kidney disease. In September 2021, the FDA granted Fast Track Designation to DM199 for the treatment of AIS.

On November 10, 2021 Ikena Oncology, Inc. (Nasdaq: IKNA, "Ikena", "Company"), a targeted oncology company focused on developing novel cancer therapies targeting key signaling pathways, reported financial results for the quarter ended September 30, 2021 (Press release, Ikena Oncology, NOV 10, 2021, View Source [SID1234595629]). The Company also provided an update across the organization and pipeline, including the acceptance of an IND for their TEAD inhibitor, IK-930, as they advance it towards initiation of a Phase 1 clinical trial for patients with tumors known to have high incidence of Hippo pathway genetic alterations.

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In addition to the Company’s progress on IK-930, Ikena is driving several programs targeting the RAS pathway. Emerging data from discovery and translational efforts have focused the team on key nodes in the RAS pathway that provide potential for clinical advancement of targeted therapies both as single agents and in combination to overcome therapeutic resistance in RAS mutated cancers. This work includes further efforts on ERK5 biology and chemistry optimization prior to candidate nomination, but also highlights new opportunities in the pathway. Multiple development candidates are expected to emerge from these RAS discovery programs over the next 18 months.

"Patients with mutations in the Hippo and RAS pathways represent cancer populations with significant unmet needs. At Ikena, we are committed to generating deep scientific support for our approach in these pathways and in the identification of therapies that could best treat their individual disease," commented Mark Manfredi, Ph.D., Chief Executive Officer of Ikena. "The team has done tremendous work exploring TEAD biology, advancing IK-930, and generating robust translational data to optimize the clinical development plan. The IND acceptance is a foundational milestone for our targeted oncology portfolio and for our approach to biomarker-driven cancer treatments. The progress across the entirety of our pipeline demonstrates our commitment to science and medicine that will lead to better therapies with the best chance of helping patients."

Summary of Recent Pipeline Progress and Corporate Update

IK-930: TEAD Inhibitor in the Hippo Signaling Pathway

IND accepted by the Food and Drug Administration (FDA); clinical trial expected to initiate in the first quarter of 2022
Program data was shared at the AACR (Free AACR Whitepaper)-NCI-EORTC 2021 Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper)

Data highlighted translational methods using a novel assay developed by Ikena and suggested YAP/TAZ activity could be a potential biomarker in determining patients that could benefit from TEAD inhibition
Preclinical data showed the importance of the Hippo pathway in resistance to EGFR and MEK inhibition and the potential of therapeutic combinations for patients, supporting our plans to evaluate combinations of IK-930 in multiple tumor types
RAS Pathway: Progressing Multiple Targets and Novel Approaches

Ikena has been expanding its discovery and translational research efforts in the RAS pathway beyond ERK5, with a particular focus on targeting nodes in the pathway that have potential for monotherapy and combinations both intra-pipeline and with other targeted agents
Continued drug discovery and additional translational efforts are being conducted prior to potential ERK5 candidate nomination. The RAS pathway discovery programs are expected to result in multiple targeted oncology development candidates in the next 18 months
IK-175 & IK-412: AHR Inhibitor and Kynurenine Degrading Enzyme Programs in Collaboration with Bristol Myers Squibb

IK-175 is currently being evaluated in a Phase 1 study to assess its impact in solid tumors and in urothelial carcinoma though aryl hydrocarbon receptor (AHR) inhibition

The study expanded its monotherapy cohort in urothelial carcinoma earlier this year and recently completed the dose escalation of the combination of IK-175 with nivolumab
The combination arm expansion cohort in urothelial carcinoma is now open for enrollment, including nuclear AHR positive enriched subset of patients
Three posters on IK-175 will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference on November 12, 2021 highlighting translational data and a trial-in-progress presentation on the Phase 1 clinical trial design
IK-175 clinical data presentation is planned for a major medical conference in 2022
As previously reported, manufacturing lead times have been delayed for IK-412 and as such, Ikena and Bristol Myers Squibb are continuing to evaluate the best path forward
IK-007: EP4 Receptor Antagonist Currently in Phase 1 Clinical Trial

The IK-007 Phase 1 study in MSS colorectal cancer is on track to complete enrollment by the end of 2021; data will be submitted to a medical conference in 2022
An investigator-initiated trial (IIT) of IK-007 in combination with the chemotherapy agent eribulin in metastatic inflammatory breast cancer (IBC) led by Naoto Ueno, M.D., of the University of Texas MD Anderson Cancer Center was launched in September 2021

IBC is a rare, aggressive form of breast cancer with high unmet medical need
Increased COX-2 expression in the EP4 pathway has been associated with poor prognosis in IBC patients
Organizational Growth: Addition of Senior Clinical and Research Expertise

Ikena recently added deep expertise in clinical development, clinical operations, and cancer biology to the leadership team:

Karim Malek, M.D.: Vice President, Clinical Development
Medical oncologist with over 30 years of experience both in the clinic and in research and development, with strong academic roots
Joined Ikena from Takeda Pharmaceuticals where he was the global clinical lead on multiple immune-oncology platforms and garnered an extensive background in clinical trial design and execution
Jennifer Schroeder, PMP: SVP Clinical Development Operations
Seasoned executive with nearly 25 years of experience ranging from start-ups to Fortune 500 companies
Served over a decade with Pfizer and was one of the founding business process owners where she helped implement clinical trial management systems and directed a globally focused team of seven leading enterprise-scale projects
Holly Koblish, Ph.D.: Vice President, Cancer Biology
Over 20 years of experience in oncology drug discovery, asset development and target selection
Extensive drug discovery background as a pharmacology leader at Incyte, where she participated in the discovery of pemigatinib and capmatinib, two medicines recently approved in the U.S. and abroad
Financial Results for the Quarter Ended September 30, 2021

As of September 30, 2021, the Company had cash and cash equivalents totaling $245.9 million, which will fund operations through 2023. Net cash used in operations was $18.0 million for the third quarter of 2021 as compared to $7.7 million for the third quarter of 2020.

Research and development expenses for the third quarter 2021 were $13.4 million, compared to $7.2 million for the third quarter 2020. The increase in research and development expense was primarily attributable to IND-enabling studies, manufacturing development costs and clinical trial start-up costs for IK-930, manufacturing costs for IK-175, the on-going IND-enabling studies for IK-412, and research activities for other discovery stage programs. In addition, research and development expenses related to personnel and overhead expenses increased due to an increase in headcount. This increase in research and development expenses was partially offset by a decrease in development activities for IK-007.

General and administrative expenses for the third quarter were $4.9 million, compared to $1.8 million for the third quarter 2020. The increase was primarily attributable to an increase in compensation expense due to an increase in headcount and in insurance expense, as well as general increases in audit, legal, consulting and facilities expenses to support our operations as a public company.

Net loss for the third quarter 2021 was $14.5 million, compared to $6.2 million for the third quarter.

On November 10, 2021 CellPoint and Hypertrust Patient Data Care (HPDC) reported the launch of xCellit (Press release, CellPoint, NOV 10, 2021, https://cellpoint.bio/cellpoint-and-hypertrust-co-develop-xcellit-platform-for-cell-therapies-at-the-point-of-care/ [SID1234616306]). The xCellit system was co-developed to make autologous cell therapies available more rapidly and efficiently by facilitating the scheduling, monitoring and data capturing of cell treatment workflows. It is based on HPDC’s X-Chain Cell & Gene Therapy Orchestration solution and fully tailored to the CellPoint point-of-care manufacturing model.

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Using xCellit in combination with a closed and automated manufacturing device developed by Lonza (Cocoon), patient vein-to-vein times can be reduced to 6 days only, as compared to the industry standard of 4-6 weeks. This improvement is made possible as point-of-care manufacturing eliminates the need for complex, time-consuming and cost-intensive logistics.

"We are excited about our partnership with Hypertrust Patient Data Care. xCellit provides an end-to-end platform to digitize, streamline and secure our point-of-care supply chains for our CAR-T treatments. The convenient dashboards and notification triggers are critical in continuously involving and informing all stakeholders along the treatment workflow", says Tol Trimborn, CEO of CellPoint.

Andreas Göbel, Managing Director at Hypertrust Patient Data Care adds: "This is a great step to further advance Cell & Gene therapies. The collaboration with CellPoint shows that even in fully decentralized setups it is possible to provide the required level of trust between multiple parties so that personalized treatment data can be handled reliably and securely."

The point-of-care model brings a new, decentralized approach to autologous cell therapies. Currently available cell therapies involve numerous sequential steps: taking material from the patient, freezing, shipment by air freight, custom clearance, thawing, central factory manufacturing, quality control and release, transport back to the patient, and finally administering the product to the same patient. This current model struggles with limited manufacturing capacity and tremendous costs. In CellPoint’s point-of-care model, the production workflow is executed directly at the hospital where the patient is treated, circumventing many steps like cryopreservation and transport, driving down time and costs.

The xCellit platform provides a comprehensive set of features: a fully blown manufacturing execution system (MES) component including batch record QA/QC (quality assurance/quality control) and release in a remote environment. At its core, xCellit offers a highly trustworthy and customizable workflow process engine supporting the entire personalized medicine supply chain from patient treatment to recovery.

More information about point-of-care treatments and the xCellit platform is also available in the joint webinar by CellPoint, Dell and HPDC.