On November 13, 2020 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants, reported financial results for the third quarter ended September 30, 2020, and provided corporate highlights (Press release, PMV Pharma, NOV 13, 2020, View Source [SID1234570910]).

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"PMV Pharma has achieved important scientific, clinical, and operational milestones over the past quarter," said David Mack, Ph.D., President and Chief Executive Officer. "The Investigational New Drug (IND) application for our lead product candidate PC14586 was cleared by the U.S. FDA in September. We believe the capital raised to date will enable us to execute on the clinical development of PC14586, while also continuing to invest in our discovery pipeline of small molecule, tumor-agnostic precision medicine products."

Corporate Highlights

Received IND clearance from the FDA to initiate a Phase 1/2 study of PC14586 in patients with advanced solid tumors that have a p53 Y220C mutation. The Phase 1/2 study will enroll patients with a p53 Y220C mutation as determined by next generation sequencing. Phase 1 will evaluate escalating doses of PC14586 to determine the recommended Phase 2 dose and to assess safety, pharmacokinetics, and preliminary anti-tumor activity. Phase 2 will determine the overall response rate and duration of response. For more information, please visit www.clinicaltrials.gov (NCT study identifier NCT04585750).
In October, the U.S. FDA granted PMV Pharma Fast Track designation to PC14586 for the treatment of patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.
Expanded PMV Pharma’s management team and Board of Directors by adding Robert Ticktin as General Counsel, along with Rich Heyman, Ph.D. as Chair of the Board of Directors, and Laurie Stelzer, as Audit Chair of the Board of Directors.
Advanced its R273H program, targeting a second p53 hot spot mutant, toward lead optimization, and progressed the Company’s pipeline derived from its proprietary discovery platform.
Raised $70.0 million in a Series D financing round in July 2020 with Avoro Capital, RA Capital Management, and Wellington Management Company joining existing investors OrbiMed Advisors, Nextech Invest, Viking Global Investors, and Boxer Capital of Tavistock Group.
Successfully completed an IPO at a public offering price of $18 per share, with gross proceeds totaling approximately $243.5 million.
Third Quarter 2020 Financial Highlights

PMV Pharma ended the third quarter with $373.1 million in cash, cash equivalents, and marketable securities compared to $101.5 million as of December 31, 2019. Net cash provided by financing activities for the nine months ended September 30, 2020 was $294.3 million compared to $0.1 million for the nine months ended September 30, 2019. Net cash used in operations was $22.4 million for the nine months ended September 30, 2020 compared to $17.0 million for the nine months ended September 30, 2019.
Net loss for the quarter ended September 30, 2020 was $8.8 million compared to $6.2 million for the quarter ended September 30, 2019.
Research and development (R&D) expenses were $6.0 million for the quarter ended September 30, 2020 compared to $4.9 million for the quarter ended September 30, 2019. The increase in R&D expenses was primarily related to increase in personnel, preclinical development, and IND filing of PC14586.
General and administrative (G&A) expenses were $2.7 million for the quarter ended September 30, 2020, compared to $1.5 million for the quarter ended September 30, 2019. The increase in G&A expenses was primarily due to an increase in personnel and other corporate costs related to building out infrastructure for growth.

On November 13, 2020 Kintara Therapeutics, Inc. (Nasdaq: KTRA) ("Kintara" or the "Company"), a biopharmaceutical company focused on the development of new solid tumor cancer therapies,reported its financial results for the first quarter ended September 30, 2020 and provided a corporate update (Press release, Kintara Therapeutics, NOV 13, 2020, View Source [SID1234570908]).

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"The first quarter of our fiscal year marked the beginning of a new era for the company as we completed the acquisition of Adgero," commented Saiid Zarrabian, Kintara’s President and Chief Executive Officer. "In conjunction with this transformational milestone, we strengthened our balance sheet with a $25 million private placement to enable us to execute a timely advance of our key programs including the clinical stage of the GCAR GBM AGILE registrational study for VAL-083 and the confirmatory cutaneous metastatic breast cancer study for REM-001."

First Quarter Highlights and Recent Developments

Consummated the acquisition of Adgero, a privately held biopharmaceutical company focused on the development of its late stage photodynamic therapy platform for the treatment of serious cutaneous oncology indications, which created a diversified biopharmaceutical company with a robust product pipeline targeting rare, unmet medical needs in oncology (August 2020).

Completed a private placement of Series C Convertible Preferred Stock for aggregate gross proceeds of approximately $25 million, or net proceeds of approximately $21.6 million (August 2020).

Executed a definitive agreement with the Global Coalition for Adaptive Research (GCAR) to include VAL-083 in GCAR’s Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) study, an adaptive clinical trial platform in glioblastoma multiforme (GBM). Kintara will supply GCAR with the VAL-083 drug along with the funding to support the VAL-083 arm of the GBM AGILE registrational study. In turn, GCAR will manage all operational aspects of the study, including site activation and patient enrollment (October 2020).

Received award notification of a Small Business Technology Transfer grant to study the use of REM-001 in the prevention of arteriovenous fistula maturation failure (AFMF), a cardiovascular-related condition that occurs in hemodialysis patients. This grant will allow Kintara to further study the use of REM-001 in the prevention of AFMF in preclinical models (July 2020).
SUMMARY OF FINANCIAL RESULTS FOR FISCAL YEAR 2021 FIRST QUARTER ENDED SEPTEMBER 30, 2020

At September 30, 2020, the Company had cash and cash equivalents of approximately $22.6 million. In August 2020, the Company completed the private placement of Series C Convertible Preferred Stock for gross proceeds of approximately $25 million, or net proceeds of approximately $21.6 million. The cash and cash equivalents at September 30, 2020, along with the proceeds from warrant exercises received subsequent to September 30, 2020, are expected to be sufficient to fund the Company’s planned operations into the fourth quarter of calendar year 2021.

For the quarter ended September 30, 2020, the Company reported a net loss of approximately $19.5 million, or $1.33 per share, compared to a net loss of approximately $1.6 million, or $0.21 per share, for the quarter ended September 30, 2019. The increase in the current quarter was largely due to the recognition of $16.0 million of non-cash expenses related to the acquisition of in-process research and development costs associated with the Adgero transaction.

On November 13, 2020 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company developing innovative therapies for the treatment of neurodegenerative diseases and cancer, and supporting multiple collaborations with large biotech and major pharma partners through a proprietary discovery platform that enables selection of high value antibodies to difficult multipass membrane receptors, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, Vaccinex, NOV 13, 2020, View Source [SID1234570906]).

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Pepinemab Clinical Updates:

Huntington’s Disease. The company reported post-hoc analysis of data from the SIGNAL phase 2 clinical trial evaluating our lead drug candidate, pepinemab, for the treatment of Huntington’s disease (HD). While this mid-stage trial did not meet pre-specified co-primary endpoints, the company believes study results show encouraging metrics across several endpoints that provide a strong rationale for continued development in neurodegenerative diseases. In particular, the company believes an important cognitive benefit is reflected in the observed effect of pepinemab on the HD-Cognitive Assessment Battery Composite score (p=0.007), a broad measure of cognitive improvement. The company believes that the treating physicians’ Clinical Global Impression of Change provided a further signal of benefit as fewer pepinemab treated patients experienced deteriorating health status. Finally, the company believes pre-specified imaging analysis of brain in patients with early manifest disease demonstrated a significant treatment-related reduction in normal disease-associated brain atrophy and loss of brain metabolic activity.
Non-Small Cell Lung Cancer (NSCLC). CLASSICAL-Lung Clinical Trial. The recently completed CLASSICAL-Lung study evaluated pepinemab in combination with the anti-PD-L1 checkpoint inhibitor BAVENCIO (avelumab) for the treatment of advanced (stage IIIB/IV) Non-Small Cell Lung Cancer (NSCLC). Near topline data for this trial presented at the virtual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) conference in late May 2020 suggested that combination therapy induces an objective response rate that is a factor of two greater than observed with avelumab alone. The company believes that it is particularly striking that this was seen in the more difficult to treat PD-L1 negative and PD-L1 low population.
Head and Neck Cancer. The company announced a clinical collaboration with Merck, known as MSD outside the US and Canada, to evaluate the combination of pepinemab with Merck’s anti-PD-1 therapy, KEYTRUDA, for the treatment of recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). This cancer indication is notable for the relatively high levels of SEMA4D expression, which has been shown to induce correspondingly high levels of myeloid suppressor cells that block anti-cancer immune responses. Pepinemab is known to have a dual mechanism of action the both increases infiltration and activation of CD8+ cytotoxic T cells and reduces myeloid derived suppressor cells (MDSC). The company believes that HNSCC is, therefore, representative of cancer indications of special interest for treatment with pepinemab in combination with KEYTRUDA. A 65-patient study is planned for H1 2021.
Alzheimer’s Disease. The mechanism of action of pepinemab in slowing cognitive deterioration and brain atrophy that were demonstrated in the SIGNAL study in Huntington’s disease, are believed to be equally applicable to neurodegeneration and cognitive decline associated with Alzheimer’s disease. The company is planning to initiate a Phase 1/2 study of pepinemab in AD in 2021 with partial funding support received from both the Alzheimer’s Association and from the Alzheimer’s Drug Discovery Foundation.
Other Trials. Pepinemab is also being evaluated in multiple investigator-sponsored trials (ISTs) being conducted by the Winship Cancer Institute of Emory University to evaluate pepinemab in combination with checkpoint inhibitors in "Window of Opportunity" studies in colorectal, pancreatic, head and neck cancer and melanoma.
Other Third Quarter Accomplishments:

As summarized above, Maurice Zauderer, Ph.D., president and chief executive officer of Vaccinex, presented additional data from the SIGNAL Huntington’s Disease study at the virtual Huntington’s Study Group (HSG) 2020 Annual Meeting.
The company has entered into several new biotech and major pharma collaborations to employ our novel discovery platform for structural studies and to select high value antibodies against difficult multipass membrane receptors.
Upcoming Anticipated Milestones:

H1 2021 – Planned initiation of a Phase 1b/2 clinical trial of pepinemab in combination with KEYTRUDA for the treatment of patients with HNSCC.
2021 – Planned initiation of Alzheimer’s disease Phase 1/2 study.
Financial Results for the Three Months Ended September 30, 2020:

Research and Development Expenses. Research and development expenses for the three months ended September 30, 2020 were $7.3 million, as compared to $6.6 million for the comparable period in 2019. This increase was driven by higher clinical trial costs as well as an increase in consulting services.

General and Administrative Expenses. General and administrative expenses for the three months ended September 30, 2020 were $1.9 million, as compared to $1.5 million for the comparable period in 2019. The increase was due to increased stock-based compensation as a result of new option awards to employees and board members, as well as increased directors’ and officers’ insurance premiums.

Cash and Cash Equivalents and Marketable Securities. Cash and cash equivalents and marketable securities on September 30, 2020 were $17.1 million. This reflects $12.3 million raised through the company’s existing at-the-market (ATM) equity facility, $8.0 million through the sale of a senior secured convertible debenture, $4.0 million through a private placement transaction, and $300,000 through the company’s existing equity line of credit facility. The company also received $575,000 of the previously announced $750,000 grant from the Alzheimer’s Association under the 2020 Part the Cloud Program.

On November 13, 2020 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing XCART, a personalized CAR T platform technology engineered to target patient- and tumor-specific neoantigens,reported its financial results for the three and nine months ended September 30, 2020 and provided a corporate update (Press release, Xenetic Biosciences, NOV 13, 2020, View Source [SID1234570903]).

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"We continue to make progress as we work to advance XCART through preclinical development and into a Phase 1 study as quickly as possible. This includes finalizing the protocol of our upcoming exploratory study in Belarus which will be used to evaluate the XCART process in a clinical setting," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic. "As we proceed through our preclinical development phase, we, along with our scientific advisors, continue to refine our analysis to identify which NHL patient populations would most benefit from XCART therapy, including those who can no longer be treated effectively with CD19 targeting therapeutics due to loss of the CD19 antigen."

XCART Platform Technology Overview: Significantly differentiated, proprietary approach to personalized CAR T therapy for the treatment of multiple tumor types of B-cell Non-Hodgkin lymphomas, an area of significant unmet need, with the potential to address an initial global market opportunity of over $5 billion annually.[1] Xenetic believes XCART has the potential to transform CAR T therapy.

Program Highlights:

Collaboration with Pharmsynthez and multiple academic institutions in Russia and Belarus to optimize the overall XCART workflow, including clinical manufacturing processes, and to ultimately dose B-cell non-Hodgkin lymphoma (NHL) patients.
Research and development collaboration with Scripps Research covering design and implementation of the preclinical development program, as well as method development activities supporting process development for clinical manufacturing.
PolyXen Platform Technology: Patent-protected platform technology designed for protein or peptide therapeutics, enabling next-generation biological drugs by prolonging a drug’s circulating half-life and potentially improving other pharmacological properties.

Program Highlights:

Exclusive License Agreement with Takeda Pharmaceuticals Co. Ltd. ("Takeda") in the field of coagulation disorders. Takeda currently has one active development program underway utilizing the PolyXen platform technology.
Royalty payments of approximately $0.3 million received in the first nine months of 2020 as the relevant product has now launched worldwide and continues to be rolled out by Takeda’s sublicensee.
Summary of Financial Results for Third Quarter 2020
Net loss for the nine months ended September 30, 2020 was approximately $9.7 million compared to a net loss of approximately $11.6 million for the same period in 2019. The results for the nine months ended September 30, 2020 included $6.3 million of non-cash expenses representing the impairment of indefinite-lived intangibles of $9.2 million less a $2.9 million income tax benefit. The results for the nine months ended September 30, 2019 included $6.3 million of non-cash expenses composed of in-process research and development expenses of $3.0 million and Goodwill impairment of $3.3 million, as well as $1.1 million of transactions costs related to our acquisition of XCART. Excluding the non-cash charges of $6.3 million for both the nine months ended September 30, 2020 and 2019, respectively, and the $1.1 million of transaction costs in 2019, adjusted net loss was $3.4 million and $4.2 million for the nine months ended September 30, 2020 and 2019, respectively. As of September 30, 2020, working capital was $7.2 million compared to $9.7 million as of December 31, 2019. The decrease in working capital was primarily due to the Company’s net loss for the nine months ended September 30, 2020. The Company ended the quarter with approximately $7.1 million of cash.

Non-GAAP Measures
Adjusted net loss is a Non-GAAP financial measure, which is utilized by management in comparing our operating performance on a consistent basis. We present this non-GAAP financial measure because we believe such measure provides important supplemental information to management and investors regarding financial and business trends relating to the Company’s financial condition and results of operations. Non-GAAP financial measures should not be considered in isolation from, or as a substitute for, financial information calculated in accordance with GAAP.

On November 13, 2020 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Phesgo, a fixed-dose combination of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase, administered by subcutaneous (SC; under the skin) injection in combination with intravenous (IV) chemotherapy, for the treatment of early and metastatic HER2-positive breast cancer (Press release, Hoffmann-La Roche, NOV 13, 2020, View Source [SID1234570902]). Based on this recommendation, a final decision regarding the approval of Phesgo is expected from the European Commission in the near future.

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"Our commitment to transforming the lives of people with breast cancer goes beyond improving efficacy outcomes," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. "Today’s recommendation is another important step forward in redefining the standard of care for people with HER2-positive breast cancer in Europe by potentially offering a faster and less invasive way to receive treatment with Perjeta and Herceptin."

SC administration of Phesgo takes approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose.1 This is compared to approximately 150 minutes for infusion of a loading dose of Perjeta and Herceptin using the standard IV formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines.2,3

The recommendation from the CHMP is based on results from the pivotal phase III FeDeriCa study, which showed that treatment with Phesgo produced non-inferior levels of Perjeta and Herceptin in the blood when compared to IV administration of the two medicines. The safety profile of Phesgo with chemotherapy was comparable to IV administration of Perjeta plus Herceptin and chemotherapy, and no new safety signals were identified, including no meaningful difference in cardiac toxicity. The most common adverse events in both arms were alopecia, nausea, diarrhoea and anaemia.1,7

The U.S. Food and Drug Administration recently expedited the approval of Phesgo for the treatment of early and metastatic HER2-positive breast cancer. Based on the decision of the treating physician and the preference of the patient, it can be administered by a healthcare professional in a treatment centre or in a patient’s home.

The Herceptin SC vial is approved for the treatment of HER2-positive breast cancer in more than 100 countries worldwide and provides a convenient treatment option for patients and cost-savings for healthcare systems.5,6 Phesgo is another step forward in highlighting Roche’s commitment to improving patients’ experience of cancer treatment, looking beyond efficacy outcomes and focusing on more flexible treatment solutions.

About the FeDeriCa study
FeDeriCa is an international, multi-centre, two-arm, randomised, open-label, pivotal phase III study evaluating the pharmacokinetics, efficacy and safety of subcutaneous injection of Phesgo in combination with chemotherapy, compared with standard intravenous (IV) infusions of Perjeta and Herceptin in combination with chemotherapy, in 500 people with HER2-positive early breast cancer treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings. The primary endpoint of the study is minimum levels of Perjeta in the blood during a given dosing interval (Ctrough), when compared to IV administration of Perjeta. Secondary endpoints include safety; minimum levels of Herceptin in the blood during a given dosing interval (Ctrough); and total pathological complete response, meaning there is no tumour tissue detectable in the tissue removed at the time of surgery.

Data from the FeDeriCa study were presented at the San Antonio Breast Cancer Symposium in December 2019. The FeDeriCa study met its primary endpoint of non-inferior levels of Perjeta in the blood. The geometric mean ratio (GMR; a type of average used when assessing pharmacokinetics) for the primary endpoint was 1.22 (90% CI: 1.14 to 1.31), with the lower limit of the 90% CI of the GMR=1.14≥0.80 (the pre-specified non-inferiority margin). A secondary endpoint of non-inferior levels of Herceptin was also met, with blood concentrations for people receiving Phesgo non-inferior to those receiving IV Herceptin (GMR=1.33 [90% CI: 1.24 to 1.43]; lower limit of 90% CI of GMR=1.24≥0.80). A non-inferiority endpoint was chosen for the study to ensure that people were receiving sufficient dosing with Perjeta and Herceptin as compared to the established IV doses at the same treatment intervals.7,8

About Phesgo
Phesgo is a new fixed-dose subcutaneous (SC) formulation that combines the same monoclonal antibodies as Perjeta and Herceptin with Halozyme Therapeutics’ Enhanze drug delivery technology.1,9 This is the first time that Roche has combined two monoclonal antibodies that can be administered by a single SC injection.

Halozyme’s Enhanze drug delivery technology may enable and optimise SC drug delivery for appropriate co-administered therapeutics. The technology is based on a proprietary recombinant human hyaluronidase PH20 (rHuPH20), an enzyme that temporarily degrades hyaluronan – a glycosaminoglycan or chain of natural sugars in the body – to aid in the dispersion and absorption of other injected therapeutic drugs.9

Pertuzumab in Phesgo is the same monoclonal antibody as in intravenous (IV) Perjeta, and trastuzumab in Phesgo is the same monoclonal antibody as in IV Herceptin. The mechanisms of action of Perjeta and Herceptin are believed to complement each other as both bind to the HER2 receptor, but in different locations. The combination of Perjeta and Herceptin is thought to provide a more comprehensive, dual blockade of the HER signalling pathways.10,11

Phesgo is approved in the US for the treatment of early and metastatic HER2-positive breast cancer. The approved indications for Phesgo mirror those of Perjeta.

The standard IV formulation of Perjeta in combination with IV Herceptin and chemotherapy (the Perjeta-based regimen) is approved in over 100 countries for the treatment of both early and metastatic HER2-positive breast cancer. In the neoadjuvant (before surgery) early breast cancer (eBC) setting, the Perjeta-based regimen has been shown to almost double the rate of pathological complete response compared to Herceptin and chemotherapy.12 Additionally, the combination has been shown to significantly reduce the risk of recurrence of invasive disease or death in the adjuvant (after surgery) eBC setting.13 In the metastatic setting, the combination has shown an unprecedented survival benefit in previously untreated (first-line) patients with HER2-positive metastatic breast cancer.14

About Roche’s medicines for HER2-positive breast cancer
Roche has been leading research into the HER2 pathway for over 30 years and is committed to improving the health, quality of life and survival of people with both early and metastatic HER2-positive disease. HER2-positive breast cancer is a particularly aggressive form of the disease that affects approximately 25-30% of patients.15 Roche has developed three innovative medicines that have helped transform the treatment of HER2-positive breast cancer: Herceptin, Perjeta and Kadcyla (trastuzumab emtansine). Eligibility for treatment with Roche’s HER2-targeted medicines is determined via a diagnostic test which identifies people who will likely benefit from these medicines at the onset of their disease.