On November 9, 2021 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, reported financial results for the quarter ended September 30, 2021 (Press release, Janux Therapeutics, NOV 9, 2021, View Source [SID1234594899]).

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"In the third quarter, we continued to utilize our novel TRACTr and TRACIr platforms to advance our pipeline of next generation immunotherapies. We remain on-track to execute key milestones, including two IND submissions next year for our PSMA-TRACTr and EGFR-TRACTr candidates," said David Campbell, Ph.D., President and CEO of Janux. "Further, we welcomed Dr. Ron Barrett and Alana McNulty to Janux’s Board of Directors. These two key leadership additions bring important expertise to the company as we work to advance our programs towards the clinic."

RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES:

TRACTr product candidates advancing as planned. Janux’s lead TRACTr programs of next-generation T cell engagers remain on-track.

In the first half of 2022, Janux expects to submit an Investigational New Drug (IND) application for its PSMA-TRACTr candidate, targeting prostate-specific membrane antigen (PSMA).
In the second half of 2022, Janux expects to submit an IND application for its EGFR-TRACTr candidate, targeting epidermal growth factor receptor (EGFR).
In 2023, Janux expects to submit an IND application for its TROP2-TRACTr, targeting trophoblast cell surface antigen 2 (TROP2).
On track for selection of TRACIr development candidate in 2022 as planned. Janux is applying its TRACIr technology to develop a costimulatory bispecific product candidate against programmed death-ligand 1 (PD-L1) and Cluster of Differentiation 28 (CD28) to further enhance the anti-tumor activity of T cells. This will be Janux’s first program derived from its TRACIr platform.
Strengthened Board of Directors with appointment of Ron Barrett, Ph.D., and Alana McNulty. Dr. Barrett is a scientist entrepreneur with more than 30 years of experience in the biopharmaceutical industry as a co-founder and leader of biopharmaceutical companies and has been responsible for advancing research that led to FDA approval of three drugs. Ms. McNulty has more than 30 years of experience in finance and business development for private and publicly traded biopharmaceutical companies, playing a key role over her career in a breadth of strategic transactions.
THIRD QUARTER 2021 FINANCIAL HIGHLIGHTS:

Cash and cash equivalents and short-term investments: As of September 30, 2021, Janux reported cash and cash equivalents and short-term investments of $387.5 million, compared to $7.8 million at December 31, 2020.
Research and development expenses: Research and development expenses for the quarter ended September 30, 2021 were $8.4 million, compared to $0.8 million for the comparable period in 2020. The increase in research and development expenses in 2021 was primarily attributable to the development of Janux’s platform technologies and programs. Janux also incurred additional personnel-related expenses, including stock-based compensation, as operations grew in support of program advances.
General and administrative expenses: General and administrative expenses for the quarter ended September 30, 2021 were $3.6 million, compared to $0.4 million for the same period in 2020. The increase in general and administrative expenses was primarily attributable to an increase in personnel-related expenses, including stock-based compensation, due to increased headcount in 2021. The increase in general and administrative expenses were also due to an increase in legal fees, professional fees, and other various general and administrative expenses, as Janux now operates as a public company.
Net loss: For the quarter ended September 30, 2021, Janux reported a net loss of $10.8 million, compared to a net loss of $1.3 million for the comparable period in 2020.

On November 9, 2021 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 program updates and financial results for the third quarter ended September 30, 2021 (Press release, Poseida Therapeutics, NOV 9, 2021, View Source [SID1234594915]).

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"The last quarter saw continued progress as Poseida reached multiple key strategic milestones including FDA clearance of our first fully allogeneic CAR-T IND for P-BCMA-ALLO1, the presentation of strong CAR-T data in a solid tumor indication with our P-PSMA-101 program and, shortly after the quarter, the announcement of a strategic collaboration with Takeda focused on non-viral in vivo gene therapy programs utilizing our platform technologies," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida.

"The initiation of our P-BCMA-ALLO1 clinical trial represents the beginning of a long-planned strategic transition to what we believe is the ‘holy grail’ of cell therapy for oncology, a fully allogeneic CAR-T with a fully humanized heavy chain BCMA binder and a high percentage of TSCM cells which we believe are the key to success," continued Ostertag. "While we believe the P-BCMA-101 autologous program has competitive advantages and would be approvable, one long-term strategic benefit of that program has been to inform our highly-differentiated allogeneic approach. With the P-BCMA-ALLO1 clinical program now underway and with very high confidence in our allogeneic platform, we will begin a planned wind down of P-BCMA-101."

Program Updates

BCMA Program
P-BCMA-ALLO1, the Company’s first fully allogeneic CAR-T product candidate, is in development for the treatment of relapsed/refractory multiple myeloma. In August of 2021, the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for P-BCMA-ALLO1. The IND clearance and the start-up of the Phase 1 clinical trial mark the beginning of the Company’s strategic shift toward focusing on P-BCMA-ALLO1 rather than the autologous P-BCMA-101 program.

While data in the autologous trial showed meaningful responses and a favorable safety profile, the Company’s strategic focus has long been on allogeneic CAR-T therapies, leveraging the learnings of the autologous CAR-T program to provide benefits beyond those of autologous CAR-T, including a more desirable off-the-shelf product profile for future commercialization while maintaining the tolerability advantage of our autologous product candidate. P-BCMA-ALLO1 has the potential to deliver up to hundreds of doses per manufacturing run, thereby dramatically reducing both clinical trial costs and ultimately commercial product cost compared to the autologous P-BCMA-101 program.

PSMA Program
P-PSMA-101 is a solid tumor autologous CAR-T product candidate being developed to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) currently in an ongoing Phase 1 dose escalation trial.

In August of 2021, the Company presented preliminary data at the 6th Annual CAR-TCR Summit virtual meeting that demonstrated meaningful patient responses while maintaining a favorable safety and tolerability profile with modest overall rates of CRS and no neurotoxicity observed at low doses. An additional update on the P-PSMA-101 program is expected in the first half of 2022.

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in preclinical development with the potential to treat a wide range of solid tumors, including breast and ovarian cancers. P-MUC1C-ALLO1 is proceeding, with an anticipated IND filing and initiation of a Phase 1 clinical trial by the end of 2021.

Liver-Directed Gene Therapy Program
P-OTC-101 is the Company’s first liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, a condition characterized by high unmet medical need. The Company is currently evaluating whether to modify the P-OTC-101 program to move to the fully non-viral nanoparticle delivery system. The Company will update expected timing on program advancement once that evaluation is complete.

Other Operational Updates and Upcoming Events

Gene Therapy Research Collaboration with Takeda
In October of 2021, the Company entered into a collaboration and license agreement with Takeda Pharmaceuticals USA, Inc. to utilize Poseida’s proprietary genetic engineering platforms for the research and development of up to eight gene therapies.

The collaboration will focus on developing non-viral in vivo gene therapy programs, including Poseida’s Hemophilia A program. The Company will receive an upfront payment of $45.0 million, of which $5.0 million is for prepaid research. Per the agreement, Takeda will fund all ongoing partnered program research performed by Poseida. The collaboration may utilize all of Poseida’s novel genetic engineering platform technologies, including the piggyBac DNA Modification System for gene addition, the Cas-CLOVER Site-specific Gene Editing System for ultra-precise gene editing, biodegradable nanoparticle technologies for gene delivery and other emerging technologies. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.

GMP Facility
In the third quarter, the Company completed qualification and commenced GMP activity in its internal pilot manufacturing plant. The pilot plant is designed to support and speed the development of allogeneic product candidates including manufacturing clinical material for early-stage trials. The first product candidate to be produced out of the pilot plant will be P-MUC1C-ALLO1.

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 36th Annual Meeting
The Company will give two virtual poster presentations at the upcoming SITC (Free SITC Whitepaper) Annual Meeting, being held in Washington, D.C., and virtually November 10-14, 2021. The full abstracts were made available on the SITC (Free SITC Whitepaper) website earlier today, with presentations taking place on November 12, 2021.

63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition
The Company will present data from the Company’s differentiated P-BCMA-101 autologous CAR-T program including how it has informed the P-BCMA-ALLO1 allogeneic CAR-T program in a poster publication at the ASH (Free ASH Whitepaper) Annual Meeting on Monday, December 13, 2021:

Session Name: 704. Cellular Immunotherapies: Clinical: Poster III
Presentation Time: 6:00 PM – 8:00 PM Eastern time
Location: Georgia World Congress Center, Hall B5

Financial Results for the Third Quarter 2021

Research and Development Expenses
Research and development expenses were $32.5 million for the third quarter ended September 30, 2021, compared to $27.0 million for the same period in 2020. For the nine months ended September 30, 2021, research and development expenses were $97.6 million, compared to $75.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, external costs related to our preclinical programs and clinical stage programs, including the ongoing enrollment and manufacturing associated with our P-BCMA-101, P-BCMA-ALLO1 and P-PSMA-101 clinical trials, and internal costs related to facilities development.

General and Administrative Expenses
General and administrative expenses were $9.1 million for the third quarter ended September 30, 2021, compared to $6.5 million for the same period in 2020. General and administrative expenses were $26.3 million for the nine months ended September 30, 2021, compared to $15.6 million for the same period in 2020. The increase was primarily due to increased stock-based compensation expense, headcount, insurance costs and professional fees.

Net Loss
Net loss was $42.4 million and $126.4 million for the three and nine months ended September 30, 2021, respectively, and $34.4 million and $93.6 million for the three and nine months ended September 30, 2020, respectively.

Cash Position
As of September 30, 2021, our cash and cash equivalents balance was $197.8 million.

On November 9, 2021 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported financial results for the third quarter ended September 30, 2021 and provided an update on clinical and corporate developments (Press release, Fusion Pharmaceuticals, NOV 9, 2021, View Source [SID1234594931]).

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Clinical Update

FPI-1434

Fusion now expects to provide multi-dose safety and imaging data, and recommended Phase 2 dose, in the second half of 2022 rather than in the first half of 2022. The shift in timelines is primarily associated with a decrease in patient enrollment rates attributable to the impact of the COVID-19 pandemic.

Chief Executive Officer John Valliant, Ph.D. commented, "Despite our efforts to mitigate the impacts of the COVID-19 pandemic, including the addition of new trial sites in multiple geographic regions, we have seen patient enrollment rates decline largely due to resourcing and reduced staffing issues at trial sites. Longer timelines to enroll patients have persisted and therefore we are shifting our expectation for multi-dose data to the second half of 2022. We continue to make patient recruitment a top priority, are working closely with our trial sites to support their efforts and believe that with increased vaccination rates recruitment will return to previous levels."

Fusion continues to anticipate the initiation of a Phase 1 combination study with FPI-1434 and KEYTRUDA (pembrolizumab) to occur six to nine months following determination of the recommended Phase 2 dose of FPI-1434 monotherapy. In addition, the study arm exploring the impact on biodistribution and tumor uptake of administration of a dose of naked ("cold") IGF-1R antibody prior to each dose of FPI-1434 is ongoing.

Dr. Valliant continued, "Although the Phase 1 study of FPI-1434 is experiencing delays during these unprecedented times, we continue to believe in the significant opportunity we have to address a broad array of tumor types with high unmet need and we are working diligently to optimize the development program and progress the study for the benefit of patients."

FPI-1966

The Phase 1, non-randomized, open-label clinical trial of FPI-1966 in patients with solid tumors expressing FGFR3, intended to investigate safety, tolerability and pharmacokinetics and to establish the recommended Phase 2 dose, has been initiated with the first study site open to recruitment. Fusion expects to dose the first patient in the first quarter of 2022 and expects interim data from the first patient cohort in the first quarter of 2023.

FPI-2059

FPI-2059 is a small molecule radioconjugate in development as a targeted alpha therapy for various solid tumors. The molecule targets neurotensin receptor 1 (NTSR1), a promising target for cancer treatment, that is overexpressed in multiple solid tumors. FPI-2059 combines Ipsen’s IPN-1087, which Fusion acquired in 2021, with actinium-225. Fusion continues to anticipate submitting an IND application for FPI-2059 in the first half of 2022.

Recent News and Highlights

In August, Fusion and TRIUMF, Canada’s particle accelerator centre, announced that the companies have entered into the next phase of their collaboration agreement for the development, production, and supply of actinium-225. Fusion will provide to TRIUMF funding to further develop technology to produce actinium-225 and in return Fusion will have rights, including preferred access and pricing, to the resulting alpha-emitting medical isotope.
Fusion recently expanded its executive leadership team with the appointments of Mohit Rawat as president and chief business officer, and Christopher Leamon, Ph.D. as chief scientific officer, and Eric S. Hoffman, Ph.D. as senior vice president, business development.
In October, Fusion announced the presentation of preclinical data that provide further support of its FPI-1966 and FPI-2059 targeted alpha therapies (TATs) at the 34th Annual European Association of Nuclear Medicine Congress. These data reinforce the clinical dosing regimen of FPI-1966 and highlight the potential of FPI-2059 as an actinium-225 labelled precision medicine targeting NTSR1.
Dr. Valliant said, "We are building a fully integrated radiopharmaceutical company based upon our platform, reflecting a diverse pipeline of TATs in development. We are also making strategic investments in critical areas of manufacturing and supply chain to support the growth of our business, and we are attracting top industry leaders to join Fusion in our mission to impact the cancer therapy landscape with this new generation of targeted radiopharmaceuticals."

Third Quarter 2021 Financial Results

Cash and Investments: As of September 30, 2021, Fusion held cash, cash equivalents and investments of $238.2 million, compared to cash, cash equivalents and investments of $299.5 million as of December 31, 2020. Fusion expects its cash, cash equivalents and investments as of September 30, 2021 will enable the Company to fund its operations through the end of 2023.
Collaboration Revenue: For the third quarter of 2021, Fusion recorded $0.3 million of revenue under the AstraZeneca collaboration agreement.
R&D Expenses: Research and development expenses for the third quarter of 2021 were $12.7 million, compared to $4.5 million for the same period in 2020. The increase was primarily related to costs associated with the FPI-1434 Phase 1 clinical trial, as well as preclinical research and manufacturing costs, platform development and personnel-related costs.
G&A Expenses: General and administrative expenses for the third quarter of 2021 were $7.2 million, compared to $5.8 million for the same period in 2020. The increase was primarily related to personnel-related costs and general corporate costs, partially offset by a decrease in professional fees.
Net Loss: For the third quarter of 2021, Fusion reported a net loss of $19.4 million, or $0.45 per share, compared with a net loss of $10.0 million, or $0.24 per share, for the same period in 2020.
Impact of COVID-19

Fusion is experiencing material delays in patient recruitment and enrollment as a result of continued resourcing issues related to COVID-19 at trial sites.

There also remains uncertainty relating to the trajectory of the pandemic, hospital staffing and resource issues, and whether they may cause further delays in patient study recruitment. The impact of related responses and disruptions caused by the COVID-19 pandemic may result in further difficulties or delays in initiating, enrolling, conducting or completing the planned and ongoing trials and the incurrence of unforeseen costs as a result of disruptions in clinical supply or preclinical study or clinical trial delays. The continued impact of COVID-19 on results will largely depend on future developments, which are highly uncertain and cannot be predicted with confidence.

On November 9, 2021 Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, reported the publication of a study showing that the addition of Switch-Blocker technology to common PCR-based liquid biopsy assays significantly increased sensitivity in detecting rare cancer mutations (Press release, Biocept, NOV 9, 2021, View Source [SID1234594947]). The abstract was published in the November 2021 issue of the Journal of Molecular Diagnostics.

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Biocept’s proprietary Switch-Blocker technology enriches oncogenic mutations of interest while suppressing wild-type (normal) DNA, resulting in ultra-high sensitivity, specificity and accuracy. In this study, Switch-Blockers were combined with conventional real-time PCR and droplet digital PCR (ddPCR) assays.

"Our quantitative Switch-Blocker technology demonstrates an unprecedented ability to find and distinguish extremely rare genetic events—even in blood that contains mostly DNA from normal white blood cells," said Michael Dugan, Chief Medical Officer and Medical Director of Biocept. "This can greatly enhance the clinical sensitivity of our cell-free tumor DNA assays and has broad application in the continued development of highly sensitive and quantitative molecular diagnostic assays used to evaluate cerebrospinal fluid or blood from patients with cancer. Switch-Blocker-based assays can help detect cancer biomarkers that otherwise might be missed, improving treatment selection. They can also be used to evaluate treatment-related changes, find minimal residual disease or identify early disease recurrence."

Results showed that the addition of Switch-Blockers increased the sensitivity of allele-specific primer assays by more than 200 times, from about 1% minor allele frequency (MAF) to better than 0.01%. The sensitivity of multiplex competitive allele-specific TaqMan assays, commonly used with PCR amplification, were increased greater than 1,000 times, from about 10% MAF to 0.01% or better. The ability to significantly increase the sensitivity of conventional mutation assays using Switch-Blocker technology is critical for helping to find rare genetic events in a wide range of applications, including solid tumor cancers, where a majority of biomarkers in blood occur at less than 1% MAF.

The abstract (#TT33), titled "The Use of Switch-Blocker Probes for the Ultra-High Sensitivity of Detection of Rare Genetic Events Using Conventional Real-Time and Droplet Digital PCR Assays," can be accessed here.

About Switch-Blocker Technology

Biocept’s proprietary Switch-Blocker platform is the basis for the company’s Target Selector assays and can be used with tissue, blood and cerebrospinal fluid (CSF) samples. The technology enables industry-leading sensitivity for the detection of mutations/variants from circulating tumor DNA (ctDNA). It has been validated to 0.05% minor allele frequency in blood, which provides significant advantages for identifying actionable cancer biomarkers and assessing therapeutic tumor response. Switch-Blockers enhance the performance and specificity of the PCR method, the most widely used amplification approach for clinical diagnostic applications and can be customized to aid in biopharmaceutical research for the development of targeted therapies for cancer. Switch-Blocker technology also has been validated and found to be highly sensitive, quantitative and reproducible in detecting the presence of the SARS-CoV-2 virus that causes COVID-19 infections.

On November 9, 2021 Ascendis Pharma A/S (Nasdaq: ASND) reported two poster presentations featuring new non-clinical data for its investigational TransCon TLR7/8 Agonist product candidate at SITC (Free SITC Whitepaper) 2021, the annual meeting for the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) taking place virtually and in person November 10-14 in Washington, D.C (Press release, Ascendis Pharma, NOV 9, 2021, View Source [SID1234594982]).

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The data show that, as designed, TransCon TLR7/8 Agonist, which leverages the Company’s innovative TransCon hydrogel technology, provides sustained activation of both innate and adaptive immune mechanisms with low systemic cytokine levels.

The posters being presented at SITC (Free SITC Whitepaper) 2021 are:

Poster #769
Friday,
November 12 A Single Dose of Intratumoral TransCon TLR7/8 Agonist Monotherapy Promoted Sustained Activation of Antigen Presenting Cells Resulting in CD4+ and CD8+
T Cell Activation and Tumor Growth Inhibition
Poster #16
Saturday,
November 13
Tumor Growth Inhibition Mediated by a Single Dose of Intratumoral TransCon TLR7/8 Agonist Associated with Activated Circulating T and B cells and Sustained Low Levels of Systemic Cytokines

"We are designing TransCon TLR7/8 Agonist for sustained and controlled release of resiquimod, a potent TLR7/8 agonist, with the goal of maximizing therapeutic benefit and addressing the known limitations of current approaches, including serious systemic toxicity and rapid effusion from the tumor," said Juha Punnonen, Ascendis Pharma’s Senior Vice President and Head of Oncology. "We are incredibly pleased to confirm with these non-clinical studies that a single intratumoral injection of sustained release unmodified resiquimod delivered through our unique hydrogel technology worked in the expected way. We look forward to sharing initial data from our Phase 1/2 clinical trial soon."

About TransCon TLR7/8 Agonist
TransCon TLR7/8 Agonist is an investigational long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8 designed to provide sustained activation of intratumoral antigen-presenting cells driving tumor antigen presentation and induction of immune stimulatory cytokines for weeks or months with a single intratumoral injection.

Ascendis Pharma is currently conducting a Phase 1/2 study, called the transcendIT-101 Study (ClinicalTrials.gov Identifier: NCT04799054), to evaluate TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab in dose escalation and dose expansion cohorts. The primary objectives are to evaluate safety and tolerability and to define the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of TransCon TLR7/8 Agonist alone or in combination with pembrolizumab.