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On October 28, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported that it will participate in the 2021 Alliance Global Partners Biotech & Specialty Pharma Conference taking place November 10, 2021. Diffusion will also be participating in one-on-one meetings (Press release, Diffusion Pharmaceuticals, OCT 28, 2021, View Source [SID1234592095]).

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Interested investors can schedule meetings with management from 8:30 a.m. to 5:00 p.m. on November 10. Those who wish to request a meeting with Diffusion should contact their Alliance Global Partners salesperson.

On October 28, 2021 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported its interim management statement for the quarter ending September 30, 2021 (Press release, Molecular Partners, OCT 28, 2021, View Source [SID1234592112]).

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"COVID-19 continues to be a major concern globally, and a main focus of our efforts at Molecular Partners. As new variants emerge and novel therapeutic modalities are being developed to fight the disease alongside vaccines, the need to develop efficacious and robust therapeutics is clearer than ever. Our COVID-19 program has significantly advanced in the last quarter. Having now enrolled over 700 patients across two global late stage studies, we have accomplished much and are approaching two significant milestones. In the coming weeks and months we are preparing for futility assessment in the NIH-sponsored hospitalized study and data from our phase 2b-3 EMPATHY study in outpatients," said Patrick Amstutz, Ph.D., Molecular Partners’ CEO. "In addition, we have maintained momentum across our two immuno-oncology clinical programs, and advanced our preclinical AML program, a truly differentiated CD3 engaging molecule with a unique mechanism of action which is beyond the feasibility of most traditional therapies."

Research & development highlights:

Ensovibep COVID-19 antiviral program: Two global studies ongoing
In October 2021, the Phase 2b portion of the EMPATHY (ambulatory) study reached its target recruitment of 400 patients; Topline data from Phase 2b are expected in early 2022
Also in October 2021, the ACTIV-3 (hospitalized) study reached its initial target recruitment of 300 patients. A futility analysis of the study will be conducted at the next data and safety monitoring board (DSMB) assembly, in the coming weeks. Should ensovibep pass the futility analysis, the study will advance to full enrollment. Topline data are expected in 2022
Assessment of a subcutaneous formulation of ensovibep is ongoing in healthy volunteers and will provide the rationale to initiate patient studies in the coming months
Ensovibep continues to maintain full potency in vitro against all known variants of concern, including Delta variants

AMG 506 / MP0310 (FAP x 4-1BB)
Ongoing Phase 1 trials with weekly dosing
Expecting data late in 2021 or early 2022, for Amgen and Molecular Partners’ evaluation

MP0317 (FAP x CD40):
The second immuno-oncology DARPin candidate is expected to enter the clinic in Q4 2021
Strong preclinical data supports MP0317’s potential to deliver tumor-localized immune activation while avoiding systemic toxicity seen with other CD40-targeting agents

MP0533 (CD33 x CD70 x CD123 x CD3)
Molecular Partners’ lead AML targeting candidate selected, formally termed MP0533
Preclinical data to be presented at the ASH (Free ASH Whitepaper) conference, December 2021
Expected to enter clinical trials in 2022

Abicipar:
Molecular Partners regained global rights to abicipar, the Company’s registrational-stage ophthalmology therapeutic candidate for the treatment of neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME)
Molecular Partners is evaluating the program and will determine the appropriate next steps
Operational and financial highlights:

Strong financial position with CHF 154.3 million in cash (incl. short term deposits) as of September 30, 2021
Operating loss of CHF 47.6 million and net loss of CHF 45.9 million for the 9 months ended September 30, 2021
Company funded into H2 2023, excluding any potential payments from R&D partnerships
The Q3 2021 Financial Statements are available on the company’s website
COVID-19 program rapidly advancing in two global registrational trials with Novartis and the NIH

Molecular Partners’ lead infectious disease therapeutic candidate, ensovibep, is currently being evaluated in EMPATHY, a global Phase 2b-3 study designed to explore the use of ensovibep for the treatment of COVID-19 in patients who are in the early stages of infection to prevent worsening symptoms and hospitalization. Molecular Partners’ collaboration partner, Novartis, is conducting the clinical trial for ensovibep, with Molecular Partners as a sponsor. The phase 2b portion of EMPATHY enrolled patients across six countries. Topline data for the first 400 patients are expected in early 2022, allowing for potential EUA submission and full data in 2022.

Ensovibep is additionally being evaluated in ACTIV-3 for the treatment of hospitalized COVID-19 patients as part of the National Institutes of Health’s (NIH) Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) program, which is evaluating multiple therapies for COVID-19 in hospital setting. The Phase 3 study is presently enrolling hospitalized patients globally. The study has surpassed the target enrollment of approximately 300 patients required for the pre-planned interim futility analysis.The futility analysis will be conducted in November 2021 by the Data and Safety Monitoring Board (DSMB) to determine if enrollment in the ensovibep arm of ACTIV-3 may continue to its full enrollment. Topline data are expected in 2022.

In addition to its clinical development, ensovibep continues to be regularly tested in the laboratory for its inhibition of infectivity in newly discovered variants of the virus. As presented at the ISIRV-WHO conference in October 2021, all in vitro data to-date show that ensovibep retains full potency and viral inhibition against all known SARS-CoV-2 variants of concern, including the key Delta variants.

Immuno-oncology: Phase 1 trial of MP0317 (FAP x CD40); ongoing studies of AMG 506 (MP0310); Progress in AML program

MP0317 trial enrollment is expected to take place in the Netherlands and France. Up to 30 patients are expected to be enrolled across six dosing cohorts and up to 15 patients are then expected to be enrolled in a dose expansion cohort. In addition to evaluating monotherapy dynamics, the study will gather a wide variety of biomarker data to support the establishment of combination therapies with MP0317 in specific indications.

Clinical studies of AMG 506 (MP0310) as a treatment for solid tumors are ongoing in collaboration with Amgen with weekly administration to identify a dosing regimen to obtain sustained 4-1BB activation.

MP0533, Molecular Partners’ novel AML candidate, is a DARPin designed to engage CD3 on T cells and target AML cells by the tumor associated antigens CD33, CD70 and CD123, with an addition for half-life extension. The candidate binds to cells by an avidity dependent mechanism, preferentially targeting AML cells, which express two or more of these antigens. These malignant cells are then marked for termination by CD3 T-cell activation. Additional data from MP0533 will be presented at the Company’s R&D day in December 2021 and presenters will include leading AML researchers from the University of Bern. Preclinical data from the new candidate will be presented at the ASH (Free ASH Whitepaper) conference in December 2021, with clinical development expected to be initiated in 2022.

Balance sheet: Strong cash and equity positions as of September 2021

In June 2021, Molecular Partners successfully completed an initial public offering of American Depositary Shares ("ADSs") on the Nasdaq, raising $63.8 million (CHF 58.8 million) in gross proceeds.
Ongoing strong financial position with CHF 154.3 million in cash and short-term deposits as of September 30, 2021
Net cash outflow from operating activities of CHF 71.6 million in the first nine months of 2021
Financial outlook 2021

For the full year 2021, at constant exchange rates, the Company expects total expenses of CHF 70 – 75 million, of which approximately CHF 7 million will be non-cash effective costs.

In terms of cash outflow, the Company expects a gross cash utilization of approximately CHF 90 million for the full year 2021, which includes a total of CHF 20 million payable to Novartis for the manufacturing of commercial supply (of which CHF 14.5 million occurred in the first nine months of 2021). This cash flow guidance does not include any potential receipts from R&D partnerships.

With CHF 154.3 million cash and short-term time deposits and no debt as of September 30, 2021, the Company expects to be funded into H2 2023, excluding any potential receipts from R&D partners.

Financial Calendar

December 15, 2021 R&D Day
March 15, 2022 Expected Publication of FY 2021 Annual report and audited 2021 results
April 13, 2022 Annual General Meeting
About DARPin therapeutics

DARPin therapeutics are a new class of custom-built protein therapeutics based on natural binding proteins that open a new dimension of multi-functionality and multi-target specificity in drug design. A single DARPin candidate can engage more than five targets, and its flexible architecture and small size offer benefits over conventional monoclonal antibodies or other currently available protein therapeutics. DARPin therapeutics have been clinically validated through to registration via the development of abicipar, Molecular Partners’ most advanced DARPin drug candidate. The DARPin platform is a fast and cost-effective drug discovery engine, producing drug candidates with optimized properties for development and very high production yields.

On October 28, 2021 Candel Therapeutics, Inc. (Nasdaq: CADL), a late clinical stage biopharmaceutical company developing novel oncolytic viral immunotherapies, reported that data on patient-reported tolerability assessment of intraprostatic injections will be presented in a virtual poster session at the 28th Annual Prostate Cancer Foundation Scientific Retreat (Press release, Candel Therapeutics, OCT 28, 2021, View Source [SID1234592129]).

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Date: Thursday, October 28, 2021
Presenter: Laura K. Aguilar, MD, PhD, Chief Medical Officer at Candel Therapeutics, Inc.
Presentation Title: Patient experience with intraprostatic injection of CAN-2409 or placebo followed by valacyclovir in a phase 3 clinical trial for localized prostate cancer in combination with standard of care radiation therapy with or without androgen suppression.
The data were generated from the company’s ongoing phase 3 clinical trial, which is evaluating safety and efficacy of intra-prostatic injection with CAN-2409 or placebo followed by oral valacyclovir prodrug in combination with standard of care (radiation therapy ± short-term androgen deprivation therapy) in patients with localized prostate cancer having intermediate-risk or a single NCCN high-risk factor.

Clinical trial enrollment has been completed with a diverse population of 745 patients from 51 sites in the U.S. More than 2,000 injection procedures have been performed (40% transperineal, 56% transrectal, 4% not reported). Information on the patient experience is being collected with a questionnaire, and data is available from 32 patients who completed the questionnaire within 3 months of completing treatment. For the transperineal procedure, 65% of patients reported the intra-prostatic injections to be "the same or better" tolerated than a prostate biopsy, 30% "a little harder" to tolerate and 4% "much harder" to tolerate than a biopsy. For the transrectal procedure, 89% of patients reported the injections to be "the same or better" tolerated than a biopsy and 11% "a little harder" to tolerate than a biopsy. All patients (100%) reported overall feeling positive about their involvement in the study.

"These data further support the tolerability and patient receptiveness of intra-prostatic injection of CAN-2409 and its comparability to routine biopsies being performed in this patient population," said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel Therapeutics. "Listening to patients is important as we look towards the commercialization of CAN-2409, if approved. We believe CAN-2409 holds great promise as a treatment for patients with localized, non-metastatic prostate cancer, which may improve disease outcome, while eliminating the need for long-term androgen deprivation therapy and its associated side effects."

Details from the presentations will be available on the Candel website at View Source

About CAN-2409

CAN-2409, Candel’s most advanced oncolytic viral immunotherapy candidate, is a replication-deficient adenovirus that delivers the herpes simplex virus thymidine kinase (HSV-tk) gene to cancer cells. HSV-tk is an enzyme that locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells. The intra-tumoral administration results in the release of tumor-specific neoantigens in the microenvironment. At the same time, the adenoviral serotype 5 capsid protein elicits a strong pro-inflammatory signal in the tumor microenvironment. This creates the optimal conditions to induce a CD8+ T cell mediated response against the injected tumor and uninjected distant metastases for broad anti-tumor activity.

Because of its versatility, CAN-2409 has the potential to treat a broad range of solid tumors. Monotherapy activity as well as combination activity with standard of care radiotherapy, surgery, chemotherapy, and immune checkpoint inhibitors have previously been shown in several preclinical and clinical settings. Furthermore, CAN-2409 presents a favorable tolerability profile; more than 700 patients have been dosed to date, supporting the potential for combination with other therapeutic strategies without inordinate concern of overlapping adverse events. Currently, Candel is evaluating the effects of treatment with CAN-2409 in localized, non-metastatic prostate cancer, non-small cell lung cancer, high-grade glioma, and pancreatic cancer in ongoing clinical trials.

About the Phase 3 Clinical Trial in Prostate Cancer

The pivotal phase 3 study of CAN-2409 immunotherapy in patients with intermediate-high risk localized prostate cancer is a placebo-controlled, randomized clinical trial to evaluate CAN-2409 treatment in combination with valacyclovir added to standard of care (radiation therapy ± short-term androgen deprivation therapy). The primary endpoint of the study is disease-free survival. Secondary endpoints include prostate cancer specific survival, overall survival, freedom from biochemical failure, patient reported health-related quality of life, and safety.

On October 28, 2021 Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM), a clinical stage biopharmaceutical company using an expanded genetic code technology platform to discover and develop Engineered Precision Biologics (EPBs), today provided a corporate update and reported financial results for the first half of 2021 (Press release, Ambrx, OCT 28, 2021, View Source [SID1234592145]).

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"I want to thank the entire Ambrx team for their efforts in making our June IPO such a success. I believe we have created a strong foundation on which to grow our company. Subsequently, we provided several clinical updates regarding our ARX788 program in gastric cancer and our ARX517 program in PSMA expressing tumors. For ARX788, the first patient was dosed in our co-sponsored ACE-Gastric-02 Phase 2/3 clinical study and updated positive data from our co-sponsored ACE-Gastric-01 study was presented at The Chinese Society of Clinical Oncology (CSCO) annual meeting. We were also able to dose the first patient in our Phase 1 trial of ARX517 in the United States," said Feng Tian, Ph.D., Chairman of the Board, President and CEO of Ambrx. "Additionally, during the period, Ambrx strengthened its leadership team with the formation of a Scientific Advisory Board, the appointment of Sonja Nelson as CFO, and the addition to our Board of Directors of Olivia Ware and Katrin Rupalla. I am proud of how far Ambrx has come and look forward to carrying forward the momentum we have built into the end of the year and beyond."

1H 2021 and Subsequent Highlights

Positive Data on ARX788 for the Treatment of HER2+ Gastric Cancer Presented at CSCO. In October, NovoCodex Pharmaceuticals Ltd., Ambrx’s partner in China, presented positive interim data from the ACE-Gastric-01 Phase 1 clinical study of ARX788 for the treatment of HER2+ metastatic gastric / gastroesophageal junction (GEJ) cancer at CSCO. The data presented added to and updated initial trial data presented by both companies at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2021.
Advanced the Development of ARX788 in Three Trials. Subsequent to the end of first half of the year, Ambrx dosed the first patient in a global Phase 2/3 clinical study of ARX788 (ACE-Gastric-02) in patients with HER2+ gastric/GEJ cancer, initiated a global Phase 2 basket clinical study of ARX788 (ACE-Pan tumor-02) for HER2-mutated or HER2-amplified/over-expressed solid tumors, and initiated a Phase 2 clinical study of ARX788 (ACE-Breast-03) in patients with HER2+ metastatic breast cancer.
First Patient Dosed in a Phase 1 Trial for ARX517. In August, Ambrx announced that the first patient had been dosed in a Phase 1, multicenter, dose-escalation, and dose expansion study to evaluate the safety, pharmacokinetics, and anti-tumor activity of ARX517, an ADC being developed to treat subjects with prostate specific membrane antigen (PSMA) expressing tumors.
BMS Provided Update on FA Relaxin Program. In August, BMS provided notice that it closed the FA Relaxin Studies CV019-008 and CV019-010 based on internal considerations and not upon any observed safety concerns.
Completed Successful Initial Public Offering. In June, Ambrx successfully completed its initial public offering raising $126.0 million in gross proceeds prior to deducting underwriting discounts, commissions and offering expenses. Ambrx’s American Depository Shares (ADSs) began trading on the New York Stock Exchange under the symbol "AMAM" on June 18, 2021. In addition, in early July, the underwriters partially exercised their greenshoe option resulting in additional gross proceeds to Ambrx of approximately $16.1 million prior to deducting underwriting discounts, commissions and offering expenses.
Reorganization. During the second quarter of 2021, Ambrx completed a reorganization of its corporate structure. As a result, Ambrx Shanghai and Ambrx Hong Kong became wholly owned subsidiaries of Ambrx Biopharma Inc.
Formed a Scientific Advisory Board (SAB). In June, Ambrx announced the formation of a Scientific Advisory Board comprised of industry leaders in oncology and protein sciences. The board is chaired by Peter Schultz, Ph.D., President and CEO of Scripps Research and a professor of chemistry. The other founding members of the Ambrx SAB include: Stuart Lutzker, M.D., Ph.D., an industry veteran with decades of oncology drug development experience; Jakob Dupont, M.D., a renowned expert in oncology, immunology and cell therapy; Melissa Starovasnik, Ph.D., an accomplished scientific leader and 28-year Genentech veteran; Luisa Salter-Cid, Ph.D., M.Sc., the Chief Scientific Officer at Pioneering Medicines; and Feng Tian, Ph.D., the Chairman of the Board of Directors, President and CEO of Ambrx.
Appointed Sonja Nelson as CFO. In June, Sonja Nelson joined Ambrx as its Chief Financial Officer. Ms. Nelson is an experienced financial executive, who joined Ambrx with 10 years of executive experience within the biotechnology industry. Most recently, she served as Chief Financial Officer at NantKwest, now ImmunityBio.
Strengthened Board of Directors with Two New Appointments. In April, Ambrx appointed Olivia C. Ware, an accomplished biotech and pharmaceutical executive, and Katrin Rupalla, Ph.D., Senior Vice President of Regulatory Affairs, R&D Quality and Medical Information at Lundbeck, to its Board of Directors.
Anticipated Near-Term Milestones

Additional data from the Phase 1 clinical study of ARX788 (ACE-Breast-01) in patients with HER2+ metastatic breast cancer in 2H 2021.
Submit an Investigational New Drug (IND) application for ARX305 in patients with cancer (renal cell carcinoma (RCC) and other cancers) in 1H 2022.
Financial Highlights

Cash and Cash Equivalents: Cash and cash equivalents were $167.2 million as of June 30, 2021. In July, the underwriters partially exercised the greenshoe option resulting in net proceeds of $14.9 million to Ambrx.
Revenue: Revenue was $5.1 million for the six months ended June 30, 2021, as compared to $6.5 million for the six months ended June 30, 2020. The decrease was primarily driven by revenue recognized associated with upfront payments related to our R&D agreements, partially offset by increased 3rd party reimbursable charges.
Research and development (R&D) expenses: R&D expenses were $22.1 million for the six months ended June 30, 2021, as compared to $9.9 million for the six months ended June 30, 2020. The increase of $12.2 million was mainly due to increased costs related to new clinical trial programs and related manufacturing costs as well as personnel related costs including stock-based compensation expense.
General and administrative (G&A) expenses: G&A expenses were $8.4 million for the six months ended June 30, 2021, as compared to $2.6 million for the six months ended June 30, 2020. The increase of $5.8 million was mainly attributable to professional services and fees in connection with preparing for Ambrx’s IPO, expenses associated with the corporate structure reorganization, and personnel related costs including stock-based compensation expense.
Other expenses: Other expense, net, for the six months ended June 30, 2021 was $3.9 million, which is primarily due to a change in fair value of Ambrx’s redeemable noncontrolling interest liability in connection with the purchase of shares held by minority shareholders in Ambrx Shanghai which was part of Ambrx’s reorganization of its corporate structure, completed during the second quarter of 2021.
Net loss: Net loss for the six months ended June 30, 2021 was $29.0 million, as compared to $5.4 million for the six months ended June 30, 2020.