On September 16, 2020 Oasmia Pharmaceutical’s CEO, Dr Francois Martelet reported that it will present at the 20th Annual Biotech in Europe Forum from 21 – 24 September organised by Sachs Associates (Press release, Oasmia, SEP 16, 2020, View Source [SID1234565236]). The presentation will be available on Oasmia Pharmaceutical’s new website from Monday, September 21.

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The 20th Annual Biotech in Europe Forum is recognized as the leading international stage for those interested in investing and partnering in the biotech and life science

On September 16, 2020 Poseida Therapeutics, Inc., (Nasdaq: PSTX), a clinical-stage biopharmaceutical company dedicated to utilizing proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure, reported that it will present data related to its proprietary manufacturing process designed to optimize its CAR-T product candidates (Press release, Poseida Therapeutics, SEP 16, 2020, View Source [SID1234565235]). The Company will also illustrate the impact of these optimizations with preclinical data and preliminary clinical analysis with a focus on P-BCMA-101, its autologous CAR-T product candidate for multiple myeloma. The findings will be presented today at CAR-TCR Digital Week 2020 being held September 14-17, 2020.

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Utilizing its proprietary piggyBac DNA Modification System, Poseida’s non-viral manufacturing process can produce highly purified CAR-T treatment candidates comprised of a high percentage of stem cell memory T, or TSCM, cells. These high-TSCM product candidates may improve therapeutic response and tolerability profile as compared to existing CAR-T therapies using viral-based manufacturing methods.

In ongoing efforts to optimize manufacturing, the Company was able to demonstrate increased transposition frequency by using Nanoplasmid technology licensed from Nature Technology Corporation, which, when compared to a standard plasmid, yields more CAR-positive cells at the start of the process. In turn, this reduces manufacturing timelines, has resulted in a higher proliferative capacity in patients, and has the potential to create more efficacious CAR-T products with less toxicity.

Poseida also conducted a preliminary clinical analysis of P-BCMA-101 to test the impact of using a Nanoplasmid in its manufacturing process compared to a standard plasmid. The analysis conducted at a .75 X 10E6 per kg dose found that all patients (n=3) responded to treatment with Nanoplasmid-manufactured P-BCMA-101 and that responses were deep, showing a 100 percent overall response rate (ORR) as compared to an ORR of 50-67% in patients that received P-BCMA-101 manufactured using a standard plasmid at that same dose (n=3, 2 evaluable by IMWG criteria; third patient with plasmacytomas and significant response by PET scan). The three patients given Nanoplasmid-produced P-BCMA-101 at this dose reached a very good partial response (VGPR) or stringent complete response (sCR) compared to a partial response (PR) achieved with the standard plasmid. Notably, using a Nanoplasmid in the manufacturing process did not impact the safety profile of P-BCMA-101 and no incidence of cytokine release syndrome of any grade was observed in patients.

"At Poseida, we are always looking at innovative ways to further improve the performance of our CAR-T product candidates while maintaining an exceptionally low rate of cytokine release syndrome and other potential toxicities," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida. "As our most advanced product candidate, we look forward to providing further updates to our clinical program for P-BCMA-101 later in the year."

P-BCMA-101 has received regenerative medicine advanced therapy (RMAT) status and orphan drug designation from the FDA and is currently being tested in an expanded Phase 1 clinical trial for the treatment of patients with relapsed/refractory multiple myeloma to inform the potentially registrational Phase 2 clinical trial. Poseida’s portfolio includes allogeneic and autologous CAR-T product candidates in both hematological and solid tumor oncology indications, as well as liver-directed gene therapy programs in orphan genetic diseases.

Nanoplasmid-produced product candidates P-BCMA-101 for multiple myeloma and P-PSMA-101 for castrate resistant prostate cancer have both demonstrated robust expansion in patients to date. The Company is now utilizing Nanoplasmid technology to manufacture all autologous and allogeneic product candidates across its portfolio and continues to evaluate additional manufacturing optimizations that may further improve the performance of its product candidates.

The full presentation at CAR-TCR Digital Week will be available on Poseida’s website at the end of the meeting on Thursday, September 17.

On September 16, 2020 Pascal Biosciences Inc. (TSX.V:PAS) ("Pascal") reported that has been invited to present at the International Cannabinoid Derived Pharmaceutical (ICDP) Summit (Press release, Pascal Biosciences, SEP 16, 2020, View Source [SID1234565234]). Today Dr. Patrick Gray, CEO of Pascal Biosciences, will present advancements in each of Pascal’s three cannabinoid programs. The title of his presentation is "Identifying and Validating Mechanism of Action In Vivo and In Vitro".

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The Third Annual ICDP conference is the premier format for presentation of ground-breaking discoveries in cannabinoid drug development. As in past years, this conference is hosting top cannabinoid pharmaceutical experts developing therapeutics derived from cannabis. In addition to describing progress for Pascal’s programs in glioblastoma and coronavirus, Dr. Gray will also discuss plans for developing PAS-393 for treatment of cancer in combination with checkpoint inhibitors. This will be a collaborative effort with SōRSE Technology and aims to characterize the safety, pharmacology, and pharmacokinetics of PAS-393 in a Phase 1a clinical trial.

"I am very pleased for the invitation to present Pascal’s work at this impressive conference," commented Dr. Gray. "This validates the importance of our efforts, and we will be featured with other companies making significant advancements in developing cannabis-based medicines."

The session featuring Pascal’s presentation is focused on preclinical testing of cannabinoid therapeutics. Dr. Gray will also participate in a panel discussion titled "Achieving the Ideal Pharmacokinetic and Pharmacodynamic Profiles in Drug Formulation".

On September 16, 2020 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported a publication in the peer reviewed, open access journal OncoImmunology of a paper on HB-201, an arenavirus vector-based immunotherapy for Human Papillomavirus 16-positive (HPV16+) cancers currently in clinical trials (Press release, Hookipa Pharma, SEP 16, 2020, View Source [SID1234565233]).

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The paper shows that systemically administered HB-201 leads to:

Dose-dependent induction of a robust, systemic cytotoxic T cell response directed against HPV16 proteins;
Tumor infiltration of HPV16 specific cytotoxic T cells; and
Significantly delayed tumor growth or complete tumor clearance accompanied with prolonged survival.
Mice that have cleared tumors post-HB-201 administration developed long-term protection, as demonstrated by the rejection of re-administered tumors. Furthermore, the combination of HB-201 with a checkpoint inhibitor (a-PD-1) increased the anti-tumor efficacy, with more than 77% of treated mice clearing established tumors.

HB-201 is one of HOOKIPA’s lead oncology candidates. It targets HPV16 E6/E7 and is based on the Company’s replicating LCMV (TheraT) arenaviral vector platform. It is currently in Phase 1/2 clinical trials (NCT04180215) for HPV16+ cancers alone and in combination with an approved checkpoint inhibitor.

"HPV-associated cancers, especially head and neck cancers, remain a significant health concern, as no curative therapies are currently available. We are very pleased that these results suggest that the HB-201 program can be a promising therapy for HPV+ cancers," said Igor Matushansky, MD, PhD, HOOKIPA’s Chief Medical Officer and Global Head of Research and Development.

Human Papillomavirus, or HPV, is estimated to cause about 5% of the worldwide burden of cancers. This includes approximately 99% of cases in cervical, up to 60% of head and neck, 70% of vaginal and 88% of anal cancers.

The majority of these cancers are caused by the HPV serotype 16. Most infections with HPV are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This uptake can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to become cancerous. The publication, "Live-attenuated lymphocytic choriomeningitis virus-based vaccines for active immunotherapy of HPV16-positive cancer", is available online in OncoImmunology.

On September 16, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing proprietary spherical nucleic acid (SNA) technology, reported that it will host a virtual Key Opinion Leader (KOL) event to present preliminary Phase 1b efficacy and safety data for its intratumoral product candidate, cavrotolimod, the company’s SNA-enabled TLR9 agonist being developed for the treatment of solid tumors, in combination with pembrolizumab (Press release, Exicure, SEP 16, 2020, View Source [SID1234565232]). The event will take place today from 10:30 am – 12:00 pm ET.

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The principal investigators of the Phase 1b portion of the trial, Dr. Steven O’Day and Dr. Shailender Bhatia, will be presenting and joining Exicure’s leadership team in discussing the clinical data and trial progress to date.

"We are excited by the durable responses we have seen in anti-PD-1 refractory patients, and look forward to further exploring the efficacy of cavrotolimod in these highly treatment refractory Merkel cell carcinoma and cutaneous squamous cell carcinoma patients," said Dr. Douglas Feltner, Chief Medical Officer of Exicure.

The event will be webcast live today, September 16th at 10:30 am ET through a link on the Events and Presentations section of Exicure’s website. An archived webcast will also be available on Exicure’s website following the event. To RSVP for the event, please use the link here (View Source) or email [email protected].

Description of the Trial

The objectives of the Phase 1b dose-escalation stage of the clinical trial were to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of cavrotolimod alone and in combination with pembrolizumab, and to identify a recommended Phase 2 dose. Cavrotolimod was dosed weekly for 8 weeks then every three weeks thereafter. Pembrolizumab was added to the treatment regimen starting in week three of the study. The first two weeks of the trial, the period in which only cavrotolimod was dosed, allowed for the assessment of cavrotolimod safety, tolerability, pharmacokinetics and pharmacodynamics alone. Efficacy was assessed every 12 weeks. Twenty patients have been enrolled and dosed in the dose-escalation stage of the trial consisting of: ten (10) melanoma patients, five (5) MCC patients, two (2) CSCC patients, two (2) head and neck squamous cell carcinoma patients, and one (1) leiomyosarcoma patient. At the

time of enrollment, 85% of patients were experiencing progressive disease while on anti-PD-1 antibody therapy.

Highlights from the data

Highlights from the data update include:

–Confirmed ORR 21% (4/19 patients) overall in the Phase 1b dose-escalation stage
–Confirmed ORR 33% (2/6 patients) in the highest dose cohort (32 mg), which was selected as Phase 2 recommended dose
–Overall responses occurred in two patients with advanced MCC and two patients with melanoma
–Three of four responders were progressing on anti-PD-1 therapy at the time of enrollment
–In addition to the four confirmed responses, target tumor shrinkage occurred in one CSCC patient and two melanoma patients. Systemic (abscopal) effects were observed, with regression in noninjected tumors distant from injected lesions.
–The cavrotolimod pharmacodynamic profile corroborated the efficacy data, as increased serum cytokines/chemokines, activated immune cells, and tumor infiltration by immune cells were observed.

The median duration of response has not been reached as all four confirmed responders have not progressed after a median follow-up of 11 months. The longest response to date is 16 months from initial dosing and is ongoing as of the data cut-off date.

Exicure continues to observe that cavrotolimod is well tolerated with 98% of all treatment-emergent adverse events (AEs) assessed as Grade 1 or 2 in severity. No treatment-related serious adverse events were reported to date. The most common adverse events were flu-like symptoms and injection site reactions, which are commonly expected effects from a TLR9 mechanism of action.

Updated guidance

Exicure expects to provide interim ORR results from the MCC and CSCC cohorts of the Phase 2 portion of the clinical trial in the first half of 2021 and final ORR results by year end 2021.