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.

On September 16, 2020 Zai Lab Limited ("Zai Lab" or the "Company") (NASDAQ:ZLAB), an innovative commercial stage biopharmaceutical company, reported the launch of its Hong Kong public offering (the "Hong Kong Public Offering"), which forms part of the global offering (the "Offering") of 10,564,050 new ordinary shares (the "Offer Shares" or "Shares") and listing of its ordinary shares on the Main Board of The Stock Exchange of Hong Kong Limited (the "Hong Kong Stock Exchange") under the stock code "9688" (Press release, Zai Laboratory, SEP 16, 2020, View Source [SID1234565231]).

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The Company’s American depositary shares ("ADSs"), each representing one (1) ordinary share of the Company, will continue to be listed and traded on the Nasdaq Global Market ("Nasdaq"). Investors in the Offering will only be able to purchase ordinary shares and will not be able to take delivery of ADSs. Upon listing in Hong Kong, the ordinary shares listed on the Hong Kong Stock Exchange will be fully fungible with the ordinary shares represented by the ADSs listed on Nasdaq.

The Offering initially comprises 771,700 new Shares under the Hong Kong Public Offering and 9,792,350 new Shares for the international offering (the "International Offering"), representing approximately 7.3% and 92.7% of the total number of Shares in the Offering, respectively, subject to re-allocation and over-allotment. Subject to the level of any oversubscription in the Hong Kong Public Offering and pursuant to the clawback mechanism as described in the prospectus issued in Hong Kong, the total number of shares available under the Hong Kong Public Offering could be adjusted to up to a maximum of 3,591,800 new Shares, representing approximately 34% of the Offer Shares initially available under the Offering. In addition, the Company expects to grant the international underwriters an over-allotment option to purchase up to an additional 1,584,600 new Shares in the International Offering, representing not more than 15.0% of the Offer Shares initially available under the Offering.

The offer price for the Hong Kong Public Offering (the "Hong Kong Offer Price") will be not more than HK$648.00 per share (the "Maximum Offer Price"). The offer price for the International Offering tranche of the Offering (the "International Offer Price") may be set at a level higher than the Hong Kong Offer Price. The Company is expected to set the International Offer Price by September 22, 2020 Hong Kong time by making reference to, among other factors, the closing price of the ADSs on Nasdaq on the last trading day on or before September 21, 2020 and investor demand during the marketing process. The final Hong Kong Offer Price will be set at the lower of the final International Offer Price and the Maximum Offer Price of HK$648.00 per share. Shares will be traded in board lots of 50 shares.

The Company plans to use the net proceeds from the Offering for its core products, through strengthening R&D efforts and enhancing its commercialization capabilities, advancing its ongoing and planned clinical trials and preparation for registration filings of other drug candidates in its pipeline, exploring new global licensing and collaboration opportunities, funding working capital and other general corporate purposes.

Fully Electronic Application Process for the Hong Kong Public Offering

Zai Lab has decided to adopt a fully electronic application process for the Hong Kong Public Offering, with no printed copies of prospectuses or application forms to the public in relation to the Hong Kong Public Offering. A fully electronic application process is consistent with the way in which the Company’s users and stakeholders engage and interact with each other and the Company. As a company which has been highly committed to environmental, social and corporate responsibility matters since its founding, the Company believes such method will also help mitigate the environmental impact of printing and minimize the exploitation of natural resources, among others. The prospectus is available at the website of the Hong Kong Stock Exchange at www.hkexnews.hk and the Company’s website at www.zailaboratory.com.

The Company encourages applicants for the Hong Kong Public Offering to view its prospectus and apply online through the White Form eIPO service at www.eipo.com.hk, or apply through the CCASS EIPO service. The Hong Kong Public Offering will commence at 9:00 a.m. on Thursday, September 17, 2020 Hong Kong time and will close at 12:00 noon on Tuesday, September 22, 2020 Hong Kong time.

Potential applicants may call the enquiry hotline of Computershare Hong Kong Investor Services Limited if they have any question about making applications for the Hong Kong Offer Shares. The hotline number is +852 2862 8646, and will be open from 9:00 a.m. to 9:00 p.m. on Thursday, September 17, 2020, Friday, September 18, 2020 and Monday, September 21, 2020, from 9:00 a.m. to 6:00 p.m. on Saturday, September 19, 2020 and Sunday, September 20, 2020, and from 9:00 a.m. to 12:00 noon on Tuesday, September 22, 2020 Hong Kong time.

J.P. Morgan Securities (Far East) Limited (or its affiliates, as the case may be), Goldman Sachs (Asia) L.L.C. and Citigroup Global Markets Asia Limited (or its affiliate, as the case may be) are the joint sponsors, joint global coordinators, joint bookrunners and joint lead managers for the proposed Offering.

The International Offering is being made only by means of a preliminary prospectus supplement dated September 16, 2020 and the accompanying prospectus included in an automatic shelf registration statement on Form F-3ASR filed with the U.S. Securities and Exchange Commission (the "SEC") on March 29, 2019 and was subsequently amended and became automatically became effective upon filing with the SEC on January 21, 2020. The registration statement on Form F-3ASR and the preliminary prospectus supplement are available at the SEC website at: View Source Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained from: (i) J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 1-866-803-9204 or by email at [email protected], (ii) Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282 or by telephone at 1-866-471-2526, or (iii) Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at 1-800-831-9146 or by email at [email protected].

The proposed Offering is subject to market and other conditions, and there can be no assurance as to whether or when the Offering may be completed, or as to the actual size or terms of the Offering. This press release shall not constitute an offer to sell or the solicitation of an offer or an invitation to buy any securities of the Company, nor shall there be any offer or sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. This press release does not constitute a prospectus (including as defined under the laws of Hong Kong) and potential investors should read the prospectus of the Company for detailed information about the Company and the proposed offering, before deciding whether or not to invest in the Company. This press release has not been reviewed or approved by the Hong Kong Stock Exchange or the Securities and Futures Commission of Hong Kong.

The price of the Shares of the Company may be stabilized in accordance with the Securities and Futures (Price Stabilization) Rules. The details of the intended stabilization and how it will be regulated under the Securities and Futures Ordinance (Chapter 571 of the laws of Hong Kong) will be contained in the prospectus of the Company dated September 17, 2020