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PharmaCyte Biotech’s Pancreatic Cancer Therapy Passes Critical FDA-Required Tests as Countdown to IND Submission Begins

On January 27, 2020 PharmaCyte Biotech (OTCQB: PMCB) reported that its clinical trial product, which will be employed during the company’s upcoming Phase 2b clinical trial to treat locally advanced, inoperable pancreatic cancer (LAPC), has passed each of the U.S. Food and Drug Administration’s (FDA) required "release tests" for safety and functionality (Press release, PharmaCyte Biotech, JAN 27, 2020, View Source [SID1234553588]). With a clinical trial product that has proven to be safe and functional, PharmaCyte is now "on-the-clock" for its submission of an Investigational New Drug application or "IND" package to the U.S. FDA requesting a Phase 2b clinical trial in LAPC at trial sites throughout the United States.

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In a series of 10 "release tests" over two batches of the company’s clinical trial product for a total of 20 "release tests," PharmaCyte’s signature live-cell encapsulation technology, Cell-in-a-Box, performed admirably and proved to be both safe to place into humans in a clinical trial, and to function properly. During the tests, the more than 20,000 genetically modified live cells that fully fill each Cell-in-a-Box capsule were functioning as expected after being manufactured, placed into syringes, frozen, thawed and then tested for enzymatic activity, cell viability, and biologic activity among a host of other tests.

And now that "release testing" has ended successfully, Austrianova will issue 2 Certificates of Analysis to PharmaCyte—1 for each manufactured and tested batch of the company’s clinical trial product. Additionally, Austrianova will turn over all of the completed batch records to PharmaCyte from both production runs and the subsequent "release testing." The data generated from these tests are necessary information that must be entered into the company’s IND application.

PharmaCyte has methodically taken extra steps to position the company for a greater chance at success once the IND is submitted to the FDA. One such step is the manufacture and testing of two batches of its clinical trial product, which was requested by cGMP Validation, the company taking responsibility for releasing the clinical trial product into the U.S. for use in PharmaCyte’s upcoming clinical trial.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, commenting on manufacturing a second batch of the company’s clinical trial product, said, "Our GMP consultant, cGMP Validation, has informed us that while two successful manufacturing runs are not required by the FDA to request a Phase 2b clinical trial, it could go a long way in demonstrating to the FDA that our manufacturing process is robust and reproducible – manufacturing qualities that are highly embraced by the FDA."

Successfully completing the manufacturing process of PharmaCyte’s clinical trial product, conducting and successfully completing "release testing" on both batches of the company’s product, and the data being generated from those tests are all critical to the completion of PharmaCyte’s IND package. With the announced success of the "release tests," PharmaCyte has essentially conquered what was the greatest hinderance in the company’s ability to move forward with completing its IND application.

Because pancreatic cancer is the third leading cause of cancer-related deaths, this work for PharmaCyte is important and necessary, and the company has painstakingly endured a process of perfection to land it on the precipice of engaging with the FDA in an opportunity to enter into a clinical trial. In 2020 alone, an estimated 57,600 Americans will be diagnosed with pancreatic cancer, and approximately 47,050 Americans are expected to die from the disease this year.

PharmaCyte hopes to better those dismal numbers by using its targeted chemotherapy—Cell-in-a-Box plus low doses of the anticancer drug ifosfamide—in patients with LAPC who no longer see any benefit after being treated for 4-6 months with one of the two first-line therapies offered to this patient population. Its stated goal is to make inoperable tumors operable and give these patients a second chance at life. Needless to say, PharmaCyte is closer than ever to submitting its IND, so the countdown to this momentous milestone for the company’s shareholders should be "officially" underway and is certainly highly anticipated.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

Santhera Reports Net Revenues 2019 and Highlights Pipeline Progress

On January 27, 2020 Santhera Pharmaceuticals (SIX: SANN) reported net revenues of CHF 27.9 million in 2019 from sales of Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON), which was out-licensed to Chiesi Group from August 2019 (Press release, Santhera Pharmaceuticals, JAN 27, 2020, View Source [SID1234553587]). The Company made strong progress in advancing its lead neuromuscular compounds Puldysa (idebenone) and vamorolone towards market entry and regulatory submission, respectively.

"We are pleased about Santhera’s strong progress in 2019 and are excited about the prospects 2020 holds," said Dario Eklund, CEO of Santhera. "Our late-stage neuromuscular assets targeting the high value Duchenne muscular dystrophy (DMD) market are nearing key inflection points. With Puldysa (idebenone), we anticipate offering the first drug for the treatment of respiratory dysfunction for non-ambulant DMD patients who are not taking glucocorticoids. Vamorolone, a first-in-class anti-inflammatory drug candidate shown in studies to improve muscle and motor function with a favorable clinical safety profile, is being developed to replace glucocorticoids as standard of care in ambulant DMD patients. We will continue working with clinical experts, patient advocacy groups and regulators in order to bring these promising treatments to patients with currently few alternative treatment options as soon as possible."

2019 Turnover

Net revenues CHF 27.9 million
Gross income of CHF 49.3 million from Raxone out-licensing agreement
Freely available liquid funds of CHF 31.4 million (December 31, 2019)
2019 full-year net revenues slightly above guidance
In 2019, Santhera reported net revenues of CHF 27.9 million (2018: CHF 31.7 million), slightly surpassing the Company’s full-year guidance. This includes sales of Raxone in the approved indication Leber’s hereditary optic neuropathy (LHON) in the first seven months of 2019. From August 2019, after the closing of the licensing transaction and the transfer of the Raxone-business to Chiesi Group, Santhera is commercializing Raxone for LHON in France in a transitional phase.

Upfront payment from Chiesi Group following closing of licensing agreement
In August 2019, Santhera recognized an initial gross income of CHF 49.3 million (EUR 44 million) from the licensing agreement with Chiesi Group. As previously announced per the agreement, Chiesi Group has in-licensed Raxone for LHON and all other ophthalmologic indications for all territories worldwide except the US and Canada for a total consideration of up to EUR 93 million.

As of December 31, 2019, freely available liquid funds (cash and cash equivalents) amounted to CHF 31.4 million (August 31, 2019: CHF 43.7 million). In addition, the Company held CHF 1.5 million of restricted cash designated for the interest payments related to the convertible bonds issued in 2017.

Pipeline and Regulatory Update

Anticipated near-term inflection points towards approval for both DMD pipeline candidates Puldysa (idebenone) and vamorolone are:

Q2-2020: CHMP opinion on marketing authorization application for Puldysa in DMD in Europe
Q4-2020: Launch of Puldysa in first European markets
Q4-2020: Read-out of topline data of pivotal trial for vamorolone in DMD
Q1-2021: Filing a New Drug Application for vamorolone in DMD in the US
Puldysa first launch in Europe in 2020 subject to positive opinion and approval from CHMP/EMA
The review of Santhera’s application for conditional marketing authorization (CMA) for Puldysa in the treatment of respiratory dysfunction in patients with DMD who are not using glucocorticoids is ongoing and the Company expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) around mid-2020. Subject to a positive opinion and subsequent EU approval, Santhera expects to launch Puldysa in the first European markets in late 2020.

SIDEROS DMD trial enrollment with idebenone nearing completion
Patient enrollment in Santhera’s randomized, double-blind, placebo-controlled Phase 3 SIDEROS trial is expected to complete in Q2-2020 (clinicaltrials.gov: NCT02814019). With a study duration of 18 months, the last patient’s last visit is scheduled for Q4-2021. If positive, the study data will allow for regulatory submissions supporting the use of Puldysa in all DMD patients experiencing respiratory decline, irrespective of their glucocorticoid use, in Europe and the US.

Vamorolone pivotal VISION-DMD study progressing as cornerstone for US/European regulatory filings
ReveraGen BioPharma is currently enrolling the Phase 2b VISION-DMD study with vamorolone, designed as a pivotal efficacy and safety trial (VBP15-004; clinicaltrials.gov: NCT03439670). Read-out of topline 6-month data from the randomized placebo-controlled treatment period is expected by Q4-2020 followed by an NDA submission in the US by Q1-2021. Under the agreements between the parties and upon exercising its option, Santhera would receive the sub-license to ReveraGen’s vamorolone from Idorsia Ltd (SIX: IDIA) for all indications and all countries worldwide except Japan and South Korea. The marketing authorization application in Europe will require inclusion of 12-month data expected for Q2-2021.

Neuromuscular franchise a top priority for 2020
Santhera’s strategic priorities for 2020 are its neuromuscular franchise: Puldysa and vamorolone in DMD. For Puldysa, the focus is on the preparation for European market entry in DMD later in the year and the completion of enrollment into the SIDEROS trial to support planned regulatory submissions, particularly in the US. For vamorolone, the key milestone will be the VISION-DMD topline data readout, which if positive would allow for preparation of the NDA filing and pave the way for Santhera’s option exercise.

In parallel, the Company is advancing its clinical stage candidate POL6014 for cystic fibrosis and is evaluating further diversification of its platform type pipeline products, including development of additional indications in collaboration with partners.

Corporate Calendar

March 24, 2020 – Publication of the Annual Report 2019 (07h00 CET)
April 22, 2020 – Annual Shareholders’ Meeting

Bavarian Nordic Announces Closing of Sale of Priority Review Voucher

On January 27, 2020 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported the completion of the sale of its Priority Review Voucher (PRV) to an undisclosed buyer (Press release, Bavarian Nordic, JAN 27, 2020, View Source [SID1234553586]). Upon completion, the company received a cash consideration of USD 95 million.

Zymeworks Announces Closing of Public Offering and Exercise in Full of the Underwriters’ Over-Allotment Option to Purchase Additional Shares

On January 27, 2020 Zymeworks Inc. (NYSE: ZYME), a clinical stage biopharmaceutical company developing multifunctional biotherapeutics ("Zymeworks" or the "Company"), reported the closing of its previously announced underwritten public offering (the "Offering") (Press release, Zymeworks, JAN 27, 2020, View Source [SID1234553579]). The Offering consisted of 5,824,729 common shares, including the exercise in full of the underwriters’ over-allotment option to purchase 900,000 additional shares, and, in lieu of common shares, to a certain investor, pre-funded warrants to purchase up to 1,075,271 common shares. The common shares were offered at a price to the public of US$46.50 per common share and the pre-funded warrants were offered at a price of US$46.4999 per pre-funded warrant, for aggregate gross proceeds to the Company of approximately US$320.8 million, before deducting underwriting discounts and commissions and estimated Offering expenses.

The Company intends to use the net proceeds of the Offering (i) to accelerate and expand the global development of ZW25 both as a single agent and in combination with other anti-cancer agents in a variety of HER2-expressing tumors, including gastroesophageal, biliary tract, breast and other underserved cancers; (ii) to accelerate and expand the clinical development of ZW49; (iii) to advance other novel preclinical programs, including those involving non-HER2-expressing tumors; and (iv) for general corporate purposes.

J.P. Morgan Securities LLC and Citigroup Global Markets Canada Inc. acted as active bookrunners for the Offering. Stifel, Nicolaus & Company, Incorporated and Wells Fargo Securities Canada, Ltd. acted as bookrunners and Raymond James Ltd. acted as lead manager.

The securities described above were offered in Canada pursuant to Zymeworks’ final prospectus supplement, dated January 22, 2020 (the "Canadian Supplement"), to its Canadian final base shelf prospectus, dated November 18, 2019 (the "Base Prospectus"), and in the United States pursuant to Zymeworks’ final prospectus supplement, dated January 22, 2020 (the "U.S. Supplement", together with the Canadian Supplement, the "Supplements"), to its U.S. automatic shelf registration statement on Form S-3ASR, including a prospectus dated November 5, 2019 (the "Registration Statement"). The Supplements were filed in Canada and the United States on January 23, 2020.

The Supplements and the Registration Statement contain important detailed information about the Offering. A copy of the Canadian Supplement can be found on SEDAR at www.sedar.com, and a copy of the U.S. Supplement and the related Registration Statement can be found on EDGAR at www.sec.gov. Copies of the Supplements may also be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (866) 803-9204; Citigroup Global Markets Canada Inc., Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (800) 831-9146; Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, CA 94104, by telephone at (415) 364-2720, or by email at [email protected]; or Wells Fargo Securities Canada, Ltd., Attention: Equity Syndicate Department, 30 Hudson Yards, 500 West 33rd Street, New York, NY 10001, by telephone at (800) 326-5897, or by email at [email protected]. Prospective investors should read the Supplements and the Registration Statement before making an investment decision.

This news release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any province, state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such province, state or jurisdiction.

Zai Lab Announces Closing of Public Offering of American Depositary Shares and Full Exercise of Greenshoe Option

On January 27, 2020 Zai Lab Limited ("Zai Lab" or the "Company") (NASDAQ: ZLAB), a China and U.S.-based innovative commercial stage biopharmaceutical company, reported the closing of its previously-announced underwritten public offering of 5,500,000 American depositary shares ("ADSs"), each representing one ordinary share of the Company (the "Primary ADS Offering"), at a price of US$47.50 per ADS (Press release, Zai Laboratory, JAN 27, 2020, View Source [SID1234553578]). In addition, QM11 Limited, a shareholder of the Company, offered 500,000 ADSs of the Company (the "Secondary ADS Offering" and together with the Primary ADS Offering, the "Offering") at the same price.

Zai Lab and QM11 Limited had also granted the underwriters a 30-day option to purchase up to an additional 800,000 and 100,000 ADSs, respectively at the public offering price, less underwriting discounts and commissions. The underwriters fully exercised their option to purchase these additional ADSs. Given the full exercise of the option, QM11 Limited now owns approximately 12.15% of the Company’s share capital immediately following this Offering. The Offering closed on January 27, 2020.

The gross proceeds to Zai Lab from this Offering, before deducting underwriting discounts and commissions and other offering expenses, were approximately US$299.3 million. Zai Lab did not receive any proceeds from the sale of ADSs by QM11 Limited.

J.P. Morgan, Citigroup, Goldman Sachs & Co. LLC and SVB Leerink acted as joint book-running managers, and Guggenheim Securities acted as lead manager for the Offering.

The ADSs were offered pursuant to a shelf registration statement on Form F-3ASR, which became automatically effective upon filing with the U.S. Securities and Exchange Commission ("SEC") on March 29, 2019 and was subsequently amended and became automatically effective upon filing with the SEC on January 21, 2020.

The Offering was made only by means of a prospectus supplement and an accompanying prospectus included in Form-3ASR. The registration statement on Form F-3ASR and the prospectus supplement are available at the SEC’s website at: View Source Copies of the prospectus supplement and the accompanying prospectus may 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) Citigroup Global Markets Inc., c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by telephone at 1-800-831-9146, (iii) Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at 1-866-471-2526, by facsimile at (212) 902-9316 or by email at [email protected] or (iv) SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 1-800-808-7525 ex. 6132 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy ADSs or any other securities, nor shall there be any sale of ADSs in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.