On December 14, 2020 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported the closing of its previously announced upsized underwritten public offering of 5,339,286 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 696,428 shares, at the public offering price of $56.00 per share (Press release, Rocket Pharmaceuticals, DEC 14, 2020, View Source [SID1234572826]). The gross proceeds to Rocket from the offering are expected to be approximately $299 million, before deducting the underwriting discounts and commissions and other offering expenses.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Rocket intends to use the net proceeds from this offering to further fund the development of its pipeline of gene therapies for rare diseases, including filing for marketing authorization for RP-L201 in the United States and Europe, accelerating the buildout of in-house manufacturing capabilities, and for general corporate purposes.

J.P. Morgan, BofA Securities, SVB Leerink and Piper Sandler acted as the joint bookrunning managers for the public offering.

The public offering was made by Rocket pursuant to an effective shelf registration statement on Form S-3 that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC. A final prospectus supplement relating to and describing the terms of this offering was filed with the SEC on December 11, 2020. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may be obtained from J.P. Morgan Securities LLC, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, from BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at [email protected], from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at (800) 808-7525, ext. 6132, or by email at [email protected], or from Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924, or by email at [email protected]. You may also obtain these documents free of charge by visiting the SEC’s website at www.sec.gov.

This press release does not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities 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.

On December 14, 2020 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported that it has been added to the NASDAQ Biotech Index (Nasdaq: NBI), effective prior to market open on Monday, December 21, 2020 (Press release, Kezar Life Sciences, DEC 14, 2020, View Source [SID1234572825]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The NASDAQ Biotechnology Index is designed to track the performance of a set of securities listed on The Nasdaq Stock Market (Nasdaq) that are classified as either biotechnology or pharmaceutical according to the Industry Classification Benchmark (ICB). The NASDAQ Biotechnology Index is calculated under a modified capitalization-weighted methodology. Companies in the NASDAQ Biotechnology Index must meet eligibility requirements, including minimum market capitalization, average daily trading volume and seasoning as a public company, among other criteria. Nasdaq selects constituents once annually in December.

On December 14, 2020 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that it has commenced additional physical parameter testing of its CypCaps product for pancreatic cancer, in line with the recommendations provided by the U.S. Food and Drug Administration (FDA) (Press release, PharmaCyte Biotech, DEC 14, 2020, View Source [SID1234572824]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The FDA has asked that two additional methods be developed to determine the strength of PharmaCyte’s encapsulated cells (CypCaps) to be used in the company’s planned clinical trial in locally advanced, inoperable pancreatic cancer (LAPC). One method involves pressing down on the capsule and measuring either the pressure required for it to burst, or for it to deform. Since the CypCaps are very small, special machinery that can measure such tiny changes has to be used to demonstrate this. The necessary machinery is now being incorporated as a quality control test for the CypCaps.

The second method involves letting water flow into the CypCaps, effectively "blowing them up." The point at which the capsules explode will be used as a quality control parameter.

Previous work has shown the pressures and water conditions used in these tests to be well outside of the normal conditions encountered by the capsules inside the human body, so these tests are designed to simulate hypothetical conditions.

Earlier studies have shown that the capsules do not burst even when placed under very high pressure. Further, even in the very unlikely event that the capsule could break open, the cells inside will be recognized as foreign bodies by the immune system. Also, the encapsulated cells are primed for their suicide since they express the cytochrome P450 gene and thus would be killed by the low dose ifosfamide given as part of the treatment for LAPC.

Thus, these FDA mandated studies can be seen as additional release tests to ensure the reproducibility of the CypCaps.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "We are pleased to announce the development of these two new quality parameters and their incorporation into the quality testing as one of the additional studies requested by the FDA. In the meantime, PharmaCyte continues to work with its partners to address the other issues raised by the FDA that led to the clinical hold."

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

On December 14, 2020 CEL-SCI Corporation (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported that it will be presenting at the 13th Annual LD Micro Main Event Conference on Tuesday, December 15, 2020 at 10:00 a.m. ET. Geert Kersten, Chief Executive Officer of CEL-SCI, will be presenting to a live virtual audience (Press release, Cel-Sci, DEC 14, 2020, View Source [SID1234572823]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

CEL-SCI’s presentation will be broadcast live at View Source or on the Investor Relations section of CEL-SCI’s website at cel-sci.com/new-investor-information/.

LD Micro’s Main Event will feature a new format, with companies presenting for 10 minutes, followed by 10 minutes of Q&A by a panel of investors and analysts.

On December 14, 2020 Propanc Biopharma, Inc. (OTC: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported on the progress of the Company, recent developments and forecast for 2021, as the Company prepares for entering clinical development for its lead product candidate, PRP, for the treatment and prevention of metastatic cancer (Press release, Propanc, DEC 14, 2020, View Source [SID1234572822]). PRP represents a novel therapeutic approach, targeting and eradicating cancer stem cells, but leaving healthy stem cells alone, making it less toxic compared to current standard treatment options, like chemotherapy and radiotherapy. PRP does not suppress the immune system, and therefore in a post pandemic world, can offer support to cancer sufferers who are at risk of infection.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Whilst navigating the challenges presented during the global pandemic, Propanc management believes the Company has the fundamentals in place to progress PRP to a Phase Ib, First-In-Human (FIH) study in advanced cancer patients," said James Nathanielsz, Propanc’s Chief Executive Officer. "We continue to work on publishing our latest scientific research and exploiting further patent opportunities for our expanding intellectual property portfolio. PRP represents a novel approach that offers a real alternative to standard treatment options which is less toxic and supports immune function when compared to standard treatment options. In a post COVID world, we need to support cancer sufferers whose lives have been significantly affected by cancer and the threat of post treatment secondary infection, which can be life threatening. We look forward to expending every effort to meet our goals in 2021."

Recent Developments

The Company recently raised $209,000 for operating expenses. Furthermore, an S-1 Registration Statement received a notice of effectiveness from the Securities and Exchange Commission for a lead institutional investor as part of a financing agreement entered into earlier this year of up to $3 million with a total of $626,035 of securities purchased to date. The Company will continue to work with the lead institutional investor for future funding tranches to prepare the Company’s lead product candidate, PRP, for a Phase Ib, FIH study in advanced cancer patients suffering from solid tumors.

Forecast for 2021

In 2021, funds raised from the Company’s institutional investor will be used to undertake an engineering run and full scale Good Manufacturing Practice (GMP) manufacture of PRP, the Company’s lead product candidate, as well as validation of the pharmacokinetics method to analyze the distribution of the drug in advanced cancer patients for a Phase Ib, FIH study, which the Company intends to undertake in the second half of 2021, at the Peter Mac Center in Melbourne, Australia’s biggest cancer hospital.

Lead Product Candidate – PRP

PRP is a pharmaceutical composition consisting of two pancreatic proenzymes trypsinogen and chymotrypsinogen for treating cancer. PRP is a novel approach to prevent recurrence and metastasis of solid tumors by using pancreatic proenzymes that target and eradicate cancer stem cells in patients suffering from pancreatic, ovarian and colorectal cancers. PRP is a novel therapy based on the science that enzymes stimulate biological reactions in the body, especially enzymes secreted by the pancreas and could represent the body’s primary defense against cancer.

To date, preclinical development has been completed for PRP, including pharmacology and safety toxicology studies, process development activities and bioanalytical method development. Propanc Biopharma is collaborating with contract research organizations, manufacturing partners and its consultants to complete the activities prior to preparing the CTA for the Phase Ib, FIH study.

Joint Research and Drug Discovery Program – POP1

The POP1 joint research and drug discovery program is designed to produce a backup clinical compound to the lead product candidate, PRP. With the aim of producing large quantities of trypsinogen and chymotrypsinogen for commercial use, exhibiting minimal variation between lots and without sourcing the proenzymes from animals, Propanc Biopharma is undertaking a challenging research project in collaboration with the Universities of Jaén and Granada, led by research scientist Mr. Aitor González, supported by Dr. Macarena Perán, Ph.D. and Dr. Julian Kenyon, M.D. as joint supervisors, representing the Universities and Propanc, respectively.

One specific objective of the project will be to synthesize both proenzymes by an in vivo (i.e., a living organism) system to produce crystalized proteins that could be maintained for long periods without suffering degradation, even in absence of refrigeration. This will be particularly useful for a longer shelf life as well as global distribution of the drug product, particularly in warmer climates and developing regions where refrigeration may not be available.

The POP1 joint research and drug discovery program has produced synthetic recombinant versions of the two proenzymes, trypsinogen and chymotrypsinogen. Propanc Biopharma’s joint scientific researchers developed a novel expression system and are in the process of optimizing conditions to achieve high titers of recombinant trypsinogen and chymotrypsinogen. Further, the anticancer effects of the synthetic versions will be tested against the naturally derived proenzymes from bovine origin.

Propanc Biopharma recently entered into a second two-year joint research and collaboration agreement with the University of Jaén who are undertaking the research activities for the POP1 program.