On June 26, 2019 Vaccibody AS reported strong immunogenicity data from the neoantigen cancer vaccine clinical trial VB N-01 (Press release, Vaccibody, JUN 26, 2019, View Source [SID1234537300]). Immunogenicity was assessed in two patients with renal cell carcinoma (RCC) and two patients with squamous cell carcinoma of the head and neck (SCCHN) after treatment with a VB10.NEO in combination with checkpoint inhibitor therapy as per protocol. Before VB10.NEO vaccination, these patients had been treated with the checkpoint inhibitor nivolumab for 12-32 months with stable disease as best response. One patient was progressing at start of vaccination. All patients had low tumour mutational burden ranging from 1.7-3.2 mutations/Mb. The top 20 neoepitopes predicted by Vaccibody’s proprietary NeoSELECTTM algorithm was selected for each of the fully personalized VB10.NEO neoantigen vaccines. Immunogenicity to each individual neoepitope has so far been assessed after 3 to 6 vaccinations of VB10.NEO by an in vitro stimulated IFN-γ ELISpot. Strong T cell responses were observed in all these first four patients tested. T cell responses were significantly increased in post-vaccination samples towards 63% of the neoepitopes. The response to the vaccine was very solid with an average increase of more than 1200 SFU per million PBMC which is on average a 250-fold increase from baseline. The breadth and the strength increased with number of vaccinations. An amplification of existing neoepitope-specific T cells as well as de novo responses were observed in all patients.

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Agnete Fredriksen President and CSO of Vaccibody, commented: We are very pleased to see the strong T cell responses observed in all of the first four patients at the initial assessments. We are excited to see that the vaccine was able to induce so strong T cell responses even in the patients with a history of many lines of previous treatment and no objective response to long-term treatment with anti-PD-1. All these first patients had low tumour mutational burden (TMB) leaving the number of mutations limited for selection of immunogenic neoepitopes, hence we believe the high percentage of the neoantigen-specific immune responses observed substantiates Vaccibody’s unique neoepitope prediction method and delivery mechanism. We are very intrigued by the de novo responses induced by the vaccine. The baseline responses and hence the number of de novo responses were surprisingly different between the patients assessed so far and we are looking forward to characterizing the T cells in more detail and follow the clinical responses in these patients

On June 26, 2019 Yisheng Biopharma Co., Ltd. ("Yisheng Biopharma"), a biopharmaceutical company focusing on research, development, manufacturing, sales and marketing of immunological biologics and vaccines, reported that it has received Investigational New Drug (IND) clearance from the National Medical Products Administration (NMPA) of China to initiate a clinical trial of YS-ON-001, a first-in-class immuno-oncology product for the treatment of advanced solid tumors (Press release, Yisheng US Biopharma, JUN 26, 2019, View Source [SID1234537284]).

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"The IND clearance for YS-ON-001 by the China NMPA marks the third product entering clinical development based on our PIKA technology platform. This milestone once again demonstrates Yisheng’s capability and commitment to the advancement of immunological technology for biopharmaceutical market. PIKA immuno-modulating technology has been officially recognized three times since 2013 as a "National New Medical Innovation" by the Chinese government. We plan to move forward with the clinical development of YS-ON-001 in China to bring it to the market as efficiently as possible. Yisheng Biopharma will continue to expand our portfolio in new immunological therapeutics and vaccines to benefit patients in need of new options," commented by David Shao, Ph.D., President and Chief Executive Officer of Yisheng Biopharma.

About YS-ON-001

YS-ON-001 consists of a complex of protein and PIKA immunomodulating agent and is designed to reduce immunosuppressive effects within the tumor microenvironment and enhance antitumor immune responses. YS-ON-001 is capable of increasing CD4+ and CD8+ T-cell responses, increasing the proportion of natural killer and natural killer T cells and promoting higher expression level of PD-L1 receptors in tumor tissues. YS-ON-001 also showed the reduction in the number of T regulatory cells and myeloid-derived suppressor cells. YS-ON-001 can be delivered via intramuscular, subcutaneous or intratumoral injection and has demonstrated a highly effective tumor growth inhibition in preclinical studies with models of breast, lung, liver, colorectal, prostate and other cancers.

YS-ON-001 has received Orphan Drug Designation from the U.S FDA for development of the treatment for both hepatocellular cancer and pancreatic cancer, and is approved for use in Cambodia as YivykaTM.

On June 26, 2019 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") reported that a poster detailing Actinium’s preclinical work with the CD38 antibody daratumumab, a blockbuster therapy marketed as Darzalex by Johnson & Johnson for patients with Multiple Myeloma, labeled with the radioisotope Ac-225 or Actinium-225 has been selected as a top poster at SNMMI for inclusion in a competitive poster competition at the 2019 Society of Nuclear Medicine and Molecular Imaging Annual Meeting (Press release, Actinium Pharmaceuticals, JUN 26, 2019, View Source [SID1234537269]). The poster titled, "225AC-CD38 antibody targeting is effective and well tolerated in experimental models of lymphoma and multiple myeloma" was selected from more than 1,100 posters that were presented at this year’s SNMMI and awarded second place in the Oncology: Basic, Translational & Therapy track (Click here for poster).

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Highlights from the poster include:

Ac-225 labeled daratumumab, at an equimolar concentration, demonstrated superior antitumor activity to naked daratumumab in DAUDI lymphoma tumor xenograft model and provided a survival benefit
Tumor cell death in cell culture was increased as much as ten-fold following exposure to Ac-225 labeled daratumumab, approaching one-hundred percent cell death in certain cell lines
Immunoreactivity for the target antigen CD38 was similar to naked daratumumab demonstrating that arming the antibody with Ac-225 preserved daratumumab’s CD38 targeting ability
Dr. Dale Ludwig, Actinium’s Chief Scientific Officer, presented the poster at SNMMI.

Actinium’s AWE or Antibody Warhead Enabling technology platform generated the Ac-225 daratumumab ARC or Antibody Radiation-Conjugate. Actinium’s AWE platform is covered by know-how and trade secrets that cover the generation, development, methods of use and manufacturing of ARC’s. Actinium’s AWE intellectual property portfolio is comprised of 28 patent families and over 100 issued and pending patents having useful life extending out as far as 2039.

Dr. Ludwig commented, "We are honored that our poster was recognized for this competition from the over 1,100 posters and great work that was presented at this year’s SNMMI. It was apparent from SNMMI that the radiopharmaceutical field, particularly targeted radiotherapies, is experiencing significant growth and rapid innovation. Actinium is dedicated to staying at the forefront of the field. Our commitment is evidenced by our expanded research efforts that have resulted in a significant number of new patent filings and presentations at international conferences. Further, our active research collaboration with Astellas Pharma is ongoing and we are working to complete the third and final module of the program. Through our AWE technology platform, we have demonstrated the ability to utilize multiple isotopes with multiple targeting agents to generate potent ARCs, giving us unmatched breadth and abilities in therapeutics discovery and development. Together with Actinium’s clinical experience and comprehensive supply chain capabilities, we can offer partners and collaborators a turnkey solution from R&D to the clinic and beyond."

On June 26, 2019 Insmed Incorporated (Nasdaq: INSM) reported the closing on June 25, 2019 of the issuance of an additional 1,442,307 shares of common stock pursuant to the full exercise of the underwriter’s overallotment option related to the previously announced public offering of its common stock (Press release, Insmed, JUN 26, 2019, View Source [SID1234537285]). The underwriters were granted 30-day options to purchase up to an additional 1,042,307 shares of common stock from Insmed and up to 400,000 shares of common stock from William H. Lewis, the Company’s Chairman and Chief Executive Officer at a public offering price of $26.00 per share. The net proceeds to the Company from the sale of the additional shares, after deducting underwriting discounts and commissions but before expenses, were approximately $25.6 million. The Company did not receive any proceeds from the sale of Mr. Lewis’ shares.

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Insmed intends to use its net proceeds from this offering to continue to commercialize ARIKAYCE (amikacin liposome inhalation suspension); conduct further trials of ARIKAYCE, including Insmed’s required confirmatory trial to assess and describe the clinical benefit of ARIKAYCE in patients with Mycobacterium avium complex (MAC) lung disease; fund further clinical development of INS1007 and INS1009; invest in increased third‑party manufacturing capacity for ARIKAYCE; fund business expansion activities in Europe and Japan; fund working capital, potential debt repayment, capital expenditures, and general research and development; and for other general corporate purposes, which may include the acquisition or in‑license of additional compounds, product candidates, technology or businesses.

Morgan Stanley & Co. LLC, SVB Leerink LLC and Goldman Sachs & Co. LLC acted as joint book-running managers for the offering. Credit Suisse Securities (USA) LLC, Stifel, Nicolaus & Company, Incorporated and H.C. Wainwright & Co. acted as co-managers for the offering.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock described above was filed with the Securities and Exchange Commission (SEC), as amended by Post-Effective Amendment No. 1 thereto, and became automatically effective upon filing. A final prospectus supplement relating to and describing the terms of the offering was filed with the SEC and is available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained from Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, Massachusetts 02110, telephone: 1-800-808-7525, ext. 6132 or email at [email protected]; and Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 1-212-902-9316 or email at [email protected].

This press release shall 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 jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction.

On June 26, 2019 Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and a biopharmaceutical company specializing in the development of innovative gene and cellular therapies to improve the lives of patients, reported that the first patient has been dosed with PRGN-3006, a first-in-class investigational therapy using Precigen’s non-viral UltraCAR-T therapeutic platform (Press release, Intrexon, JUN 26, 2019, View Source [SID1234537270]). PRGN-3006 UltraCAR-T is an autologous chimeric antigen receptor T (CAR-T) cell therapy under investigation for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS) (clinical trial identifier: NCT03927261).

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PRGN-3006 utilizes Precigen’s transformative UltraCAR-T therapeutic platform, which eliminates ex vivo expansion and reduces manufacturing time to fewer than two days following non-viral gene transfer at the cancer center. PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilizing Precigen’s advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin‐15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS. The study is conducted in collaboration with the Moffitt Cancer Center.

"The first patient dosed using Precigen’s UltraCAR-T therapeutic platform is an important milestone for our company," said Helen Sabzevari, PhD, President of Precigen. "Timing is critical for this patient population and the ability to manufacture PRGN-3006 UltraCAR-T overnight without ex vivo expansion accelerates timing to provide treatment to patients."

"AML is an aggressive disease with very poor prognosis," said James J. Mulé, PhD, Associate Center Director and Michael McGillicuddy, Endowed Chair in Melanoma Research/Treatment at the Moffitt Cancer Center. "The first patient dosed with investigational PRGN-3006 UltraCAR-T represents a significant development for this challenging patient population with high unmet medical need."

The PRGN-3006 UltraCAR-T clinical study is a single center, nonrandomized, investigator‐initiated Phase 1/1b safety and tolerability study. The safety and tolerability of PRGN‐3006 UltraCAR-T will be assessed following intravenous administration of escalating doses in patients with relapsed or refractory AML or higher risk MDS.

"AML and MDS patients have few treatment options, and time is critical when selecting the best treatment path," said David A. Sallman, MD, lead investigator for the PRGN-3006 study at the Moffitt Cancer Center. "We are hopeful that this study will be the beginning of the development of a therapeutic that may result in a critically needed new safe and efficacious treatment option that allows for rapid treatment."

About Acute Myeloid Leukemia (AML)
AML is a cancer that starts in the bone marrow, but most often moves into the blood1. Though considered rare, AML is among the most common types of leukemia in adults2. In 2019, it is estimated that 21,450 new cases of AML will be diagnosed in the US2. AML is uncommon before the age of 45, and the average age of diagnosis is about 682. The prognosis for patients with AML is poor with an average 5‐year survival rate of approximately 25 percent overall and less than a 5 percent 5‐year survival rate for patients older than 653. Amongst elderly AML patients (≥ 65 years of age) median survival is short, ranging from 3.5 months for patients 65 to 74 years of age to 1.4 months for patients ≥ 85 years of age3.

About Myelodysplastic Syndromes (MDS)
MDS are cancerous conditions of the bone marrow4 generally found in adults in their 70s5. Incidence in the US is not known for sure, but estimates range from 10,000 each year and higher5. Sometimes referred to as pre-leukemia, about 1 in 3 MDS patients progress to AML4. As diseases of the bone marrow, MDS outlook is not based on the size of a tumor or tumor metastasis, and other factors are used to predict outlook and inform the treatment plan. Some factors have been combined to develop scoring systems, such as the Revised International Prognostic Scoring System (IPSS-R)6. Using the IPSS-R6, median survival for MDS patients can be estimated to vary from less than one year for the "very high" IPSS-R risk group to more than eight years for the "very low" IPSS-R group7.

Precigen : Advancing Medicine with PrecisionTM
Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cellular therapies using precision technology to target the most urgent and intractable diseases in immuno-oncology, autoimmune disorders, and infectious diseases. Precigen also follows the science opportunistically in pursuit of promising programs in emerging therapeutics. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. Precigen was founded as a wholly-owned subsidiary of Intrexon Corporation (NASDAQ: XON) and leverages a diverse portfolio of technology platforms to advance human health. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Precigen’s UltraCAR-TTM Therapeutic Platform
Precigen’s UltraCAR-T platform has the potential to disrupt the CAR-T treatment landscape by increasing patient access through shortening manufacturing time, decreasing manufacturing-related costs, and improving outcomes using advanced approaches for precise tumor targeting and control of the immune system. The platform brings several key advancements: 1) Non-viral gene transfer using multigenic vectors for expression of multiple effector genes leads to better precision and control of tumor targeting and eliminates the need for virus; 2) Sustained persistence and desired phenotype of infused UltraCAR-T helps address T-cell exhaustion, a common issue with current CAR-T therapies; 3) T-cell control by incorporation of kill switch technology to potentially improve the safety profile; and 4) Rapid manufacturing of UltraCAR-T cells using our proprietary non-viral gene transfer process, which eliminates the need for ex vivo propagation, thus dramatically reducing wait times for patients from weeks to fewer than two days.