On May 13, 2019 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapiesfor the treatment of solid tumors, reported that it has received Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA) to proceed with a Phase 1 clinical trial of its lead myvac candidate TG4050 as a potential treatment for ovarian cancer patients after first-line surgery and chemotherapy (Press release, Transgene, MAY 13, 2019, View Source [SID1234536206]).

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TG4050 is an individualized MVA-based immunotherapy derived from the myvac platform. It has been designed to stimulate and educate the immune system of patients to recognize and destroy tumor cells. Tumor cells accumulate mutations and each patient has a set of mutations that are unique to his tumor. TG4050 is designed to target a panel of patient specific mutations selected using a NEC’s Neoantigen Prediction System

"We are very pleased to have been granted an IND for TG4050 by the FDA allowing us to commence the first trial with our lead myvac candidate in ovarian cancer patients who have already received first-line treatment" said Maud Brandely, Chief Medical Officer of Transgene. "We believe individualized vaccination is a promising solution with significant potential to transform treatment outcomes for a wide range of solid tumors. With TG4050, we are confident that we can show that this therapeutic modality will improve patient outcome. We look forward to updating you on the progress of this clinical trial, which is expected to start later this year."

The Phase 1 clinical trial will evaluate the safety and the tolerability of TG4050 in patients with ovarian, fallopian or peritoneal serous cell carcinoma. Antitumor activity will also be measured. This multicenter, one-arm trial will recruit patients in the United States and Europe.

The study, sponsored by Transgene, will be co-financed by Transgene and its partner NEC, which will also support the trial by contributing to the therapeutic vaccine design and the selection of target neoantigens (see press release dated March 5, 2019.

Contacts Transgene:

Lucie Larguier Director Corporate Communications & IR +33 (0)3 88 27 91 04 [email protected] Media: Citigate Dewe Rogerson EU: David Dible/Sylvie Berrebi US: Marine Perrier-Barthez + 44 (0)20 7638 9571/+1 424 341 9140 [email protected]

About TG4050
TG4050 is an immunotherapy designed to stimulate the immune system of patients in order to induce a response that is able to recognize and destroy tumor cells in a specific manner. This personalized immunotherapy is developed for each patient, on the basis of mutations identified through sequencing of tumor tissue, prioritized using NEC’s Neoantigen Prediction System and delivered using the myvacTM technological platform which allows development and manufacture of a product that is specific to the patient within time frames compatible with clinical management.

About myvacTM myvacTM is a viral vector (MVA) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. The myvacTM-derived products are designed to stimulate the patient’s immune system, recognize and destroy tumors using the patient’s own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization know-how and unique manufacturing capabilities. Transgene has been awarded an "Investments for the Future" funding from Bpifrance for the development of its platform myvacTM.

About NEC’s Neoantigen Prediction System NEC’s neoantigen prediction utilizes its proprietary AI, such as graph-based relational learning, which is combined with other sources of data to discover candidate neoantigen targets. NEC comprehensively evaluates the candidate neoantigens with a primary focus placed on its in-house MHC-binding affinity prediction. These allow NEC to effectively prioritize the numerous candidate neoantigens identified in a single patient.

On May 13, 2019 Intrexon Corporation (NASDAQ: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported Randal J. Kirk, Chairman and Chief Executive Officer, will present at the Bank of America Merrill Lynch 2019 Healthcare Conference in Las Vegas on Wednesday, May 15th, at 3:40 p.m. Pacific Time (Press release, Intrexon, MAY 13, 2019, View Source [SID1234536205]). Mr. Kirk will focus on Intrexon Health’s development of targeted, controllable, multigenic therapeutics for the treatment of complex diseases and unmet medical needs, highlighting the therapeutic candidates currently in clinical development.

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A live webcast of the presentation will be available on the Investors section of Intrexon’s website at View Source, and a replay of the webcast will be available for 30 days following the event.

On May 10, 2019 Medtronic plc (NYSE:MDT) reported that it will report financial results for its fourth quarter and fiscal year 2019 on Thursday, May 23, 2019 (Press release, Medtronic, MAY 10, 2019, View Source;p=RssLanding&cat=news&id=2398239 [SID1234536173]). A news release will be issued at approximately 5:45 a.m. Central Daylight Time (CDT) and will be available at View Source The news release will include summary financial information for the company’s fourth quarter and fiscal year 2019, which ended on Friday, April 26, 2019.

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Medtronic will host a webcast at 7:00 a.m. CDT to discuss financial results for its fourth quarter and full fiscal year 2019. The webcast can be accessed at View Source on May 23, 2019.

Within 24 hours of the webcast, a replay and transcript of the prepared remarks will be available by clicking on the Investor Events link at View Source.

Looking ahead, Medtronic plans to report its fiscal year 2020 first, second and third quarter results on Tuesday, August 20, 2019, Tuesday, November 19, 2019, and Tuesday, February 18, 2020 respectively. Confirmation and additional details will be provided closer to the specific event.

On May 10, 2019 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported financial results and recent corporate highlights for the first quarter ended March 31, 2019 (Press release, Mustang Bio, MAY 10, 2019, View Source [SID1234536171]).

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The beginning of 2019 has been an exciting time for Mustang and provides great momentum for the rest of the year. Last month, we were thrilled to announce positive Phase 1/2 data published by our partner, St. Jude Children’s Research Hospital (St. Jude), in the New England Journal of Medicine, regarding the curative potential of MB-107, a lentiviral gene therapy for infants under the age of two years old with X-linked severe combined immunodeficiency, otherwise known as XSCID. Additionally, earlier this year, we partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop MB-108, oncolytic virus C134, for the treatment of glioblastoma multiforme. Most recently, Mustang announced the initiation of City of Hope’s CS1 chimeric antigen receptor (CAR) T cell therapy trial, MB-104. The Phase 1 clinical trial has begun enrolling patients and is the first autologous CAR T trial to target the CS1 protein, which is expressed by cancer cells in nearly all multiple myeloma patients."

Dr. Litchman continued, "We are also pleased to have raised a total of $69 million so far in 2019, bringing our total post-offering cash to approximately $89 million. This financial runway enables us to continue to progress the development of our eight gene and CAR T cell therapy product candidates. We anticipate achieving more exciting milestones in the coming months, including transferring the MB-107 IND from St. Jude to Mustang, filing Mustang’s first INDs for its CD123 and CS1 CAR T programs, and potentially reporting additional CAR T data in the fourth quarter."

Financial Results:

·As of March 31, 2019, Mustang’s consolidated cash, cash equivalents, short-term investments (certificates of deposit) and restricted cash totaled $41.1 million, compared to $34.6 million as of December 31, 2018, an increase of $6.5 million for the quarter.
·Research and development expenses were $7.0 million for the first quarter of 2019, compared to $4.3 million for the first quarter of 2018. Non-cash, stock-based compensation expenses included in research and development were $0.1 million for first quarter of 2019, compared to $1.5 million for the first quarter of 2018.
·Research and development expenses from license acquisitions totaled $0.5 million for the first quarter of 2019, compared to $0.1 million for the first quarter of 2018.
·General and administrative expenses were $2.3 million for the first quarter of 2019, compared to $2.1 million for the first quarter of 2018. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.7 million for the first quarter of 2019, compared to $0.5 million for the first quarter of 2018.
·Net loss attributable to common stockholders was $9.6 million, or $0.34 per share, for the first quarter of 2019, compared to $6.3 million, or $0.24 per share, for the first quarter of 2018.

Recent Corporate Highlights:

·In February 2019, Mustang announced that it partnered and entered into an exclusive worldwide license agreement with Nationwide Children’s Hospital to develop MB-108, an oncolytic virus (C134), for the treatment of glioblastoma multiforme. Mustang intends to combine MB-108 with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme.
·In April 2019, Mustang announced that it had entered into a $20 million debt financing agreement with Horizon Technology Finance Corporation. Fifteen million of the $20 million loan was funded upon closing. The remaining $5 million may be funded upon Mustang achieving certain predetermined milestones. In connection with the debt financing, Mustang issued Horizon warrants to purchase up to 288,184 shares of its common stock at an exercise price of $3.47 per share.
·Also in April 2019, the New England Journal of Medicine published St. Jude data from a Phase 1/2 clinical trial of a lentiviral gene therapy for the treatment of newly diagnosed infants under two years old with XSCID. Data demonstrate the lentiviral gene therapy achieved normalization of T-cell numbers in all eight newly diagnosed infants with XSCID to date, and disseminated infections resolved completely in all affected infants. Seven of the eight infants treated have developed normal IgM levels to date. Four of those seven infants have discontinued monthly infusions of intravenous immunoglobulin (IVIG) therapy to date. Three of those four infants who discontinued monthly IVIG infusions have responded to vaccines to date.
·In May 2019, Mustang completed an underwritten public offering, including a full over-allotment option exercise, that raised gross proceeds of $31.6 million, excluding underwriting discounts, commissions and other offering-related expenses.
·Also in May 2019, Mustang announced that City of Hope had begun enrolling patients with relapsed or treatment-resistant multiple myeloma in an innovative CS1 CAR T cell therapy (MB-104) trial.

On May 10, 2019 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), an oncology company focused on biomarker-defined cancers, reported its first quarter 2019 financial results for the period ended March 31, 2019 (Press release, Merrimack, MAY 10, 2019, View Source [SID1234536169]).

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"We continue to focus our efforts on completing the review of our strategic alternatives through which we are committed to optimizing value for our stockholders, including the preservation of potential contractual milestone payments Merrimack is eligible to receive," said Richard Peters, M.D., Ph.D., President and Chief Executive Officer of Merrimack. "To that end, we have recently implemented a series of measures to scale back operations and strengthen our balance sheet."

Corporate Update

As previously announced, Merrimack has engaged external advisors to explore the Company’s strategic alternatives. In the first quarter and more recently, Merrimack has implemented a series of measures designed to preserve its current resources as part of this ongoing process:

In April 2019, Merrimack terminated its Phase 1 study of MM-310 and discontinued the program, following a comprehensive review of available safety data indicating that the drug would not achieve an optimal therapeutic index. In parallel, the Company announced it would close out all remaining clinical activities;

In April 2019, Merrimack initiated a workforce reduction in connection with the Company’s decision to close out clinical activities and in line with prior cost-cutting measures. Merrimack estimates that it will incur approximately $1.5 million to $1.7 million in one-time charges in connection with this workforce reduction, which is expected to be substantially completed by May 31, 2019;

In April 2019, Merrimack retired its outstanding debt with Hercules Capital ahead of schedule, which totaled $16.0 million with interest and fees; and

In May 2019, Merrimack monetized certain assets to strengthen its cash position. This includes the sale of its equity position in Silver Creek Pharmaceuticals, resulting in $7.8 million in cash, and the auction of laboratory equipment from the Company’s research and development operations, resulting in approximately $1.3 million in cash.

Merrimack continues to operate with a narrow research footprint, focused on two preclinical programs: MM-401, an agonistic antibody targeting a novel immuno-oncology target, TNFR2; and MM-201, a highly stabilized agonist-Fc fusion protein targeting death receptors 4 and 5. Merrimack plans to prudently advance these programs, with deference to the Company’s ongoing strategic process.

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, held March 29 – April 3, 2019 in Atlanta, Georgia, Merrimack presented six posters highlighting its preclinical work, of which three featured MM-401 and two featured MM-201.

First Quarter 2019 Financial Results

The following summarizes Merrimack’s financial results for the three months ended March 31, 2019:

Research and development expenses for the three months ended March 31, 2019 were $6.4 million, compared to $13.1 million for the three months ended March 31, 2018. Research and development spending for the first quarter of 2019 was lower versus the comparable period in 2018, primarily due to phasing and close out of several clinical development programs;

General and administrative expenses for the three months ended March 31, 2019 were $3.7 million, compared to $4.3 million for the three months ended March 31, 2018. General and administrative spending for the first quarter of 2019 was less than expenditures over the comparable period in 2018, primarily due to a decrease in corporate expenses related to reduced headcount levels and stock-based compensation;

Net loss for the three months ended March 31, 2019 was $10.5 million, or $0.78 per share, compared to a net loss attributable to Merrimack’s continuing operations of $17.8 million, or $1.33 per share, for the three months ended March 31, 2018; and

As of March 31, 2019, Merrimack had 13.3 million shares of common stock, $0.01 par value per share, outstanding.

Financial Outlook

As of March 31, 2019, Merrimack had cash, cash equivalents and marketable securities of $58.5 million. Accounting for events subsequent to the quarter close, together with possible additional restructuring and cost-cutting measures that Merrimack could implement in the future, but excluding any potential contractual milestone payments, Merrimack continues to believe that its cash position has the potential to fund operations into at least the second half of 2022.

Merrimack remains eligible to receive additional milestone payments from Servier and Ipsen, resulting from Merrimack’s asset sale to Ipsen in 2017:

Merrimack is entitled to receive up to $5 million in milestones from Servier, triggered by Ipsen and Servier’s decision to progress their ongoing multi-part clinical trial evaluating ONIVYDE in small-cell lung cancer (SCLC) into the second randomized portion of the trial focused on efficacy; and

Merrimack is also entitled to receive up to an aggregate of $450 million in regulatory-based milestones from Ipsen, which Merrimack has said it expects to pass through to its stockholders, net of any taxes owed and subject to there being sufficient surplus at that time, consisting of:

$225 million upon approval by the FDA of ONIVYDE for the first-line treatment of metastatic adenocarcinoma of the pancreas, subject to certain conditions;

$150 million upon approval by the FDA of ONIVYDE for the treatment of SCLC after failure of first-line chemotherapy; and

$75 million upon approval by the FDA of ONIVYDE for an additional indication unrelated to those described above.