Transgene presents initial Phase I data of TG6002, highlighting the potential of the intravenous administration of its oncolytic viruses

On April 9, 2021 Transgene (Paris:TNG) (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapeutics against cancer, reported initial promising results from a Phase I study combining intravenous (IV) oncolytic virus TG6002 and oral 5-FC in patients with advanced gastrointestinal carcinomas (Press release, Transgene, APR 9, 2021, View Source [SID1234621819]). These data provide a clinical proof of concept for Transgene’s double deleted VVcopTK-RR- patented virus backbone: after IV administration, TG6002 reached the tumor, multiplied within tumor cells, and induced the local expression of its payload (the FCU1 gene).

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These results will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) virtual meeting taking place from April 10-15, 2021.

DATA CONFIRM THAT THE CHEMOTHERAPY AGENT 5-FU IS PRODUCED IN PATIENTS’ TUMORS AFTER INTRAVENOUS ADMINISTRATION

TG6002 is a novel oncolytic virus that has been engineered to combine multiple mechanisms of action. It has been designed to:

selectively replicate within cancer cells. This is due to the deletion of the viral genes encoding TK and RR, which reduces the virus’s ability to grow in normal cells. This selective viral replication leads to the breakdown of the infected tumor cells in a process called oncolysis,
prime an immune response against the primary tumor and metastases,
and to induce the local expression of a biologically active enzyme able to convert 5-FC into its active cytotoxic metabolite 5-FU, directly in the tumor.
The data demonstrate that high concentration and continuous production of 5-FU chemotherapy can be obtained within the tumors through the local conversion of the pro-drug 5-FC (administered orally). This mechanism of action is based on the in-tumor expression of the proprietary FCU1 gene that has been integrated within the genome of TG6002.

In this study, extensive analyses are being performed including metastasis biopsy with synchronous blood sampling, assessment of virus presence, quantification of 5-FC and 5-FU and assessment of neutralizing antibody titers.

These analyses have allowed Transgene to document TG6002’s pharmacokinetics (PK) and biodistribution, and the functioning of the FCU1 gene when given by IV administration.

Detailed results:

✔ TG6002 infects tumors after intravenous administration, remains active and effectively express FCU1 gene selectively in tumor tissue;
✔ Absence of widespread virus distribution in the body and association of FCU1 activity with high virus concentration in tumor tissue suggest that the replication of TG6002 is concentrated in tumor cells;
✔ None of the patients presented clinical signs of extra-tumoral dissemination of the virus suggesting a high tumor specificity of the viral replication;
✔ The study is continuing with escalating dosing of TG6002.

CLINICAL PROOF OF CONCEPT OF THE FEASIBILITY OF THE IV ADMINISTRATION OF TRANSGENE’S PROPRIETARY ONCOLYTIC VIRUS

To-date, the only oncolytic virus that has received regulatory approval is only approved for intra-tumoral administration, restricting its use to superficial lesions.

Transgene aims to enlarge the number of solid tumors, such as gastro-intestinal tumors, that could be addressed by an oncolytic virus, by developing oncolytics that can be administered intravenously.

The findings that will be presented at AACR (Free AACR Whitepaper) demonstrate the relevance of intravenous administration of Transgene’s next generation oncolytic viruses including TG6002.

These data also suggest that candidates derived from Transgene’s unique Invir.IO platform could also be given intravenously, extending the use of these therapies to a broad range of solid tumors.

Title of the poster: "Oncolytic virus TG6002 locates to tumors after intravenous infusion and induces tumor-specific expression of a functional pro-drug activating enzyme in patients with advanced gastrointestinal carcinomas"
Authors: Kaidre Bendjama, Philippe Cassier, Victor Moreno, Bernard Doger, Emiliano Calvo, Maria De Miguel, Christiane Jungels, Philippe Erbs, Damien Carpentier, Alain Sadoun.
Abstract/Poster Number: LB179
Session: PO.IM02.11 – Vaccines
The e-poster presentation will be available on the AACR (Free AACR Whitepaper) website beginning at 8:30 am US EDT on Saturday, April 10, until Monday, June 21. The text of this abstract will be posted at 12:01 am US EDT on Friday, April 9 on the AACR (Free AACR Whitepaper) website.

About the trial (NCT03724071)
This trial is a single-arm open-label Phase I/II trial evaluating the safety and tolerability of multiple ascending doses of TG6002 administered intravenously in combination with oral 5-FC, a non-cytotoxic pro-drug that can be converted in 5-FU, its active metabolite. Based on the safety profile of TG6002, several dose levels have been added to the initial Phase I clinical protocol. At the end of this Phase I part, Phase II patients will receive the recommended dose of TG6002. The trial has safety as primary endpoint for the Phase I part and efficacy for the Phase II part. The trial also evaluates pharmacokinetic properties and biodistribution of TG6002, along with immune modulation of the tumor micro-environment. This European study will enroll up to 40 patients suffering from advanced gastrointestinal carcinomas who have failed and/or are intolerant to standard therapeutic options in the Phase I part. Patients with colon cancer and liver metastases will be enrolled in the Phase II part.

Dr. Philippe Cassier, M.D., PhD, head of the early-phase trials unit at Centre Léon Bérard (Lyon, France) is the principal investigator of the trial.

About TG6002
TG6002 has been engineered to directly kill cancer cells (oncolysis), to enable the production of a chemotherapy agent (5-FU) within the tumor, and to elicit an immune response by the body against the tumor cells. In preclinical experiments, TG6002 has been shown to induce the shrinkage of the primary tumor as well as the regression of distant metastases (Foloppe, et al., Molecular Therapy Oncolytics, View Source).

The production of 5-FU directly in the tumor aims to achieve a better anti-tumoral effect with limited chemotherapy-induced side effects.

TG6002 induces the production of 5-FU in the cancer cells it has infected, by enabling the local conversion of the pro-drug 5-FC (administered orally) into 5-FU. 5-FU is a common chemotherapy agent for patients with gastro-intestinal cancers. This mechanism of action is based on the in-tumor expression of the proprietary FCU1 gene that has been encoded in the genome of TG6002, taking advantage of the virus selective replication in the tumor cells.

When administered systemically, 5-FU is associated with side effects that can lead to treatment discontinuation. With TG6002, 5-FU is produced within the tumor where it is expected to be present at a high concentration level in contrast to the very low levels anticipated in the rest of the patient’s body.

Sana Biotechnology Presents Preclinical Data at American Association for Cancer Research Annual Meeting 2021

On April 9, 2021 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported data from its T cell programs are being presented at the virtual American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 (Press release, Sana Biotechnology, APR 9, 2021, View Source [SID1234584004]).

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Sana is investing in multiple platform technologies to engineer cells, and several of these have the potential to address unmet needs for patients with cancer. Two of these platforms are highlighted in posters to be presented at AACR (Free AACR Whitepaper). Sana’s fusogen platform has the potential to deliver genetic payloads to specific cells in vivo, or inside a patient’s body, including delivery to T cells of the gene needed to make a chimeric antigen receptor (CAR). Sana’s hypoimmune platform has the potential to enable transplants of allogeneic cells without immunosuppression, including allogeneic CAR T cells.

"We are excited to share data for the first time at AACR (Free AACR Whitepaper), as we are optimistic that Sana’s platforms can be applied to help cancer patients," said Steve Harr, MD, Sana’s President and CEO. "CAR T cells have shown enormous potential for certain cancer patients, and Sana’s goal is to make better and more accessible CAR T therapies so that more patients can benefit."

"The data presented in these abstracts highlight the potential of our fusogen and hypoimmune platforms to make high quality, functional CAR T cells without the logistical complexities of autologous CAR T cell therapies," said Terry Fry, MD, Sana’s Head of T Cell Therapeutics. "The goal of our CD8-targeted fusogen program is to deliver the CAR gene directly to the T cell in vivo, and our data highlight the potential of these genetically modified CAR T cells to kill tumors. Separately, we modify gene expression in donor T cells to create hypoimmune allogeneic CAR T cells, and data highlight the potential of these cells to evade both the innate and adaptive immune systems while retaining anti-tumor effects. These results represent important progress in validating Sana’s platforms as we continue towards the clinic."

Data from two late-breaker abstracts were made available to the AACR (Free AACR Whitepaper) community today and are outlined below. The full posters will be available to conference participants online beginning Saturday, April 10 at 8:30 a.m. Eastern Time.

In vivo CAR T therapy: targeted in vivo gene delivery of a CAR using a CD8-specific fusogen results in tumor eradication
Authors: Terry Fry, MD et al.

Key takeaways include:

A single intravenous delivery of a CD8 fusogen containing a second-generation CD19 CAR transgene resulted in the generation of CD8 CAR Ts that eradicated the CD19+ tumor xenografts;
CD8 fusogen delivery resulted in a high percentage of T cells engineered to express the CAR with specificity for the CD8+ cells; and
The fusogen was able to generate a functional CAR response regardless of prior activation status of the T cells.
Overexpression of CD47 protects hypoimmune CAR T cells from innate immune cell killing
Authors: Sonja Schrepfer, MD, PhD et al.

Key takeaways include:

Innate immune cell assays show that CD47 overexpression protects HLA-I/II deficient CAR T cells from natural killer cell and macrophage killing both in vitro and in vivo;
Hypoimmunogenic CAR T cells have shown the ability to functionally evade the innate and adaptive immune system in allogeneic recipients with cytotoxic anti-tumor capacity; and
Hypoimmune CAR T cells have the potential to provide universal CAR T cells that are able to persist without immunosuppression.

Miravo Healthcare™ to Present at Upcoming Investor Conferences

On April 9, 2021 Nuvo Pharmaceuticals Inc. (TSX:MRV; OTCQX:MRVFF) d/b/a Miravo Healthcare (Miravo or the Company), a Canadian focused healthcare company with global reach and a diversified portfolio of commercial products, reported Jesse Ledger, Miravo’s President & Chief Executive Officer and Mary-Jane Burkett, Miravo’s Vice President & Chief Financial Officer will be presenting at upcoming investor conferences (Press release, Nuvo Pharmaceuticals, APR 9, 2021, View Source [SID1234580415]).

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Bloom Burton & Co. Healthcare Investor Conference

The Bloom Burton & Co. Healthcare Investor Conference brings together U.S., Canadian and international investors who are interested in the latest developments in the Canadian healthcare sector. Attendees will have an opportunity to obtain corporate updates from the premier Canadian publicly traded and private companies through presentations and private meetings.

Miravo Healthcare Presentation

DATE: Wednesday, April 21st, 2021
TIME: 10:00 a.m. EST
LINK: Conference Registration

Planet MicroCap Showcase

The Planet MicroCap Showcase VIRTUAL EVENT brings together the most promising companies and the top dealmakers in MicroCap Finance for three (3) days of company presentations, 1:1 meetings and educational panels in The Premier Virtual Event in MicroCap Finance.

Miravo Healthcare Presentation

DATE: Thursday, April 22nd, 2021
TIME: 10:00 a.m. EST
LINK: Miravo Presentation

Werewolf Therapeutics looks to make a killing in the public markets with a $100M IPO

On April 9, 2021 Werewolf Therapeutics reported that now wants an IPO (Press release, Werewolf Therapeutics, APR 9, 2021, View Source [SID1234577882]).

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If you need to bring your breakthrough cell and gene therapy to market successfully, only Marken provides you with a single cell and gene supply chain solution capable of taking you wherever you want to go. Talk to a Marken expert today.
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The early-stage biotech is working on shape-shifting cytokine drugs, i.e., ones that deliver the promise of cytokines without their nasty side effects. Known as Indukines, Werewolf’s candidates are "switched off" when they’re given to patients and activate only when they arrive in cancer cells.

Cytokines, such as interleukin-2, emerged decades ago as promising cancer treatments. But they evolved as signaling molecules and not as drugs, so it’s no surprise that they’re missing some key pharmaceutical features that would make them good medicines.

"These mechanisms are a very powerful way to stimulate the immune system to attack tumors, but unfortunately, a number of cytokines can activate the immune system in an adverse way where it can attack normal tissues," Werewolf CEO Daniel Hicklin, Ph.D., said in January. Werewolf’s protein engineering technology is designed to limit its drugs’ effects on healthy tissues.

The biotech, which is preclinical but wants the cash to chase these drugs into the clinic, got off a $72 million raise in January just over a year after it nabbed $56 million series A back in November 2019. Now, according to its SEC-1 filing, it wants a $100 million IPO, though don’t be surprised if, like so many other biotechs over the past year, it ends up getting more than that.

Its two leading assets, WTX-124 and WTX-330, are seeking INDs with the FDA from next year, according to its filing, after which it plans to conduct phase 1/1b trials in patients with multiple tumor types both as standalone treatments and in combination with an immune checkpoint inhibitor.

It also has a third asset, WTX-613, which is much further back in the pipeline, with clinical trials not slated until 2023. This drug is designed to be a systemically delivered, conditionally activated interferon alpha, or IFN-a, molecule for the treatment of solid tumors and blood cancers.

Other players hoping to make better cytokines for cancer treatment include Synthekine, which launched in September with $82 million, and Bright Peak Therapeutics, which debuted in July with $35 million.

And Sanofi bet big on the approach in December 2019 when it inked a $2.5 billion deal to acquire Synthorx, with some early data dropping this weekend at the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

Bionomics Successfully Completes A$22.9 million Equity Raise

On April 9, 2021 Bionomics Limited (ASX: BNO,OTCQB:BNOEF) (Bionomics) reported that it has completed its 1 for 6 pro rata non–renounceable entitlement offer that was announced on 8 March 2021 (Entitlement Offer) and concurrent placement that was announced on 17 March 2021 (Concurrent Placement) which collectively raised approximately A$22.9 million (Press release, Bionomics, APR 9, 2021, View Source [SID1234577811]).

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The Entitlement Offer closed at 5.00pm (Adelaide time) on 30 March 2021 and raised approximately A$20.4 million from the offer of approximately 140.9 million new fully paid ordinary shares in Bionomics (New Shares) at the offer price of A$0.145 per New Share.

The Entitlement Offer was well supported by eligible shareholders who applied for approximately 85.6 million shares aggregating to approximately A$12.4 million of New Shares pursuant to their entitlements (representing a take up rate of approximately 60.7%).

Eligible shareholders subscribed for a further approximately 70.3 million additional New Shares in excess of their entitlement (up to a maximum of 100% of their entitlement) aggregating to approximately A$10.2 million in excess of their entitlement through the Oversubscription Facility. Applications under the Oversubscription Facility were in excess of the approximately 55.3 million shares or A$8 million shortfall and were scaled back in accordance with the terms of the Entitlement Offer on a pro rata basis.

Commenting on the outcome, Bionomics’ Executive Chairman, Dr Errol De Souza said, "Following on from the strong support received under the Company’s oversubscribed capital raise in February 2021, the success of the Entitlement Offer and Concurrent Placement demonstrates the confidence of Bionomics’ shareholders in the direction of the Company and the opportunities presented by progressing the development programme for BNC210 for the treatment of PTSD. We remain on track to starting the Phase 2b BNC210 PTSD trial in mid-2021".

As announced on 17 March 2021, Bionomics also conducted the Concurrent Placement concurrently with the Entitlement Offer pursuant to which certain investors were entitled to apply for new shares at the same price and on the same pro rata basis as was offered to subscribers under the Entitlement Offer. The investors to whom this Concurrent Placement offer was extended were those who participated in the Bionomics placement announced on 9 February 2021 and which completed on 2 March 2021, but who were unable to participate in the Entitlement Offer due to having a registered address outside of Australia or New Zealand.

The Concurrent Placement which was made on substantially the same terms as the Entitlement Offer raised approximately A$2.5 million from the offer of approximately 17.2 million new fully paid ordinary shares in Bionomics (New Concurrent Shares) at the offer price of A$0.145 per New Concurrent Share. New Concurrent Shares applied for under the Concurrent Placement were scaled back on a pro rata basis in the same manner as shareholders who applied under the Entitlement Offer.

The New Concurrent Shares issued under the Concurrent Placement were allocated from Bionomics’ expanded placement capacity following the Entitlement Offer.

Allotment of New Shares and New Concurrent Shares under the Entitlement Offer and the Concurrent Placement occurred on Thursday, 8 April 2021.

New Shares and Concurrent New Shares issued under the Entitlement Offer and the Concurrent Placement rank equally in all respects with existing shares. Shareholders should confirm their holding before trading in New Shares and Concurrent New Shares.