On August 10, 2020 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported financial results and operational updates for the second quarter ended June 30 (Press release, Turning Point Therapeutics, AUG 10, 2020, View Source [SID1234564368]).

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"We achieved many important milestones since our first quarter update in May, including advancing our pipeline of four drug candidates, sharing our preclinical combination data for repotrectinib in KRAS cancer models, raising gross proceeds of $374 million through our May stock offering, completing a strategic agreement with Zai Lab to develop repotrectinib in Greater China and strengthening our team with the hiring of our chief commercial officer, Andy Partridge," said Athena Countouriotis, M.D., president and chief executive officer.

"Turning to the second half of 2020, we remain focused on advancing our pipeline, as we continue to navigate the COVID-19 pandemic and monitor its impact to our timelines. For the TRIDENT-1 Phase 2 registrational study of repotrectinib, site activations and enrollment have improved since our last update in May, and we are planning to report early interim data from the study in the third quarter. Our goal is to achieve full global site activation in the TRIDENT-1 study in early 2021.

"Overall, I am pleased with how our team has adapted and advanced our clinical development during these challenging times and I want to again thank the dedicated health care providers worldwide who continue to treat patients suffering from COVID-19."

Second quarter and recent highlights include:

Progress in the TRIDENT-1 Phase 2 registrational study of repotrectinib, where the company anticipates reporting early interim data in the third quarter. The preliminary efficacy and safety data by physician assessment are expected to be from 30 to 40 patients across multiple Phase 2 cohorts, including registrational and exploratory cohorts. The company’s goal is to achieve full site activation in the study in early 2021.

Three additional trials ongoing, including the Phase 1/2 open-label study to assess repotrectinib in pediatric patients with ALK-, NTRK- or ROS1-positive advanced solid tumors; the Phase 1 study of TPX-0022, Turning Point’s MET/CSF1R/SRC inhibitor; and the Phase 1/2 study of TPX-0046, Turning Point’s RET/SRC inhibitor.

Completing an exclusive license agreement with Zai Lab for the development and commercialization of repotrectinib in Greater China, which includes mainland China, Hong Kong, Macau and Taiwan. Under the terms of the agreement, Zai Lab obtained exclusive rights to develop and commercialize repotrectinib in Greater China and Turning Point Therapeutics will receive a $25 million upfront payment, with the potential to receive up to an additional $151 million in development, regulatory and sales-based milestone payments. Turning Point will also be eligible to receive mid-to-high teen royalties based on annual net sales of repotrectinib in Greater China.

Appointing Andrew Partridge as executive vice president and chief commercial officer. Mr. Partridge has more than 20 years of global pharmaceutical sales and marketing experience leading more than 20 commercial launches across multiple indications, including oncology, hematology and rare diseases.
Second Quarter Financial Update
Operating expenses for the second quarter totaled $32.7 million compared to $18.5 million in the second quarter of 2019. Primary drivers of the year-over-year increase were investments made to develop repotrectinib, TPX-0022 and TPX-0046, as well as personnel expenses.

Excluding a one-time non-cash stock-based compensation charge in the first quarter of $31.4 million, non-GAAP operating expenses for the first half totaled $64 million compared to $32.5 million in the first half of 2019. Year-to-date net cash used in operating activities was $51.2 million.

Cash, cash equivalents and marketable securities at June 30 totaled $710.4 million, an increase of $329.6 million from Mar. 31 driven by proceeds from the company’s May stock offering. Turning Point Therapeutics projects its cash position funds current operations into 2023.

Upcoming Milestones
Key milestones anticipated into early 2021 include:

Early interim data from approximately 30 to 40 patients across multiple TRIDENT-1 Phase 2 cohorts in the third quarter.

Additional preclinical data highlighting the potential for repotrectinib to increase the effectiveness of KRAS-G12C inhibitors in the fourth quarter.

Early interim data from initial patients treated with TPX-0022 in the fourth quarter.

Submitting the IND for TPX-0131 by early 2021.

Early interim data from initial patients treated with TPX-0046 in early 2021

On August 10, 2020 Maryland has become a key part of the strategy for Kite, a Gilead company, and its mission to find a cure for cancer (Press release, Kite Pharma, AUG 10, 2020, View Source [SID1234563629]). As the California-based company focuses more and more on cutting-edge cell therapy research and manufacturing, Kite is expanding its toehold in the BioSpace Hotbed region known as BioCapital.

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Kite first moved into Maryland in 2018 following the forging of a cell therapy-focused partnership with the National Cancer Institute to develop adoptive cell therapies targeting patient-specific tumor neoantigens, which are mutations found on the surface of cancer cells that are unique to each person and tumor.

Kite announced it leased a 26,000 square-foot facility in Gaithersburg, Maryland, which is just miles away from Bethesda, Maryland, home of the NCI. The Gaithersburg site is focused on research and clinical manufacturing of TCR-based, adoptive t-cell therapies for solid tumor cancers.

After two years, Kite has more than 60 employees in its Gaithersburg facility. More employees will be needed at the facility as Kite invests more money to expand the facility to support the solid tumor research.

Matthew Levy, Associate Director, Talent Acquisition with Kite told BioSpace in an interview that the work being accomplished at the Gaithersburg facility is exciting due to the cutting-edge technology. As the facility expands, Levy said Kite will continue to hire the best and brightest to help the company in its mission of curing cancer.

With Maryland’s rich life sciences ecosystem, one that includes multiple government agencies like NCI, the National Institutes of Health and the U.S. Food and Drug Administration, Levy said Kite will be able to draw on a highly skilled talent pool as it expands its headcount in Gaithersburg. Not only does Maryland have a strong pool of talent already in place, Levy said the state is a nice place to live and offers residents a lot of cultural and recreational amenities. Although Kite has not identified a target number of employees it will hire for the Gaithersburg site, Levy said the company is already hiring for a number of positions there and will be expanding further next year.

Gaithersburg isn’t the only site Kite has in the state of Maryland. In 2019, the company announced it will build a third cell therapy manufacturing facility to support the company’s CAR-T manufacturing needs. Kite selected Frederick, Maryland in addition to its two other facilities in California and The Netherlands. "This will enable us to meet the future needs for cell therapies," Levy said.

Following the FDA-approval of Yescarta, its CAR-T treatment for some blood cancers, Levy said the company realized that having additional capacity in manufacturing would be beneficial for the cutting-edge treatment. Logistically, Levy said it was beneficial to have a manufacturing facility on the East Coast of the United States. He noted that the individualized CAR-T treatment cannot be stockpiled like some other medications can and having the third facility better serves Kite’s patients.

In addition to the manufacture of Yescarta, which was approved in 2017 two months after Kite was acquired by Gilead Sciences for $12 billion, the Maryland facility will also be ready to produce future commercial cell therapies.

"We’re sparing no expense to bring the best science to bear for patients," Levy said.

The 280,000 square-foot Frederick facility is expected to come online in 2022. The site is expected to employ a few hundred people over the next several years and Levy said the company is actively recruiting for positions there. Types of positions the company is recruiting for include manufacturing, facilities and engineering, supply chain and quality.

When it comes to potential candidates, Levy said the company is looking for candidates who are patient-centric in their work. Ideal candidates will be passionate about using their technical skills to benefit cancer patients. Levy stressed a strong sense of connectivity between the work performed at Kite and the patients the company serves. For example, Cell Therapy Specialists – Kite’s manufacturing technicians – literally hold patients’ cells in their hands; that personalized approach drives home the point that patients are at the center of everything the company does, Levy noted.

"If someone is looking for an opportunity to positively impact cancer patients through cutting-edge work in cell therapy, Kite is a great place to be," Levy said. "We really mean it when we say that cancer is personal to us."

On August 10, 2020 Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, reported the appointment of Gallia Levy, M.D., Ph.D., as chief medical officer (Press release, Sparx Therapeutics, AUG 10, 2020, View Source [SID1234563389]). Dr. Levy will be responsible for strategic and operational leadership across all functions in the product development lifecycle, including setting the global development strategy for current and future pipeline programs.

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"We are thrilled to welcome Dr. Gallia Levy to our growing gene therapy company striving to create a world where no life is limited by genetic disease," said Jeffrey D. Marrazzo, chief executive officer, Spark Therapeutics. "Dr. Levy’s passion for hematology and gene therapy research are immediately evident and is exactly the perspective needed to achieve our goal of unlocking the full potential of gene therapy. Especially during this pivotal time in hemophilia research, Dr Levy’s deep understanding of rare blood disorders and the community will help accelerate our ability to deliver potentially transformative gene therapies for hemophilia, while progressing potential gene therapies for other genetic disease across our pipeline."

Dr. Levy joins Spark Therapeutics from Genentech, a member of the Roche Group, where she served as the Vice President and Global Head of the Rare Blood Disorders franchise in Product Development. In this role, she was responsible for the clinical development of HEMLIBRA for hemophilia A as well as treatments for other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). She has played a key role in the evolution of gene therapy as a new modality within the Roche group.

"I’ve spent my career working to find new, innovative treatment approaches for patients affected by rare, life-altering disorders, and it is with great pride that I join the Spark team to help advance novel gene therapy programs and create next-generation solutions for patients," said Dr. Levy. "Spark Therapeutics shares the same affinity for breaking barriers and putting the patient first, and I look forward to what we will achieve together."

Dr. Levy first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications, and returned to Genentech in 2014 to lead the hemophilia program.

Dr. Levy is board-certified in hematology and holds an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan. She completed her residency in internal medicine at Stanford University and a fellowship in hematology and oncology at the University of California, San Francisco. She also holds an M.S. in of Molecular and Cellular Biology from the University of Paris, VI and a B.A. from the University of California, Berkeley.

On August 10, 2020 RhoVac reported that the first patient in the USA is included in clinical phase IIb study, called BRaVac (Press release, RhoVac, AUG 10, 2020, View Source [SID1234563381]). The first US clinic to include a patient in the study was Carolina Urologic Research Center. BraVac is a randomized, placebo controlled and double-blind study, with the primary objective of evaluating if treatment with the drug candidate RV001 can prevent or limit the development of advanced prostate cancer after curative intent treatment. BRaVac is an international, multi-centre study, which will recruit over 175 patients in six European countries (Denmark, Finland, Germany, Belgium, Sweden, and the UK) plus the USA. The study has already commenced in Denmark, Finland, Germany, Belgium and now in the USA. RhoVac anticipates recruitment to start very soon also in Sweden and the UK, and the company expects the phase IIb recruitment to be complete by end 2020, and the trial to conclude end 2021.

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Anders Månsson, CEO, comments: "We have truly reached a great milestone in now recruiting patients also in the USA, which is the largest and most important market in the world for our drug candidate RV001. I am very happy and proud that we have been able to keep our important clinical phase IIb trial going, and that more and more countries are joining the study, despite the impact of the pandemic."

On August 10, 2020 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company developing a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases, reported the closing of an underwritten public offering of 5,750,000 shares of its common stock at a public offering price of $18.00 per share, which includes the exercise in full by the underwriters of their option to purchase an additional 750,000 shares (Press release, Immunic, AUG 10, 2020, View Source [SID1234563380]).

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Immunic received total proceeds from the offering, before deducting the underwriting discounts and other offering expenses, of $103.5 million.

SVB Leerink acted as sole bookrunning manager for the offering. Wedbush PacGrow and Ladenburg Thalmann acted as co-managers for the offering.

The Company intends to use the net proceeds of the offering to fund the ongoing clinical development of its three lead product candidates, IMU-838, IMU-935 and IMU-856, including to investigate IMU-838 in a potential phase 3 program in relapsing-remitting multiple sclerosis, and for other general corporate purposes.

The securities described above were offered by Immunic, Inc. pursuant to a shelf registration statement filed by Immunic, Inc. with the Securities and Exchange Commission (SEC), which was declared effective on June 13, 2018. A final prospectus supplement and accompanying prospectus related to the offering was filed with the SEC on August 5, 2020 and is available for free on the SEC’s website at www.sec.gov.

Copies of the final prospectus supplement and the accompanying prospectus related to the offering may be obtained from: SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA, 02110, by email at [email protected], or by telephone at (800) 808-7525, ext. 6218.

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