On May 25, 2021 INOVIO (NASDAQ:INO) a biotechnology company focused on bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, cancer, and HPV-associated diseases, reported that Dr. Joseph Kim, President and CEO, is scheduled to participate in a fireside chat and 1×1 investor meetings at the Jefferies 2021 Virtual Healthcare Conference on Tuesday, June 1, 2021 at 2:00 PM EDT (Press release, Inovio, MAY 25, 2021, View Source [SID1234580544]). A webcast of the event will be available at this link: View Source

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About INOVIO’s DNA Medicines Platform

INOVIO has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including coronaviruses associated with MERS and COVID-19 diseases being developed under grants from the Coalition for Epidemic Preparedness Innovations (CEPI) and the U.S. Department of Defense. DNA medicines are composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

INOVIO’s DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using INOVIO’s proprietary hand-held smart device called CELLECTRA. The CELLECTRA device uses a brief electrical pulse to reversibly open small pores in the cell to allow the plasmids to enter, overcoming a key limitation of other DNA and other nucleic acid approaches, such as mRNA. Once inside the cell, the DNA plasmids enable the cell to produce the targeted antigen. The antigen is processed naturally in the cell and triggers the desired T cell and antibody mediated immune responses. Administration with the CELLECTRA device ensures that the DNA medicine is efficiently delivered directly into the body’s cells, where it can go to work to drive an immune response. INOVIO’s DNA medicines do not interfere with or change in any way an individual’s own DNA. The advantages of INOVIO’s DNA medicine platform are how fast DNA medicines can be designed and manufactured; the stability of the products, which do not require freezing in storage and transport; and the robust immune response, safety profile, and tolerability that have been observed in clinical trials.

With more than 3,000 patients receiving INOVIO investigational DNA medicines in more than 7,000 applications across a range of clinical trials, INOVIO has a strong track record of rapidly generating DNA medicine candidates with potential to meet urgent global health needs.

On May 25, 2021 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported that Jack Khattar, President and CEO of Supernus Pharmaceuticals, will present a company overview at the Jefferies Virtual Healthcare Conference on Tuesday, June 1, 2021, at 1:30 p.m. ET (Press release, Supernus, MAY 25, 2021, View Source [SID1234580560]).

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A live webcast of the presentation can be accessed by visiting Events & Presentations in the Investor Relations section on the Company’s website at www.supernus.com. An archived replay will be available for 60 days on the Company’s website following the conference.

On May 25, 2021 Cellaria Inc (Wakefield, MA, USA), a scientific innovator with breakthrough tools for cancer research, now offers five stable, reported that fully authenticated lung cancer cell models to support the development of more effective and targeted drug therapies (Press release, Cellaria, MAY 25, 2021, View Source [SID1234580576]). Lung cancer was responsible for around 1.80 million deaths globally in 20201, more than any other cancer, making it an important area of clinical research. Each Cellaria model is securely linked to a well-defined patient tumor enabling the in vitro study of disease progression and drug response in a specific patient biology with defined genetic and epigenetic characteristics. Such studies accelerate progress towards better therapies for specific patient populations.

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Cellaria’s lung cancer cell models are all derived from tumors associated with non-small cell lung cancer (NSCLC), which accounts for around 85% of lung cancer cases. In recent years considerable progress has been made towards optimizing treatment for different subtypes of NSCLC with the targeting of specific genetic mutations proving particularly productive. Cellaria’s new lung cancer models – Bastion, Boulder, Pincer and Throne – join Jacket (an existing model), to provide a diverse patient cohort for the extension of such studies. All the patients within the cohort were over 60 years of age and had a history of smoking, but they exhibited notable, clinically significant differences including:

Documented variation in consumption and duration of smoking
Comorbidities – Throne had a previous diagnosis melanoma
Throne and Jacket have a KRAS genetic mutation, Pincer, Boulder and Bastion did not.
All Cellaria models are extremely stable and supplied with a Certificate of Analysis, to support optimal application. In addition, Cellaria produces drug response reports showing data for three baseline relevant treatments. For the lung cancer cohort this report includes results for etoposide and cisplatin and for two targeted treatments: 1) larotrectinib which is prescribed for patients with a neurotrophic receptor tyrosine kinase (NRTK) gene fusion and 2) erlotinib, which is prescribed for patients with a KRAS mutation. Detailed information such as this makes it straightforward to use Cellaria models effectively from the outset to implement reproducible and relevant research and maximize the benefit of in vitro studies.

"With the increase in availability of targeted therapeutics like larotrectinib and erlotinib, patient-specific models are adding essential utility," comments David Deems President and Founder, Cellaria Inc. "Jacket has mutations that make the patient a candidate for either larotrectinib or erlotinib. This unique patient-derived model enables the interrogation of each drug’s performance with the model to determine differences."

To find out more about the new lung cancer models view our product overviews here.

1 WHO Cancer Fact Sheet Detail. 3rd March 2021. Available to view at: View Source

On May 25, 2021 Rain Therapeutics Inc. ("Rain"), a clinical-stage company developing precision oncology therapeutics, reported that financial results for the first quarter that ended March 31, 2021, along with an update on the company’s key developments, business operations and upcoming milestones (Press release, Rain Therapeutics, MAY 25, 2021, View Source [SID1234580592]).

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"We are thrilled with the progress made so far this year, which included the successful completion of our IPO in April 2021," said Avanish Vellanki, co-founder and chief executive officer of Rain. "The net proceeds from the IPO position Rain well to execute on our pipeline of precision oncology programs, led by RAIN-32, an oral MDM2 inhibitor. We also strengthened our leadership team and advisors with the appointments of Dr. Robert Doebele as president, Dr. Richard Bryce as chief medical officer, Stefani Wolff to the board of directors and Dr. Simon N. Powell to the scientific advisory board. We are confident these industry leaders will be invaluable as we continue to evaluate RAIN-32 in various clinical trials for a variety of MDM2-amplified oncology indications and advance the RAD52 research program towards the clinic. I am extremely proud of the achievements of our team over the past year despite the challenges that come with a global pandemic."

Mr. Vellanki continued, "We are looking forward to the remainder of 2021, as we launch our Phase 3 trial evaluating RAIN-32 as a potential treatment for WD/DD liposarcoma, as well as in MDM2-amplified solid tumors in an open-label, tumor-agnostic Phase 2 basket trial. We also continue to expect the start of a third clinical study, in intimal sarcoma by early 2022."

Key Developments and Operational Updates

Initial Public Offering
Rain completed its IPO in April 2021 in which the company received net proceeds of $121.9 million, net of estimated offering costs from the sale of a total of 7,845,011 shares in the IPO, including shares sold pursuant to the exercise of the underwriters’ option to purchase additional shares. The shares began trading on The Nasdaq Global Select Market on April 23, 2021 under the ticker symbol "RAIN."
Appointment of Robert Doebele, M.D., Ph.D. as President
Rain announced the appointment of Rain co-founder Robert Doebele, M.D., Ph.D. as president effective on May 14, 2021. Dr. Doebele co-founded Rain with Mr. Vellanki in April 2017 and has served as executive vice president and chief scientific officer ("CSO") since September 2020. As president, Dr. Doebele will play a critical role in shaping the company’s corporate strategy and operations and will continue to provide his precision oncology expertise to advance Rain’s clinical pipeline and lead the company’s research and development efforts as CSO.
Appointment of Richard Bryce, MBChB, as Executive Vice President and Chief Medical Officer
Rain announced the appointment of Richard Bryce, MBChB, as executive vice president and chief medical officer on April 7, 2021. Dr. Bryce brings over 30 years of experience in oncology, clinical research and biopharmaceutical roles to the Rain team. He joined the company as it prepares to initiate multiple clinical studies for RAIN-32, an oral MDM2 inhibitor, including a Phase 3 clinical trial for patients with WD/DD liposarcoma.
Appointments of Stefani Wolff to the board of directors and Simon N. Powell, M.D., Ph.D. to the scientific advisory board
Rain announced the appointments of Stefani Wolff to the board of directors and Simon N. Powell, M.D., Ph.D. to the scientific advisory board on April 13, 2021. Stefani Wolff brings extensive leadership across clinical development and into commercial launch preparation which will be indispensable as Rain continues to plan for the future of RAIN-32. In addition, Dr. Powell’s appointment to Rain’s scientific advisory board is significant given his role in discovering RAD52 as a synthetic lethal hit for critical homologous recombination deficiencies in cancer.
Anticipated Near-term Milestones

RAIN-32 WD/DD Liposarcoma
Phase 3 trial start expected to commence in 2H 2021
RAIN-32 MDM2 Basket Study
Phase 2 trial start expected to commence in 2H 2021
RAIN-32 Intimal Sarcoma
Phase 2 trial start expected to commence by early 2022
RAD52 Research Program HRD+ Tumors
Lead candidate selection expected in 2022
First Quarter Financial Results

For the first quarter ended March 31, 2021, Rain reported a net loss of $6.8 million, as compared to a net loss of $2.6 million for the first quarter of 2020. Net loss per share for the first quarter of 2021 was $1.93, as compared to a net loss per share of $0.82 for the first quarter of 2020.

Research and development ("R&D") expenses were $5.3 million for the first quarter of 2021, as compared to $1.8 million for the first quarter of 2020. The increase in R&D expenses was primarily due to increases in third-party R&D costs for our lead product candidate, RAIN-32, as well as personnel costs. Non-cash stock-based compensation expense included in R&D expenses was approximately $0.1 million in each of the first quarters of 2021 and 2020.

General and administrative ("G&A") expenses were $1.5 million for the first quarter of 2021, as compared to $0.7 million for the first quarter of 2020. The increase in G&A expenses of $0.8 million was primarily due to increases in various third-party general and administrative costs, as well as personnel costs. Non-cash stock-based compensation expense included in G&A expenses was approximately $0.1 million in each of the first quarters of 2021 and 2020.

Total non-cash stock-based compensation expense was approximately $0.2 million in each of the first quarters of 2021 and 2020.

As of March 31, 2021, Rain had $53.1 million in cash and cash equivalents which does not include the net proceeds from the IPO in April 2021 of $121.9 million.

As of May 20, 2021, Rain had 26.5 million shares of common stock outstanding which includes a total of 7,845,011 shares sold in Rain’s IPO in April 2021.

The Company expects the full year 2021 net cash used in operating and investing activities to be approximately $50.0 million to $60.0 million and a projected year end cash balance of approximately $137.0 million to $147.0 million in cash and cash equivalents.

On May 25, 2021 AstraZeneca reported that $39 billion acquisition of Alexion is now being investigated by the UK’s Competition and Markets Authority (CMA), to determine if the deal is anti-competitive (Press release, AstraZeneca, MAY 25, 2021, View Source [SID1234583260]). On March 17, 2021, it was reported that the U.S. Federal Trade Commission (FTC) organized a multinational working group to evaluate pharmaceutical mergers.

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Along with the FTC, the group included the CMA, the Canadian Competition Bureau, the European Commission Directorate General for Competition, the U.S. Department of Justice Antitrust Division, and Offices of State Attorneys General.

The commission’s goal is to "identify concrete and actionable steps to review and update the analysis of pharmaceutical mergers."

At that time, the FTC was reviewing the AstraZeneca-Alexion deal. Alexion had filed with the U.S. Securities and Exchange Commission (SEC) at the time, indicating it planned to withdraw and refile its notification and report form under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, "in order to give the FTC additional time to review the proposed transaction."

The deal has since received the greenlight from the U.S., Canada, Brazil and Russia, but decisions are still pending from the UK, the EU and Japan. The deal was announced in December 2020. AstraZeneca wanted to bolster its immunology presence.

Two weeks ago, Alexion shareholders approved the deal.

"We’re very pleased with today’s affirmative shareholder vote, which brings us one step closer to completing a transaction that will accelerate the combined company’s ability to develop and provide access to life-changing medicines for patients with rare and devastating diseases around the world," said Ludwig Hantson, Alexion’s chief executive officer, at the time.

Under the terms of the deal, Alexion shareholders would receive $60 in cash and 2.1243 AstraZeneca American Depositary Shares (ADS), with each ADS worth half of an ordinary AstraZeneca share. That comes to about $175 per share.

AstraZeneca’s market presence has typically been on oncology, cardiovascular, renal and metabolism and respiratory diseases. It increased its immunology R&D efforts in immune-mediated diseases.

Alexion’s focus is on complement inhibition, a part of the human immune system. Its branded products focus on immune-mediated rare diseases caused by abnormal activation of the complement system. These include Soliris (eculizumab), a first-in-class anti-complement component 5 (C5) monoclonal antibody.

Soliris is approved in several countries for paroxysmal hemoglobinuria (PNH), atypical hemolytic uremic syndrome, generalized myasthenia gravis and neuromyelitis optica spectrum disorder. The company’s Ultomiris (ravulizumab) is a second-generation C5 monoclonal antibody. Other products include Strensiq (asfotase alfa) and Kanuma (sebelipase alfa).

If the merger is finalized, the two companies will work to advance Alexion’s pipeline of 11 molecules across more than 20 clinical programs, many in rare diseases. Alexion’s immunology expertise is expected to support AstraZeneca’s presence in immunology and rare diseases.

At the time of the announcement, Pascal Soriot, AstraZeneca’s chief executive officer, said, "Alexion has established itself as a leader in complement biology, bringing life-changing benefits to patients with rare diseases. This acquisition allows us to enhance our presence in immunology. We look forward to welcoming our new colleagues at Alexion so that we can together build on our combined expertise in immunology and precision medicines to drive innovation that delivers life-changing medicines for more patients."