Birmingham’s Southern Research appoints CEO, EVP

On May 14, 2021 Southern Research, the groundbreaking scientific discovery and research institution headquartered in Birmingham, on Thursday reported that its board of directors has appointed Josh Carpenter, Ph.D., as its new president and CEO, and Allen Bolton as its new executive vice president for Strategy and Finance (Press release, Southern Research Institute, MAY 14, 2021, View Source [SID1234580024]).

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Both appointments are effective June 1.

Founded in the Magic City in 1941, Southern Research is an independent, non-profit scientific research organization where more than 400 scientists and engineers work across three divisions: Life Sciences, Engineering, and Energy & Environment. Southern Research has attracted national research partnerships with leading industries in the fields of pharmaceuticals, biotechnology, defense, aerospace, the environment and energy.i

"Josh and Allen are forward-thinking leaders who are ready to chart a bold new course for Southern Research," stated University of Alabama at Birmingham President Ray L. Watts, who is chairman of the Southern Research board of directors. "They have the full support of the Board as they begin to lead an amazing team of scientists, engineers and innovators who are working to solve problems and change the world for the better."

Southern Research generates over $150 million in annual economic impact and supports more than 1,000 Alabama jobs.

"The work of Southern Research results in life-changing advancements and innovative solutions," said Southern Research board member Mark Crosswhite, who also serves as the chairman, president and CEO of Alabama Power Company. "Josh and Allen’s vision and expertise will play an important role in leading this institution forward as a continued center of excellence."

Carpenter most recently served as director of Innovation and Economic Opportunity for the City of Birmingham, where he led the City’s efforts in workforce development, COVID recovery and business expansion. Previously, he served as the director of External Affairs at UAB. He earned his doctorate in political economy from the University of Oxford where he studied on a Rhodes Scholarship.

"I am truly honored to take the helm of Southern Research and lead this incredible team that is finding solutions to improve people’s lives around the world. Southern Research has had 80 successful years, but I know the best is yet to come," commented Carpenter.

Bolton most recently served as senior vice president for Finance and Administration at UAB, where he was also a member of the board for Southern Research. He was previously an executive in finance and strategy at the Medical College of Wisconsin, and was senior associate dean for Administration and Finance at the UAB School of Medicine as well as executive administrator at the Comprehensive Cancer Center, the largest research center in the University of Alabama System.

"Discoveries made at Southern Research have provided breakthroughs in cancer research, pushed the boundaries of science and saved lives. I am grateful for this opportunity to work alongside dedicated and talented people who have an unmatched passion for science and discovery," concluded Bolton.

Regulus Therapeutics to Present at the Oppenheimer Rare & Orphan Disease Summit

On May 14, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs ("Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the Oppenheimer Rare & Orphan Disease Summit on Friday, May 21, 2021 at 11:35 A.M. ET (Press release, Regulus, MAY 14, 2021, View Source [SID1234580022]).

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A live webcast of the presentation will be available on the investor relations section of the Company’s website at www.regulusrx.com. A replay of the webcast will be archived for 30 days following the presentation date.

Protalix BioTherapeutics Reports First Quarter 2021 Financial Results
and Business Update

On May 14, 2021 Protalix BioTherapeutics, Inc. (NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx plant cell-based protein expression system, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Protalix, MAY 14, 2021, View Source [SID1234580019]).

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"While the receipt of the Complete Response Letter last month from the FDA was disappointing, we are encouraged that the FDA did not report any potential safety or efficacy concerns for PRX-102," said Dror Bashan, Protalix’s President and Chief Executive Officer. "We are working closely with the FDA and anticipate the required inspection and subsequent assessment will be completed once the FDA’s travel restrictions are lifted. We continue to advance our earlier stage pipeline and anticipate continued progress throughout 2021. We are grateful for the support of our clinicians, patients, shareholders, Board members, employees and external partners and look forward to building stockholder value," concluded Mr. Bashan.

2021 First Quarter and Recent Business Update

Regulatory Updates

On April 28, 2021, the Company, together with its development and commercialization partner, Chiesi Farmaceutici S.p.A., or Chiesi, announced the receipt of a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Biologics License Application (BLA) seeking accelerated approval of pegunigalsidase alfa, or PRX–102, for the proposed treatment of adult patients with Fabry disease. The CRL did not report any concerns relating to the potential safety or efficacy of PRX-102 in the submitted data package. In the CRL the FDA noted an inspection of the Company’s manufacturing facility in Carmiel, Israel, including the FDA’s subsequent assessment of any related findings is required before the FDA can approve the BLA. Due to travel restrictions relating to the COVID-19 pandemic, the FDA was unable to conduct the required inspection during the review cycle. The FDA explained that it will continue to monitor the public health situation as well as travel restrictions, and is actively working to schedule outstanding inspections.
Clinical Advancements

On February 23, 2021, the Company, together with Chiesi, announced positive topline results from the phase III BRIGHT clinical trial, a study designed to evaluate the safety, efficacy and pharmacokinetics of PRX–102 treatment, 2 mg/kg every four weeks, in up to 30 patients with Fabry disease previously treated with a commercially available enzyme replacement therapy (ERT) (agalsidase alfa – Replagal or agalsidase beta – Fabrazyme). Topline results indicate that 2 mg/kg of PRX-102 administered by intravenous infusion every four weeks was found to be well tolerated among treated patients, and stable clinical presentation was maintained in adult Fabry patients.
Corporate & Financial Developments

Given the receipt of the CRL, the Company believes that it is prudent to secure short-term funds in order to continue development of PRX-102 while waiting for the FDA’s required inspection and subsequent assessment described in the CRL. To do so, on May 13, 2021, the Company and Chiesi entered into a binding term sheet pursuant to which they amended the two exclusive license and supply agreements for PRX–102 in order to provide the Company with near-term capital. Chiesi agreed to make a $10.0 million milestone payment to the Company before the end of the second quarter in exchange for a $25.0 million reduction in a longer-term regulatory milestone payment in the Ex-US Exclusive License and Supply Agreement. All other regulatory and commercial milestone payments remain unchanged. The Company and Chiesi also agreed to negotiate certain manufacturing related matters.
On February 18, 2021, the Company announced the closing of a public offering of common stock raising gross proceeds of approximately $40.2 million before deducting the underwriting discount and estimated expenses of the offering.
On February 10, 2021, the Company entered into an exclusive partnership with SarcoMed USA Inc. for the worldwide development and commercialization of alidornase alfa, or PRX-110, for use in the treatment of any human respiratory disease or condition including, but not limited to, sarcoidosis, pulmonary fibrosis and other related diseases via inhaled delivery.
Financial Results

For the three months ended March 31, 2021, compared to the three months ended March 31, 2020

The Company recorded revenues from selling goods of $4.5 million during the three months ended March 31, 2021, a decrease of $0.5 million, or 10%, compared to revenues of $5.0 million for the same period of 2020. The decrease of $3.0 million in sales to Brazil was partially offset by an increase of $2.5 million in sales to Pfizer Inc. or Pfizer.
Revenue from licenses and R&D services was $6.8 million for the three months ended March 31, 2021, a decrease of $9.8 million, or 59%, compared to revenues from license and R&D services of $16.6 million for the same period in 2020. Revenues from license and R&D services are comprised primarily of revenues we recognized in connection with the license and supply agreements with Chiesi. The decrease resulted primarily from revenues for the three months ended March 31, 2020 recognized in connection with an updated costs estimation throughout the trials until completion, made in 2020, in the amount of $6.7 million and from revenues recognized in connection with the progress of our clinical trials that have been completed during 2020.
Cost of goods sold was $4.8 million for the three months ended March 31, 2021, an increase of $1.4 million, or 41%, from cost of goods sold of $3.4 million for the same period in 2020. The increase in cost of goods sold was primarily the result of higher manufacturing costs.
Research and development expenses were $7.1 million for the three months ended March 31, 2021, a decrease of $3.2 million, or 31%, compared to $10.3 million of research and development expenses for the same period of 2020. The decrease is primarily due to the completion of two out of the three phase III clinical trials of PRX–102 and reduced costs related to the BALANCE Study. The Company expects research and development expenses to continue to be its primary expense as it enters into a more advanced stage of preclinical and clinical trials for certain of its product candidates.
Selling, general and administrative expenses were $3.1 million for the three months ended March 31, 2021, a decrease of $0.1 million, or 3%, compared to $3.2 million for the same period of 2020.
Financial expenses, net were $1.8 million for the three months ended March 31, 2021, a decrease of $1.2 million, or 40%, compared to financial expenses net of $3.0 million for the same period of 2020. The decrease resulted primarily from a decrease in expenses related to the Company’s outstanding convertible notes equal to $1.3 million.
Cash, cash equivalents and short-term bank deposits were approximately $70.4 million on March 31, 2021. During the first quarter of 2021, the Company raised gross proceeds of $8.8 million from the sale of common stock under its ATM program and gross proceeds of $40.2 million via the public offering of its common stock.
Net loss for the three months ended March 31, 2021 was approximately $5.5 million, or $0.14 per share, basic and diluted, compared to a net income of $1.7 million, or $0.10 per share, basic and diluted, for the same period in 2020.
Conference Call and Webcast Information
The Company will host a conference call today, May 14, 2021 at 8:30 am Eastern Daylight Time, to review the clinical, corporate, and financial highlights, which will also be available by webcast. To participate in the conference call, please dial the following numbers prior to the start of the call:

Please access the websites at least 15 minutes ahead of the conference to register, download and install any necessary audio software.

The conference call will be available for replay for two weeks on the Events Calendar of the Investors section of the Company’s website, at the above link.

Prescient strengthens ties with leading CAR-T research centre

On May 14, 2021 Biotech company Prescient Therapeutics (ASX:PTX) reported that strengthening its ties in research and development with the world-renowned Peter MacCallum Cancer Centre (Peter Mac) (Press release, Prescient Therapeutics, MAY 14, 2021, View Source;utm_medium=rss&utm_campaign=prescient-strengthens-ties-with-leading-car-t-research-centre [SID1234580018]).

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Based in Melbourne, the Peter Mac centre is at the cutting edge of research and manufacturing advancements in CAR-T therapies – considered a breakthrough treatment in the fight against cancer.

The deal will see PTX get direct access to world-class facilities and a team of CAR-T experts led by Professor Phil Darcy, who will assist in the pre-clinical development of Prescient’s OmniCAR programs.

In that context, extending its partnership with the Peter Mac centre provides the ideal environment for further development of its next-generation OmniCAR programs.

Development opportunity
Prescient is currently developing three OmniCAR platforms used in the treatment of acute myeloid leukaemia (AML), Her2+ solid tumours and glioblastoma multiforme (GBM) – an aggressive form of cancer that usually begins in the brain.

The company is developing OminCAR "as a next generation CAR-T platform, capable of being deployed by other CAR-T and oncology companies under licence to advance their own programmes".

Known as Chimeric Antigen Receptor T-cells, CAR-T cells are specifically engineered to hunt and destroy abnormal cells, including cancerous cells.

And as part of its agreement with Peter Mac, it will now have two post-doctoral scientists and two research assistants working full-time on the development of its research suite.

"Prescient has also secured grant funding of $100,000 from the Federal Government’s Innovation Connections scheme towards this research," the company highlighted.

For Darcy, the use of Prescient’s OmniCAR treatment presents a pathway to achieve the replication of breakthrough CAR-T treatments in solid tumours.

"We are excited by the opportunity and potential offered by the OmniCAR platform to make headway into solid tumours and other blood cancers, and to greatly enhance and improve the clinical control and efficacy of existing CAR-T cancer therapies," Darcy said.

While PTX will get access to the Peter Mac centre’s leading team of researchers and scientists, it will fully own any intellectual property that arises from work carried out.

Prescient managing director Steven Yatomi-Clarke said the deal is reflective of the company’s strong medical networks, as it builds out its OmniCAR research programs "to the highest standard".

"This latest research program is an important part of Prescient’s development plans, which include institutional and commercial laboratories," he said.

"We continue to work very closely with Professor Darcy and the team at Peter Mac, as we progress this exciting development of controllable and adaptable next generation CAR-T therapies."

NeuBase Therapeutics to Participate in Upcoming Investor Conferences in May 2021

On May 14, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will participate in a virtual fireside chat at the RBC Capital Markets Virtual Global Healthcare Conference, as well as present a corporate overview and business update at the Oppenheimer Rare & Orphan Disease Summit, which are both being held in May 2021 (Press release, NeuBase Therapeutics, MAY 14, 2021, View Source [SID1234580017]).

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Conferences Details:

Event: RBC Capital Markets Virtual Global Healthcare Conference
Format: Fireside Chat
Date: Wednesday, May 19th
Time: 11:30 a.m. ET
Location: Webcast Link – or at the company’s website (click here)

Event: Oppenheimer Rare & Orphan Disease Summit
Format: Presentation
Date: Friday, May 21st
Location: Webcast Link – or at the company’s website (click here)