On May 4, 2021 Exelixis, Inc. (Nasdaq: EXEL) and GamaMabs Pharma SA reported that they have entered into an agreement under which Exelixis will, upon the future closing of the asset purchase and subject to certain conditions to closing, acquire all rights, title and interest in GamaMabs’ AMHR2 antibody technology (Press release, Exelixis, MAY 4, 2021, View Source [SID1234579096]). Exelixis will pay GamaMabs $5 million upon signing of the agreement, make additional payments upon completion of closing conditions, and make additional milestone payments after closing, contingent upon various events. Once the transfer is completed, Exelixis will control 100% of GamaMabs’ AMHR2 franchise technology including all assets pertaining to GamaMabs’ monoclonal antibody drug product murlentamab (GM-102).

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"GamaMabs has generated a compelling body of preclinical data supporting the potential of AMHR2 as a target for novel oncology therapies and demonstrated the safety of an anti-AMHR2 monoclonal antibody in human clinical trials," said Peter Lamb, Ph.D., Executive Vice President, Scientific Strategy and Chief Scientific Officer of Exelixis. "Based on these data, we believe that applying our ADC capabilities to GamaMabs’ panel of antibodies against AMHR2 could yield a promising new addition to our biotherapeutics portfolio. Acquiring GamaMabs’ extensive know-how related to this target, as well as existing drug product and related manufacturing cell lines, will allow us to reduce significantly the development timeline compared with starting an AMHR2 program de novo. This is consistent with our strategy of advancing novel cancer therapies as rapidly as possible in order to enable new treatment options that may provide improved patient benefit."

While AMHR2 expression is normally restricted to ovary, testis and adrenal tissues, it is also expressed in ovarian, endometrial, renal, liver, colon and lung tumors. Murlentamab, a monoclonal antibody targeting AMHR2, was well tolerated when administered on its own in Phase 1 and 2 studies, with no dose-limiting toxicities observed. In early 2021, GamaMabs discontinued development of murlentamab given the modest single-agent efficacy observed in these trials. Although it does not have plans to move murlentamab forward, Exelixis is encouraged by the potential of the AMHR2 target as it pursues the further discovery and development of novel biologics.

"The preclinical and clinical data generated to date for murlentamab support its first-in-class potential in a variety of cancer indications, and we expect that Exelixis’ ADC capabilities and expertise in the development and commercialization of novel cancer therapies will help to realize the full potential of this antibody in addressing unmet patient need," said Stéphane Degove, Chief Executive Officer at GamaMabs. "We believe that placing our AMHR2 franchise with Exelixis will enable rapid and effective development of AMHR2-targeting cancer therapies while providing us with near-term revenue that can support development of our other pipeline programs and technologies."

On May 4, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2021 (Press release, PTC Therapeutics, MAY 4, 2021, View Source [SID1234579112]).

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"Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington’s disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success."

Key First Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC’s strongest quarterly commercial revenues to date.
Broader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
Emflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
In March 2021, the European Medicines Agency (EMA) approved Evrysdi (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
PTC received Gallup’s Don Clifton Strengths-Based Culture Award, which reflects the Company’s ongoing deep commitment to its employees.
First Quarter Clinical Updates:

PTC has multiple clinical trials ongoing, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19 with an expected data readout in the second half of 2021.
The second Bio-e compound, PTC857 healthy volunteer study was recently completed, and data will be communicated in the second quarter.
The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in mid-2021.
The Committee for Medicinal Products for Human Use (CHMP) has requested a clock stop in the aromatic L-amino acid decarboxylase (AADC) deficiency review process to allow for completion of its pre-approval inspections, which were delayed due to COVID-19. The CHMP opinion is now anticipated in the third quarter of 2021.
Due to COVID-related surgical delays, the AADC-deficiency biologics license application submission to the U.S. Food and Drug Administration is anticipated to be delayed by at least one quarter.
First Quarter 2021 Financial Highlights:

Total revenues were $117.9 million for the first quarter of 2021, compared to total revenues of $68.3 million for the first quarter of 2020, a 32% increase. Total revenue includes net product revenue of $91.3 million and collaboration and royalty revenue of $26.7 million in the first quarter of 2021.
Translarna net product revenues were $46.5 million for the first quarter of 2021, compared to $40.5 million for the first quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion into new territories.
Emflaza net product revenues were $43.5 million for the first quarter of 2021, compared to $27.5 million for the first quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported Evrysdi first quarter 2021 sales of approximately CHF 80 million. During the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC, which was reported as collaboration revenue.
U.S. GAAP (generally accepted accounting principles) R&D expenses were $134.5 million for the first quarter of 2021, compared to $90.1 million for the first quarter of 2020. The increase in R&D expenses reflects costs associated with increased investment in research programs, and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense, compared to $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $61.1 million for the first quarter of 2021, compared to $58.2 million for the first quarter of 2020. The increase in SG&A expenses was associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
Non-GAAP SG&A expenses were $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense, compared to $51.2 million for the first quarter of 2020, excluding $7.0 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $0.1 million for the first quarter of 2021, compared to $0.9 million for the first quarter of 2020. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
Net loss was $128.6 million for the first quarter of 2021, compared to net loss of $112.7 million for the first quarter of 2020.
Cash, cash equivalents and marketable securities was $988.4 million at March 31, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of March 31, 2021 were 70,405,905.
PTC Reaffirms Full Year 2021 Guidance as Follows:

PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 million.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the first quarter of 2021 corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 4292410. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On May 4, 2021 BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, reported that the company will present virtually at the 2021 RBC Capital Markets Global Healthcare Conference on Thursday, May 20th, 2021 at 12:05 pm ET (Press release, BioTheryX, MAY 4, 2021, View Source [SID1234579129]).

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Rob Williamson, President and CEO, will present an overview on the company’s targeted protein degradation platform, its clinical stage oncology pipeline, and the management team will participate in 1x1s with investors.

On May 4, 2021 eFFECTOR Therapeutics, Inc., a leader in the development of selective translation regulator inhibitors (STRIs) for the treatment of cancer, reported a $5.0 million cooperative agreement sponsored by the Defense Advanced Research Projects Agency (DARPA) and the Defense Health Agency (DHA) to fund a planned Phase 1b study of zotatifin (eFT226) as an anti-viral agent in an outpatient setting for those with mild to moderate COVID-19 disease (Press release, eFFECTOR Therapeutics, MAY 4, 2021, View Source [SID1234579502]). The agreement is a subaward of a DARPA research program awarded to the Quantitative Biosciences Institute (QBI) at University of California, San Francisco (UCSF), headed by Brian Shoichet, Ph.D., professor, department of pharmaceutical chemistry, to identify agents active against SARS-CoV-2 infections. eFFECTOR’s efforts will support the Phase 1b clinical study of zotatifin in patients with COVID-19 and related drug development activities.

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Zotatifin is an investigational small molecule inhibitor of eukaryotic initiation factor 4A (eIF4A), an enzyme that unwinds complex RNA structures important to producing key disease-driving proteins. SARS-CoV-2 is an RNA virus that hijacks the human cellular machinery—including eIF4A—to replicate. Research led by Nevan Krogan, Ph.D., director of QBI, and the QBI Coronavirus Research Group (QCRG) at UCSF, identified host factor vulnerabilities of SARS-CoV-2 replication published in Nature in April 2020. This research demonstrated that zotatifin was one of the most effective agents in blocking viral replication of SARS-CoV-2 out of 69 compounds evaluated.

"This research agreement allows us to evaluate zotatifin’s antiviral therapeutic potential in non-hospitalized patients suffering from mild to moderate SARS-CoV-2 infections," said Steve Worland, Ph.D., president and CEO of eFFECTOR. "Zotatifin may have broad utility against a number of coronaviruses, as in vitro studies have demonstrated that it is a potent inhibitor of SARS-CoV-2 and other coronavirus strains, including MERS-CoV."

Davide Ruggero, Ph.D., professor at the UCSF Helen Diller Cancer Center; an American Cancer Society Research Professor; and co-founder of eFFECTOR, added, "The interdisciplinary approach taken to establish zotatifin’s in vitro activity against SARS-CoV-2, which included proteomics to identify interactions between host and viral proteins and recognition of clinical-stage drug candidates that impacted the identified pathway, exemplifies the broad collaborative efforts taken by QCRG scientists around the world in response to COVID-19."

About Zotatifin (eFT226)
Zotatifin is a potent and sequence-selective inhibitor of eukaryotic translation initiation factor 4A (eIF4A) mediated translation. eIF4A is responsible for unwinding complex structures in the non-coding 5’ untranslated region of messenger RNA. Zotatifin is designed to inhibit the translation of mRNAs encoding several important oncogenes and survival factors, including receptor tyrosine kinases (RTKs), KRAS, Cyclin D, CDK4/6, and MYC. In vivo studies have shown potent in vivo tumor regression in multiple tumor models dependent on these factors, including non-small cell lung cancer and breast cancer. Since zotatifin inhibits the translation of mRNA in the non-coding region of mRNAs, it is not limited to any KRAS activating mutation subtypes. Zotatifin is currently being evaluated as an intravenous (IV) infusion in a Phase 1/2 clinical trial in patients with solid tumors.

Zotatifin will also be evaluated in a Phase 1b clinical trial in patients with mild to moderate COVID-19 infections pursuant to this grant sponsored by DARPA.

On May 4, 2021 twoXAR Inc., a Delaware corporation, focused on discovering and bringing first-in-class small molecules to market, reported that the company will rebrand as Aria Pharmaceuticals (Press release, Aria Pharmaceuticals, MAY 4, 2021, View Source [SID1234635533]). The name reflects the artistry and complexity that defines how the company and its scientific teams approach medicine from discovery to FDA approval in a way that is scientifically sound. Aria Pharmaceuticals also announced a pipeline update that includes novel preclinical compounds targeting fibrotic and immunological diseases.

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"We’ve built a significant and promising pipeline and assembled a team of top pharmaceutical R&D talent over the last few years, radically changing the trajectory of the company and how we approach pharmaceutical development," said Andrew Radin, CEO of Aria Pharmaceuticals. "Our name should reflect our commitment to sound science and our ability to not only discover but to develop and research novel treatments from preclinical to clinical studies."

Since its founding in 2015, the company has built its own proprietary pipeline as well as focused its partnership strategy on working with biotechnology companies on drug discovery research. With successful preclinical efficacy and safety data emerging from several of the company’s proprietary candidates, Aria Pharmaceuticals will transition from discovery collaborations with pharmaceutical companies to advancing its own pipeline into clinical research, starting with IND-enabling studies for novel candidates in chronic kidney disease, lupus and idiopathic pulmonary fibrosis.

To help validate and progress its pipeline through to clinical testing, in March 2020 Aria Pharmaceuticals hired pharmaceutical research leaders Mark Eller, Ph.D. as the Senior Vice President of Research and Development and Anjali Pandey, Ph.D. as the Senior Vice President of Nonclinical R&D and Chemistry. Both Dr. Eller and Dr. Pandey have led multiple successful pharmaceutical research and development programs from discovery through to approval and post marketing studies.

"We have made record progress in the last year in building and developing an exciting pipeline with very promising early data," said Mark Eller, Senior Vice President of Research and Development. "With our rapid progress in identifying potential treatments, we’re now exploring not only the addition of new therapeutic areas to our pipeline but building out our research programs for our existing pipeline."

To date, Aria Pharmaceuticals has identified 18 potential novel treatment candidates for complex diseases including, lupus, glioblastoma, chronic kidney disease and glaucoma. The company has announced positive preclinical safety and efficacy data in seven of its candidates with an average timing to complete predictions, select hits and begin in vivo testing of four weeks, significantly faster than traditional drug discovery processes which typically take years to reach similar milestones. In addition, Aria Pharmaceuticals’ approach to R&D has been proven to produce a 30-fold increase in hit rates at in vivo efficacy milestones over traditional methods. For more information, please visit www.ariapharmaceuticals.com