On May 5, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported business highlights and financial results for the first quarter ended March 31, 2022 (Press release, Agios Pharmaceuticals, MAY 5, 2022, View Source [SID1234613667]).

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"The U.S. approval of PYRUKYND in adults with PK deficiency was Agios’ first step toward changing treatment paradigms for people with genetically defined diseases, starting with developing the first disease-modifying therapy for a rare, debilitating, lifelong hemolytic anemia," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We are now poised to expand our impact for many more patients, including adults with PK deficiency in the EU, pediatric PK deficiency patients and people living with thalassemia, sickle cell disease and low- to intermediate-risk myelodysplastic syndrome. I am tremendously proud of our team’s incredible work on the U.S. launch of PYRUKYND, ongoing regulatory interactions in the EU, five pivotal clinical trials planned or underway, the expansion of our PK activation portfolio with AG-946 and our innovative research engine. We continue to execute in each of these arenas, motivated and inspired by our connections to patients."

First Quarter 2022 & Recent Highlights

Received approval from the U.S. Food and Drug Administration (FDA) for PYRUKYND, the first therapy for the treatment of hemolytic anemia in adults with PK deficiency and Agios’ first genetically defined disease medicine.
Executed commercial launch of PYRUKYND and generated approximately $0.8 million in net U.S. revenue for the first partial quarter following launch.
Published results from the ACTIVATE Phase 3 clinical study evaluating PYRUKYND in adults with PK deficiency who do not receive regular transfusions in the New England Journal of Medicine.
Completed the single ascending dose and multiple ascending dose healthy volunteer cohorts of the Phase 1 study of novel PK activator AG-946 and identified doses for the Phase 1 sickle cell disease cohort and Phase 2a study in low- to intermediate risk myelodysplastic syndrome (MDS).
Initiated the sickle cell disease cohort of the Phase 1 study of AG-946.
Published 2022 Environmental, Social and Governance (ESG) Report disclosing ESG initiatives and metrics aligned with the United Nations Sustainable Development Goals (UN SDGs) and the standards for the Biotechnology and Pharmaceuticals industry set by the Sustainability Accounting Standards Board (SASB).
Key Upcoming Milestones & Priorities

Agios expects to execute on the following key milestones and priorities in 2022:

Adult PK Deficiency: Receive European Medicines Agency (EMA) regulatory decision for PYRUKYND in adults with PK deficiency by year-end.
Pediatric PK Deficiency: Initiate Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in not regularly transfused and regularly transfused pediatric patients with PK deficiency, respectively, in mid-2022.
Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease by year-end.
Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
Data Presentations

Submitted new clinical and translational data to the European Hematology Association (EHA) (Free EHA Whitepaper) congress, to be held June 9-12 in Vienna and virtually, including:
New patient-reported outcomes (PRO) data from ACTIVATE Phase 3 study of PYRUKYND
Data demonstrating the normalization of hemoglobin levels with long-term treatment of PYRUKYND in adults with PK deficiency
Additional PK deficiency comorbidities and complications data from the PEAK registry
Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
First Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND for the first quarter of 2022 was $0.8 million. This revenue reflects the first partial quarter of PYRUKYND launch, following FDA approval on February 17, 2022.

Cost of Sales: Cost of sales for the first quarter of 2022 were $0.3 million.

Non-Operating Income: Non-operating income included approximately $2.7 million from TIBSOVO (ivosidenib) royalties for the first quarter of 2022.

Research and Development (R&D) Expenses: R&D expenses were $70.1 million for the first quarter of 2022 compared to $57.7 million for the first quarter of 2021. The year-over-year increase in R&D was driven primarily by start-up costs for the PYRUKYND pivotal studies in thalassemia and sickle cell disease and planned increases in research activities, offset by closeouts of the ACTIVATE and ACTIVATE-T studies.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $31.5 million for the first quarter of 2022 compared to $33.6 million for the first quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to lower workforce expenses.

Net Loss from Continuing Operations: Net loss from continuing operations was $94.8 million for the first quarter of 2022 compared to a net loss of $90.9 million for the first quarter of 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2022, were $1.2 billion compared to $2.4 billion as of March 31, 2021. The year-over-year decrease is attributable to operating expenses and 16.2 million shares of common stock that the company repurchased for $802.5 million during the second through fourth quarters of 2021. Agios expects that its cash, cash equivalents and marketable securities will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss first quarter 2022 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 5738266. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

On May 5, 2022 Ashvattha Therapeutics, ("Ashvattha"), a clinical stage company developing novel hydroxyl dendrimer therapeutics, reported that Jeffrey Cleland, Ph.D., Chairman, CEO & President of Ashvattha, will provide a corporate overview followed by a moderated Q&A at the UBS Global Healthcare Conference 2022 taking place in New York, NY, May 23-25, 2022 (Press release, Ashvattha Therapeutics, MAY 5, 2022, View Source [SID1234613683]).

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Additional details can be found below:

A webcast from the presentation may be accessed on the "News" page of the Ashvattha website following the event.

On May 5, 2022 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, reported that it has entered into a securities purchase agreement with certain institutional investors providing for the purchase and sale of registered securities of the Company in a registered direct offering for gross proceeds to Aeglea of approximately $45 million, prior to deducting placement agent fees and estimated offering expenses (Press release, Aeglea BioTherapeutics, MAY 5, 2022, View Source [SID1234613699]). The financing includes participation from Bain Capital Life Sciences, LP, Great Point Partners, LLC, clients of Nantahala Capital Management, Sio Capital Management, LLC and other institutional investors.

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The financing includes 10,752,688 shares of the Company’s common stock at a price of $1.60 per share and pre-funded warrants to purchase 17,372,397 shares of common stock, at a price of $1.5999 per pre-funded warrant, which represents the per share offering price for the common stock less the $0.0001 exercise price for each pre-funded warrant. The offering is expected to close on or about May 9, 2022, subject to customary closing conditions.

JonesTrading Institutional Services LLC is acting as placement agent for the registered direct offering.

The shares of common stock and pre-funded warrants were offered pursuant to a shelf registration statement on Form S-3 (File No. 333-239706), which was declared effective by the United States Securities and Exchange Commission ("SEC") on July 14, 2020. The Company intends to use the proceeds from the offering, together with its existing cash resources, to fund the Company’s activities related to our ongoing Biologics License Application submission for pegzilarginase and its potential commercialization in the United States for patients with Arginase 1 Deficiency, advance the clinical development of AGLE-177 through its Phase 1/2 clinical trial and prepare for a potential Phase 3 trial for the treatment of patients with Homocystinuria, and to advance AGLE-325 for Cystinuria through IND-enabling activities, and the remainder to fund continued research and development, manufacturing, working capital and general corporate purposes. The Company expects the net proceeds from this offering, together with its existing cash, cash equivalents, and marketable securities, to fund operations through the second quarter of 2023.

A prospectus supplement relating to the shares of common stock will be filed by the Company with the SEC. When available, copies of the prospectus supplement, together with the accompanying prospectus, can be obtained at the SEC’s website at www.sec.gov or from JonesTrading Institutional Services LLC at [email protected].

On May 5, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the first quarter ended March 31, 2022 (Press release, Repare Therapeutics, MAY 5, 2022, View Source [SID1234613715]).

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"The first quarter was marked by significant progress in our RP-3500 program, including the comprehensive new dataset from the TRESR trial which was part of the featured oral presentation at the AACR (Free AACR Whitepaper) conference this year, and that the United States Adopted Names (USAN) council has adopted ‘camonsertib’ as the nonproprietary (generic) name in the United States, and the International Nonproprietary Names (INN) council has accepted ‘camonsertib’ as proposed INN for RP-3500," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "The findings continue to demonstrate camonsertib’s promising clinical benefit given as monotherapy in patients with solid tumors with multiple genotypes and potential best-in-class safety and tolerability profile for ATR inhibitors. Beyond ATM, encouraging data in other genotypes suggest further validation of our STEP2 platform to identify other synthetic lethalities as we expand TRESR and develop our pipeline of synthetic lethal candidates. We look forward to providing updates on the potential of camonsertib as monotherapy and initial data of camonsertib in combination with PARP inhibitors or gemcitabine expected in the second half of this year."

First Quarter 2022 Review and Operational Updates:

Announced updated clinical data from the ongoing Phase 1/2 TRESR (Treatment Enabled by SNIPRx) clinical trial of camonsertib, a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase) for the treatment of solid tumors with specific synthetic-lethal genomic alterations including those in the ATM gene (Ataxia-Telangiectasia mutated kinase) at the 2022 AACR (Free AACR Whitepaper) Annual Meeting.
Updated data showed camonsertib monotherapy continues to appear safe and well tolerated. Expectedly, Grade 1-2 anemia was the most common treatment-related adverse event and was well controlled in patients. Only 24.2% of all patients in the 3 days on 4 days off schedule experienced Grade 3 anemia, and none experienced Grade 4 anemia.

Camonsertib monotherapy resulted in durable clinical benefit across tumor types and genomic alterations. Overall clinical benefit rate (CBR) for all patients was 43%, and 47% in patients after PARP inhibitor (PARPi) failure.

Promising results were observed particularly in patients with advanced ovarian cancer (n = 20). 90% of evaluated patients had prior PARPi failure, and 85% of evaluated patients were platinum resistant. In these patients, overall response (OR) was 25%, including one complete response (CR), three partial responses (PR) as determined by RECIST 1.1 criteria, and one durable and ongoing CA125 response. CBR was 75% and median progression-free survival (mPFS) was 35 weeks.

Clinical benefit was also observed in patients with tumors harboring BRCA1 and BRCA2 genomic alterations, as predicted by SNIPRx. In patients with BRCA1/2 mutated tumors (n = 37), ORR was 14% and included two patients with ovarian cancer, and one each with breast cancer, head and neck squamous cell carcinoma, and melanoma. CBR was 43% with a mPFS of 15 weeks in the BRCA1/2 population; in patients specifically with BRCA1 mutations, the CBR was 48%.

In patients with ATM loss-of-function (LOF) tumors (n = 34), ORR was 9% including one RECIST 1.1 confirmed/unconfirmed response, and two prostate specific antigen (cPSA) responses. CBR in patients with ATM LOF was 44%, with mPFS of 17 weeks.

New sequencing data demonstrated biallelic gene LOF, an emerging biomarker for synthetic lethal therapies, can potentially be leveraged to further enrich for patients most likely to benefit from camonsertib. CBR in patients with biallelic LOF was significantly higher (47%) compared to the CBR in patients with non-biallelic tumors (15%).

A poster presentation, titled "Circulating tumor DNA (ctDNA) determinants of improved outcomes in patients (pts) with advanced solid tumors receiving the ataxia telangiectasia and Rad3-related inhibitor (ATRi), RP-3500, in the phase 1/2a TRESR trial (NCT04497116)" (abstract #367586) will be presented at the 2022 ASCO (Free ASCO Whitepaper) Annual meeting in June.

Announced camonsertib dose selection Phase 1 monotherapy safety data from the Phase 1/2 TRESR Trial at the 2022 ESMO (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress
This comprehensive safety analysis from three monotherapy dosing schedules of camonsertib at therapeutic doses confirmed the acceptable tolerability of the recommended phase 2 dose (160mg 3 days on/4 days off).

Anemia was the most common reported toxicity with less than 25% of patients experiencing grade 3 toxicities.

A dose modification plan that includes 2 alternative dosing schedules was established to mitigate the on-target toxicity of anemia and maintain patients on a camonsertib dosing schedule that targets antitumor activity.

A nomogram, based on cycle 1 assessment of toxicities, is being prospectively evaluated to identify patients at increased risk of anemia and inform early intervention.

Announced publication of preclinical data in Nature demonstrating the potential of PKMYT1 inhibitor RP-6306 in tumors with CCNE1 amplification
In April 2022, the Company announced that preclinical data demonstrating inhibition of CCNE1-amplified tumor growth in vivo by selective inhibition of PKMYT1 using RP-6306, a first-in-class small molecule candidate targeting PKMYT1, were published in Nature.

SNIPRx, the Company’s proprietary, genome-wide, CRISPR-based screening approach, was used to uncover CCNE1 amplification as synthetic lethal to PKMYT1 inhibition. Data show PKMYT1 inhibition is a promising therapeutic target in CCNE1-amplified cancers.

Phase 1 clinical trials are currently evaluating RP-6306 as a monotherapy (MYTHIC) as well as in combination with gemcitabine (MAGNETIC) for the treatment of molecularly selected advanced solid tumors. The Company recently initiated an additional Phase 1 (MINOTAUR) clinical trial of RP-6306 in combination with FOLFIRI also for the treatment of molecularly selected advanced solid tumors.

Initiated patient enrollment in a new arm of its Phase 1 MYTHIC clinical trial designed to evaluate the safety and tolerability of RP-6306 in combination with camonsertib in patients with advanced solid tumors.
Appointed Philip Herman to Executive Leadership Team as EVP, Commercial & New Product Development
In January 2022, the Company appointed Philip Herman as EVP, Commercial & New Product Development. Mr. Herman was most recently Chief Commercial Officer of Y-mAbs Therapeutics and led the successful launch of DANYELZA (naxitamab).
First Quarter 2022 Financial Results:

Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of March 31, 2022 were $311.7 million.
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $26.5 million and $16.5 million for the three months ended March 31, 2022 and 2021, respectively. The year-over-year increase in Net R&D expenses was primarily due to direct external costs related to the Company’s camonsertib and RP-6306 programs, and personnel related expenses, including share-based compensation.
General and administrative (G&A) expenses: G&A expenses were $8.8 million and $5.2 million for three months ended March 31, 2022 and 2021, respectively. The year-over-year increase in G&A expenses was primarily due to personnel related costs, including share-based compensation, as we scale the organization, and professional fees related to the Company’s transition from emerging growth company and smaller reporting company status at the end of 2021.
Net loss: Net loss was $34.8 million, or $0.83 per share in the three months ended March 31, 2022, and $21.4 million, or $0.58 per share in the three months ended March 31, 2021.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

On May 5, 2022 Daewoong Pharmaceutical (Daewoong) (CEO Sengho Jeon & Changjae Lee) reported its financial results for the first quarter of 2022 (separate basis) (Press release, Daewoong Pharmaceutical, MAY 5, 2022, View Source [SID1234613732]). In the first quarter, the separate sales and operating profit marked KRW 272.2 billion and KRW 26.8 billion, up 12.6% and 32.6% year-over-year, respectively . In the same quarter, the consolidated sales and operating profit marked KRW 298.4 billion and KRW 23 billion, up 10.7% and 2.2% y-o-y, respectively.

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Daewoong achieved record-high annual sales and operating profit in 2021 and again broke its record in the highest quarterly operating profit (separate basis) in the first quarter of this year. The sales growth of highly profitable ethical-the-counter (ETC) drugs and the increase in the export of Nabota were led by improvements in profitability, operating profit, and gross profit margin (GPM).

Sales of ETC drugs recorded KRW 197.6 billion, a 9% increase from KRW 181 billion y-o-y, leading to an increase in earnings. It is characterized by more than 20% growth compared to the same period of the previous year in highly profitable product lines such as an antiulcer drug Nexiad, a dyslipidemia drug Litorvazet, a gastric ulcer drug Axid, a hyperlipidemia drug Crezet, and an antithrombotic drug Cloart.

Sales of Nabota surged by 98% y-o-y, from KRW 15.4 billion to KRW 30.7 billion exports. In particular, its exports increased by 189% y-o-y, from KRW 7.9 billion to KRW 22.8 billion. For example, the exports to its U.S. sales partner Evolus amounted to KRW 18.3 billion, risen by three times y-o-y. Favorable exchange rates also supported the earnings. Nabota is expected to drive sales growth continuously, as Evolus is preparing to officially launch Nabota in Europe in the third quarter, and Nabota shows strong market presence in the countries where it has already been released.

Sales of over-the-counter (OTC) drugs recorded KRW 29.7 billion, a 12% increase from KRW 26.4 billion y-o-y. Due to the spread of COVID-19 and a surge in home treatment, the company’s representative cold medicine EZN6 grew by 77.3% y-o-y, and the quasi-drugs related to physical fatigue Urshot and a wet patch Easyderm also contributed to the growth. Sales of supplements also surged by 169% y-o-y, owing to its strategic reinforcement of the supplement portfolio for the liver, centered on the liver-specialized brand Ener Thistle, blood circulation, intestine, and vitamins, and focus on online sales channels.

"With export expansion of Nabota based on superior product power and quality, the highly profitable ETC product lines continued to grow, leading to another record-breaking highest quarterly operating profit," said Sengho Jeon, Daewoong Pharmaceutical CEO. "For the second quarter and the second half of this year, we are expected to achieve solid growth and increasing profitability, thanks to the expansion of Nabota’s overseas sales channels, the launch of highly profitable new products, such as Fexuclue tablets, a new drug for gastroesophageal reflux disease, and an increase in the gross profit margin."