PERRIGO REPORTS FOURTH QUARTER & FISCAL YEAR 2021 FINANCIAL RESULTS FROM CONTINUING OPERATIONS

On March 1, 2022 Perrigo Company plc (NYSE: PRGO) ("Perrigo" or the "Company"), a leading provider of Consumer Self-Care Products, reported financial results for the fourth quarter and fiscal year ended December 31, 2021 (Press release, Perrigo Company, MAR 1, 2022, View Source [SID1234609285]). All comparisons are against the prior year fiscal fourth quarter and fiscal year, unless otherwise noted.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

President and CEO, Murray S. Kessler commented, "In the face of another year of unprecedented pandemic-related challenges, the Perrigo team achieved the Company’s three primary strategic objectives – divesting the generic RX business, reallocating the sale proceeds to acquire a star consumer self-care asset in HRA Pharma, and significantly reducing uncertainty by favorably settling the Irish Tax assessment. Perrigo’s transformation into a focused, consumer-centric Self-Care company is now complete and our focus going forward is on long-term, profitable growth."

Kessler continued, "While our big three strategic objectives were achieved, COVID-19 related business interruption in the form of historically low cough/cold sales early in the year and supply chain disruptions and material price inflation later in the year significantly impacted our 2021 business results. However, sales growth improved sequentially each quarter during 2021 as consumer demand rebounded strongly as the year progressed behind a normalized level of cough, cold and flu illnesses. Actions were also taken to address third quarter supply chain disruptions in the U.S. allowing us to meet strong fourth quarter demand for our customers, albeit at a higher cost. We are entering 2022 with significant topline momentum."

Kessler concluded, "We are also entering 2022 with a higher level of cost inflation and COVID-19 related productivity challenges, which negatively impacted our second half 2021 gross margin. We expect to overcome these headwinds in the second half of 2022, driven in part by increased pricing, higher volumes and productivity gains. Our guidance reflects strong topline growth and depressed margins in the first half of 2022, anticipating that as gross margin pressure eases and as the highly accretive acquisition of HRA closes by mid-year, Perrigo is poised for turbocharged sales and earnings growth. We are excited about our future and remain confident in our consumer Self-Care strategy. I am especially grateful to all of Perrigo’s employees worldwide who have kept the Company running throughout the pandemic, and also want to acknowledge our Ukrainian colleagues and their families. Our hearts and support are with you."

Refer to Tables I – IV at the end of this press release for a reconciliation of non-GAAP adjustments to the current year and prior year periods and additional non-GAAP information. The Company’s reported results are included in the attached Consolidated Statements of Operations, Balance Sheets and Statements of Cash Flows.

Fourth Quarter 2021 Perrigo Results from Continuing Operations

Perrigo net sales for the fourth quarter were $1.1 billion, an increase of $52 million, or 4.9%. Acquisitions accounted for 0.2 percentage points of growth, while unfavorable currency movements offset growth by 0.8 percentage points. Organic net sales growth was 5.5%. Of note, the prior year fourth quarter included six more shipping days than the current quarter.

Net sales in the quarter were driven by 1) the addition of $41 million in contract manufacturing sales to the divested RX business, 2) higher incidences of cough/cold and flu-like illnesses leading to an increase of $38 million in cough/cold sales, 3) higher net sales in the CSCA Nutrition and CSCI Skincare and Personal Hygiene categories, and 4) increased pricing across both Consumer Self-Care segments. These drivers also benefited from new product sales and e-commerce growth. These increases were partially offset by 1) lower net sales in the CSCA Healthy Lifestyle, CSCI VMS and Global Oral Care categories, 2) discontinued products of $11 million, 3) net unfavorable currency movements of $9 million, and 4) lower net sales in the CSCA Mexico business of $7 million.

Fourth quarter reported operating income was $47 million in 2021 compared to $38 million in 2020. Adjusted operating income increased $15 million, or 12.5%, to $132 million in 2021. This increase was driven by 1) higher profit new products, and 2) lower operating expenses, including planned lower advertising & promotion investments, and Project Momentum cost savings. These results were partially offset by $28 million in lower operating efficiencies, including lower volumes resulting in unfavorable plant overhead absorption, higher materials costs and increased freight expenses.

Reported net income was $32.1 million, or $0.24 per diluted share, compared to a net loss of $52.2 million, or $0.39 per diluted share in the prior year period. Excluding certain charges as outlined in Table I, fourth quarter 2021 adjusted net income was $82 million, or $0.60 per diluted share, compared to $64 million, or $0.47 per diluted share, last year due to the factors described above.

Fourth Quarter 2021 Business Segment Results from Continuing Operations

Consumer Self-Care Americas Segment

CSCA fourth quarter net sales of $736 million increased $35 million, or 5.0%. Primary category drivers are provided below.

Upper Respiratory
Net sales of $137 million increased 26.4% due primarily to higher incidences of cough/cold and flu-like illnesses that led to strong demand for cough/cold products, particularly store brand liquid-based cough/cold and pseudoephedrine-based allergy products.

Digestive Health
Net sales of $131 million were flat due primarily to growth in e-commerce, offset by temporary packaging constraints on a specific product, which is expected to alleviate during the second half of 2022, and timing of shipments in antacids.

Pain & Sleep-Aids
Net sales of $113 million increased 3.4% due primarily to strong demand for children’s analgesics products due to higher incidences of cough/cold and flu-like illnesses, partially offset by share declines in the national brand of naproxen sodium, which impacted demand for the store brand version, and lower sales in Mexico.

Nutrition
Net sales of $109 million increased 12.2% due primarily to new product launches in infant formula, including hypoallergenic formula and contract manufacturing formulas. Net sales were also driven by growth in U.S. store brand infant formula and continued growth in the oral electrolytes business, partially offset by lower infant formula sales to Canadian customers.

Oral Care
Net sales of $85 million decreased 1.4% due primarily to delayed receipt of products manufactured outside the U.S., leading to unfulfilled customer orders.

Healthy Lifestyle
Net sales of $83 million decreased 13.9% due primarily to the discontinuation of diabetes products and lost distribution of certain smoking cessation products that annualized in the quarter.

Skincare & Personal Hygiene
Net sales of $53 million decreased 3.4% due primarily to lower sales of creams for topical fungal infections and minoxidil, partially offset by growth in e-commerce.

Vitamins, Minerals, and Supplements ("VMS") and Other
Net sales of $26 million increased 43.6% due primarily to contract manufacturing sales to the divested RX business.

Reported operating income was $93 million in 2021 compared to $117 million in 2020. Adjusted operating income decreased $22 million to $110 million driven by lower operating efficiencies, including lower volumes resulting in unfavorable plant overhead absorption, higher materials costs and increased freight expenses. These factors were partially offset by lower operating expenses, including Project Momentum cost savings.

Consumer Self-Care International Segment

CSCI net sales of $369 million increased $16 million, or 4.6% including a positive impact of 0.6 percentage points from acquisitions partially offset by a negative impact of 2.4 percentage points from unfavorable currency movements. Organic net sales growth was a strong 6.4%. Primary category drivers are provided below.

Skincare & Personal Hygiene
Net sales of $86 million increased 13.1%, or 15.7% excluding the impact of currency, driven primarily by increased market share in the ACO skincare franchise, new product launches in the Sebamed skincare portfolio, and the October 30, 2020 acquisition of three Eastern European OTC Dermatology Brands. This growth was partially offset by lower sales in Australia.

Upper Respiratory
Net sales of $82 million increased 29.7%, or 32.6% excluding the impact of currency, as the higher incidences of cough/cold and flu-like illnesses led to strong demand for cough/cold products, particularly Bronchostop,Coldrex and U.K. store brands. New products also drove growth in the quarter.

VMS
Net sales of $55 million decreased 10.6%, or 8.5% excluding the impact of currency, due primarily to strong performance in the prior year period of vitamin D-based products as well as residual impact from the third quarter 2021 recall of certain batches of Davitamon and Abtei.

Pain & Sleep-Aids
Net sales of $53 million decreased 1.8%, or an increase of 0.4% excluding the impact of currency, due primarily to an increase in U.K. store brand, which was offset by lower demand in Italy for Optalidon, a paracetamol-based analgesics product, and lower sales in Australia.

Healthy Lifestyle
Net sales of $39 million decreased 4.7%, or 2.7% excluding the impact of currency, as benefits from competitor supply issues and growing demand for NiQuitin smoking cessation products were more than offset by lower net sales in the XLS Medical weight management franchise due primarily to lower category consumption.

Oral Care
Net sales of $23 million decreased 19.3%, or 17.6% excluding the impact of currency, due primarily to delayed receipt of product manufactured outside the E.U., leading to unfulfilled customer orders.

Digestive Health and Other
Net sales of $30 million increased 9.4%, or 12.3% excluding the impact of currency, due primarily to new distribution of Kijimea, a medical food for the dietary management of irritable bowel syndrome, and higher sales of other distribution brands.

Reported operating income was $13 million in 2021 compared to an operating loss of $13 million in 2020. Adjusted operating income increased $26 million, or 77.7%, to $60 million due primarily to 1) higher gross profit flow-through resulting from increased net sales, and 2) lower operating expenses, including lower planned advertising & promotion investments, and Project Momentum cost savings.

Fiscal Year 2021 Perrigo Results from Continuing Operations

Perrigo net sales for the fiscal year were $4.1 billion, an increase of $51 million, or 1.2%, including positive impacts of 1.5 and 1.1 percentage points from favorable currency movements and acquisitions, respectively. These gains were partially offset by 0.7 percentage points from divestitures. Organic net sales declined 0.7%, including a negative impact of 1.7 percentage points related to lower net sales of cough/cold products worldwide.

Net sales growth was driven by 1) $61 million from contract manufacturing sales to the divested RX business, 2) $46 million from acquisitions, 3) growth in the CSCI Skincare and Personal Hygiene and VMS categories, and in CSCA Nutrition and Skincare & Personal Hygiene categories, 4) $61 million in net favorable currency movements, and 5) increased pricing across both Consumer Self-Care segments. These drivers also benefited from new product sales and strong e-commerce growth. These increases were partially offset by 1) a decline in net sales of $68 million from cough/cold products, 2) discontinued products of $38 million, 3) $29 million from divestitures, and 4) lower net sales in the CSCA Healthy Lifestyle category.

Fiscal year 2021 reported operating income increased $145 million to $410 million, compared to $265 million in 2020. Adjusted operating income decreased $61 million to $479 million in 2021. This decrease was driven by 1) $80 million in lower operating efficiencies, including lower volumes resulting in unfavorable plant overhead absorption, higher materials costs and increased freight expenses, and 2) lower gross profit flow-through, principally from the decline in cough/cold sales. These results were partially offset by 1) the net impact from acquisitions and divestitures, and 2) lower operating expenses, including lower advertising & promotion investments compared to the prior year, and Project Momentum cost savings.

Reported net loss was $131 million, or a loss of $0.98 per diluted share, versus reported net income of $44 million, or $0.32 per diluted share, in the prior year. Excluding certain charges as outlined in Table I, fiscal 2021 adjusted net income was $278 million, or $2.06 per diluted share, versus $320 million, or $2.33 per diluted share, last year due to the factors described above.

Fiscal Year 2021 Business Segment Results from Continuing Operations

Consumer Self-Care Americas Segment

Consumer Self-Care Americas reported net sales of $2.7 billion were flat compared to the prior year, including a positive impact of 0.9 percentage points from acquisitions. Organic net sales declined 1.1%, including a negative impact of 1.4 percentage points due to lower net sales of cough/cold products compared to the prior year. Global supply chain disruptions, including a lack of truck drivers in the U.S. and record backups at global shipping ports, dampened net sales by an additional 1.4 percentage points. Primary category drivers are provided below.

Upper Respiratory
Net sales of $483 million decreased 4.5% due primarily to the historically weak 2020/21 cough/cold season and the recall of an allergy product in the third quarter of 2021. Increased pricing and new products partially offset these declines.

Digestive Health
Net sales of $475 million increased 0.8% due primarily to sales of ‘national brand better’ products, new products and e-commerce. These drivers were mostly offset by competition for a proton pump inhibitor and the re-launch of a national brand acid reducer, which gained market share from competing store brand products.

Pain & Sleep-Aids
Net sales of $405 million decreased 6.7% due primarily to the historically weak 2020/21 cough/cold season, partially offset by higher sales of store brand diclofenac 1%.

Nutrition
Net sales of $402 million increased 3.5% driven by new products, including in the infant formula contract manufacturing business, and continued growth in oral electrolytes. These drivers were partially offset by lower sales of U.S. store brand infant formula due primarily to supply constraints earlier in the year.

Oral Care
Net sales of $312 million increased 8.2% due primarily to one quarter of inorganic growth stemming from the April 2020 acquisition of Dr. Fresh and strong growth in the base business during the first half of 2021. These drivers were partially offset by delayed receipt of product manufactured outside the U.S. in the second half, leading to unfulfilled customer orders.

Healthy Lifestyle
Net sales of $298 million decreased 15.5% due primarily to the discontinuation of diabetes products and lost distribution of certain smoking cessation products that annualized in the fourth quarter.

Skincare & Personal Hygiene
Net sales of $219 million increased 9.3% due primarily to higher sales in the minoxidil franchise and the Scaraway brand, partially offset by lower sales of creams for topical fungal infections.

VMS and Other
Net sales of $99 million increased 90.4% due primarily to contract manufacturing sales to the divested RX business.

Reported operating income was $207 million in 2021 compared to $465 million in 2020. Adjusted operating income decreased $94 million to $434 million driven by lower operating efficiencies, including lower volumes resulting in unfavorable plant overhead absorption, higher materials costs and increased freight expenses. These factors were partially offset by lower advertising and promotion expenses and Project Momentum cost savings.

Consumer Self-Care International Segment

CSCI net sales of $1.4 billion increased $50 million, or 3.6%, including positive impacts of 4.0 percentage points and 1.6 percentage points from favorable currency movements and acquisitions, respectively. These gains were partially offset by 2.1 percentage points from divestitures. Organic net sales growth was flat, including a negative impact of 2.2 percentage points due to lower net sales of cough/cold products compared to the prior year. Primary category drivers are provided below.

Skincare & Personal Hygiene
Net sales increased 12.1%, or 7.2% excluding the impact of currency, to $394 million driven primarily by the October 30, 2020 acquisition of three Eastern European OTC Dermatology Brands, increased market share in the ACO skincare franchise and new product launches in the Sebamed skincare portfolio. These drivers were partially offset by a decline in the anti-parasite portfolio and lower sales in Australia.

Upper Respiratory
Net sales of $226 million decreased 11.3%, or 13.7% excluding the impact of currency, due primarily to the historically weak 2020/21 cough/cold season, partially offset by new products.

VMS
Net sales of $217 million increased 8.2%, or 4.0% excluding the impact of currency, due primarily to a strong performance of Granufink, herbal medicines to keep bladder function healthy, and the launch of the Probify line of probiotics.

Pain & Sleep-Aids
Net sales of $202 million increased 6.0%, or 2.1% excluding the impact of currency, as higher sales of U.K. store brand and Tiger Balm were partially offset by declines in other pain products due primarily to the historically weak 2020/21 cough/cold season.

Healthy Lifestyle
Net sales of $179 million increased 8.4%, or 3.7% excluding the impact of currency, as growing demand for NiQuitin smoking cessation products and higher net sales in Australia were partially offset by lower net sales in the XLS Medical weight management franchise due primarily to lower category consumption.

Oral Care
Net sales of $96 million decreased 2.0%, or 5.9% excluding the impact of currency, due primarily to delayed receipt of product manufactured outside the E.U. in the second half of the year, leading to unfulfilled customer orders.

Digestive Health and Other
Net sales of $131 million decreased 2.2%, or 6.1% excluding the impact of currency, due primarily to lower distribution sales in the E.U. partially offset by higher sales in Australia.

Reported operating income was $36 million in 2021 compared to $32 million in 2020. Adjusted operating income increased $13 million, or 6.7%, to $212 million as lower operating expenses, including lower planned advertising & promotion investments compared to the prior year and Project Momentum cost savings, more than offset unfavorable product mix.

Fiscal 2022 Outlook

Based on current currency exchange and spot rates, and excluding expected benefits from the acquisition of HRA Pharma, the Company expects:

Net sales growth of 3.5% to 4.5%, inclusive of negative impacts of approximately 2.0 percentage points from the planned divestiture of the Latin American businesses and 1.5 percentage points from adverse currency movements,
Organic net sales growth of 7.0% to 8.0%,
First half margin compression and second half margin expansion,
An adjusted effective tax rate of approximately 23%,
Adjusted diluted EPS of $2.10 to $2.30,
Cash flow from operations as a percentage of adjusted net income of 95% to 105%.
Assuming a June 30, 2022 closing and based on current currency exchange rates, the Company expects the acquisition of HRA Pharma to add:

Net sales of $170 – $190 million and adjusted diluted EPS of approximately $0.30 in 2022.
The Company cannot reconcile its expected adjusted diluted earnings per share to diluted earnings per share under "Fiscal 2022 Outlook" without unreasonable effort because certain items that impact net income and other reconciling metrics are out of the Company’s control and/or cannot be reasonably predicted at this time.

Pieris Pharmaceuticals Reports Full-Year 2021 Financial Results and Provides Corporate Update

On March 1, 2022 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported financial results for the fiscal year ended December 31, 2021 and provided an update on the Company’s recent and anticipated future developments (Press release, Pieris Pharmaceuticals, MAR 1, 2022, View Source [SID1234609284]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Last year was one of steady execution as we laid the groundwork for 2022, which we believe promises to be the most important catalyst year in the Company’s history, including key efficacy data for cinrebafusp alfa and PRS-060/AZD1402. We announced a collaboration with R&D leader Genentech, reported data from several clinical and preclinical programs, and initiated multiple clinical trials. Later this year, we expect to report topline data from the phase 2a trial of PRS-060/AZD1402, which has recently cleared the safety gate for the 1 mg and 3 mg dose cohorts. Beyond PRS-060/AZD1402, we currently have two bispecific immuno-oncology programs in clinical development and plan on initiating clinical development for our proprietary inhaled-delivery idiopathic pulmonary fibrosis (IPF) drug program, PRS-220, later this year, with generous grant support from the Bavarian government," said Stephen S. Yoder, President and Chief Executive Officer of Pieris. "Through these initiatives, we have advanced our pipeline assets while strengthening our balance sheet through partnerships, grant funding, and focused utilization of our ATM facility, all with the focus of driving toward our key inflection points."

PRS-060/AZD1402 and AstraZeneca Collaboration: Enrollment of part 2a (efficacy of 1 mg and 3 mg cohorts) and part 1b (safety of 10 mg cohort) has begun following successful completion of the sponsor safety review of part 1a (safety of 1 mg and 3 mg cohorts) of the multi-center, placebo-controlled phase 2a study of dry powder inhaler-formulated PRS-060/AZD1402. PRS-060/AZD1402 is an IL-4 receptor alpha inhibitor under development in collaboration with AstraZeneca for the treatment of moderate-to-severe asthma. Pieris and AstraZeneca expect to announce topline data from the phase 2a study this year, although the companies are actively evaluating the feasibility of study timelines in the current geopolitical environment and will update guidance in the orderly course of business, if needed.Upon completion of the study, which is being sponsored and funded by AstraZeneca, Pieris may choose to exercise its co-development option, which would be on a 25% cost-share basis with a cost cap or a 50% cost-share basis without a cost cap. Separately, Pieris will have a future option to co-commercialize PRS-060/AZD1402 in the United States.
Cinrebafusp Alfa (PRS-343 ) : In January 2022, the first patient was dosed in the phase 2 study of cinrebafusp alfa, a 4-1BB/HER2 Anticalin-based bispecific for the treatment of HER2-expressing gastric cancer. The two-arm, multicenter, open-label phase 2 study is evaluating the efficacy, safety, and tolerability of cinrebafusp alfa in combination with standard of care agents ramucirumab and paclitaxel in patients with HER2-high gastric cancer and in combination with tucatinib in patients with HER2-low gastric cancer. Pieris plans to report data from the HER2-low arm this year. Separately, the Company plans to disclose data from the HER2-high arm in 2023.
PRS-344/S095012 and Servier Collaboration: The first patient was dosed in November 2021 in the phase 1/2 study of PRS-344/S095012, a 4-1BB/PD-L1 Anticalin-based bispecific for the treatment of solid tumors, triggering an undisclosed milestone payment to Pieris. Pieris holds exclusive commercialization rights for PRS-344/S095012 in the United States and will receive royalties on any ex-U.S. sales for this program. Additionally, Servier is continuing development of PRS-352, an undisclosed Anticalin-based bispecific beyond 4-1BB.
PRS-220: Pieris remains on track to begin a phase 1 trial this year for PRS-220, a proprietary inhaled Anticalin protein targeting connective tissue growth factor for the treatment of IPF.
Year End Financial Update:

Cash Position – Cash and cash equivalents totaled $117.8 million for the year ended December 31, 2021, compared to a cash and cash equivalents balance of $70.4 million for the year ended December 31, 2020. The increase since December 2020 is due to cash received from new and existing collaboration agreements, including milestone achievements. In addition, during 2021, the ATM program was utilized to raise a total of $38.5M in net proceeds at an average price of $4.85 per share. These increases were partially offset by cash used to fund operations in 2021.

R&D Expense – R&D expenses were $66.7 million for the year ended December 31, 2021, compared to $46.5 million for the year ended December 31, 2020. The increase reflects higher spending on preclinical and manufacturing activities for PRS-220, an increase in manufacturing costs across multiple immuno-oncology programs, higher clinical costs on cinrebafusp alfa and higher employee related costs. These increases were partially offset by lower manufacturing costs on PRS-060, which were fully reimbursed.

G&A Expense – G&A expenses were $16.5 million for the year ended December 31, 2021, compared to $16.7 million for the year ended December 31, 2020. Total G&A spending was consistent year-over-year as higher fixed and variable compensation and higher insurance costs in 2021 were offset by lower legal, accounting, and project management costs, along with lower one-time office and building equipment costs related to the move to the new R&D facility in Hallbergmoos, Germany in the prior year.

Other Income – For the year ended December 31, 2021, $3.7 million of other income was recorded for PRS-220 program costs that qualified for reimbursement under the Bavarian grant that was announced in June 2021. The Bavarian government reimburses these qualifying program costs as incurred over the PRS-220 development period.

Net Loss – Net loss was $45.7 million or $(0.71) per share for the year ended December 31, 2021, compared to a net loss of $37.2 million or $(0.68) per share for the year ended December 31, 2020.

Conference Call :

Pieris management will host a conference call beginning at 8:00 AM EST on Tuesday, March 1, 2022, to discuss the full-year financial results and provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). Alternatively, a listen-only audio webcast of the call can be accessed here .

For those unable to participate in the conference call or listen to the webcast, a replay will be available on the Investors section of the Company’s website, www.pieris.com .

United Therapeutics Corporation to Present at the Cowen 42nd Annual Health Care Conference

On March 1, 2022 United Therapeutics Corporation (Nasdaq: UTHR) reported that Michael Benkowitz, President and Chief Operating Officer, will provide an overview and update on the company’s business during a fireside chat session at the Cowen 42nd Annual Health Care Conference (Press release, United Therapeutics, MAR 1, 2022, View Source [SID1234609283]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The session will take place virtually on Tuesday, March 8, 2022, from 11:10 a.m. to 11:40 a.m., Eastern Standard Time, and can be accessed via a live webcast on the United Therapeutics website at View Source An archived, recorded version of the session will be available approximately 24 hours after the session ends and can be accessed at the same location for 90 days.

Repare Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2021 Financial Results

On March 1, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Repare Therapeutics, MAR 1, 2022, View Source [SID1234609282]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We achieved several key milestones in 2021 to advance our innovative, synthetic lethality-based pipeline across multiple clinical programs," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "We are particularly pleased with important progress in our RP-3500 program and to have initiated our second clinical program, RP-6306, a first-in-class oral PKMYT1 inhibitor, and look forward to starting IND-enabling studies of our Polθ inhibitor program in the first half of 2022 as part of our growing clinical pipeline."

Mr. Segal added: "2022 is expected to be another exciting year for Repare, beginning in the first quarter 2022 with the Phase 2 (Module 2) expansion of the RP-3500 TRESR trial and the initiation of the TRESR Phase 1 monotherapy pediatric module in the first quarter of 2022. We anticipate comprehensive monotherapy TRESR Module 1 clinical data in the second quarter of 2022, and also look forward to initial data from the Phase 1 RP-6306 monotherapy MYTHIC trial in late 2022."

2021 Highlights and 2022 Outlook:

Announced initial Phase 1 RP-3500 monotherapy data from Phase 1/2 TRESR trial at AACR (Free AACR Whitepaper)-NCI-EORTC
Initial Phase 1 results provided clinical proof of concept and validated Repare’s SNIPRx platform for molecular selection of tumors for therapy with RP-3500.
Preliminary data showed that monotherapy RP-3500 appears safe and well tolerated, with compelling early efficacy signals across multiple genotypes and tumor types in heavily pretreated patients.
Recommended Phase 2 dose and schedule for further monotherapy RP-3500 evaluation was determined to be 160mg, taken weekly for 3 days on and 4 days off.
Key TRESR Milestones in 2022:
Initiated a monotherapy Phase 2 TRESR trial of RP-3500 for the treatment of solid tumors with specific synthetic-lethal genomic alterations including those in the ATM gene (ataxia telangiectasia mutated kinase), in tumors with ATM loss of function and in tumors with other STEP2 genomic alteration in February 2022.
Initiated recruitment in a Phase 1 pediatric module of TRESR trial of RP-3500 monotherapy and enrollment of a first patient is expected in the first quarter of 2022.
Comprehensive monotherapy Phase 1 (Module 1) clinical data from 120 patients enrolled in the Phase 1/2 TRESR trial of RP-3500 is expected in the second quarter of 2022.
Determination of recommended Phase 2 dose of RP-3500 in combination with gemcitabine, a trial that began enrolling patients in December 2021, is expected in the second half of 2022.
Early clinical data readout for PARPi combination from Phase 1/2 TRESR trial and ATTACC trial of RP-3500 in combination with, collectively, three marketed PARP inhibitors is expected in the third quarter of 2022.
Advanced RP-6306, a first-in-class, oral PKMYT1 inhibitor both as monotherapy and in combination with gemcitabine and in combination with FOLFIRI
In December 2021, the Company began dosing in a Phase 1 clinical trial of RP-6306, a first-in-class small molecule candidate targeting PKMYT1, in combination with gemcitabine for the treatment of molecularly selected advanced solid tumors.
In February 2022, the Company initiated recruitment in the Phase 1 MINOTAUR clinical trial of RP-6306 in combination with FOLFIRI for the treatment of molecularly selected advanced solid tumors.
Early readout of monotherapy Phase 1 clinical data is expected in late 2022.
Advanced the development of earlier stage discovery programs
The Company is expected to initiate IND-enabling studies of its third synthetic lethal asset, the Polθ inhibitor program, in the first half of 2022.
Recent Corporate Updates:

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).
In November 2021, the Company announced the closing of an upsized follow-on public offering yielding aggregate gross proceeds of approximately $101.2 million, or net proceeds of approximately $94.3 million, after deducting underwriting commissions and offering expenses of $0.8 million.
Fourth Quarter and Full Year 2021 Financial Results:

Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of December 31, 2021 were $341.9 million, including the proceeds from the follow-on public offering in November 2021. Repare believes its current cash and cash equivalents and marketable securities will be sufficient to fund planned operations through 2023.
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $28.0 million and $90.0 million for the three and twelve month periods ended December 31, 2021, respectively, as compared to $12.4 million and $40.1 million for the three and twelve month periods ended December 31, 2020. The increase in Net R&D expenses for the three and twelve month periods were primarily due to increases in development costs related to the Company’s RP-3500 and RP-6306 programs, as well as increases in personnel related expenses, including share-based compensation.
General and administrative (G&A) expenses: G&A expenses were $7.6 million and $26.2 million for the three and twelve month periods ended December 31, 2021, respectively, as compared to $4.8 million and $14.3 million for the three and twelve month periods ended December 31, 2020, respectively. The increase in G&A expenses for the three and twelve month periods were due to personnel related costs, including share-based compensation and, for the twelve month period, the increase was also due to D&O insurance which increased as a result of the Company’s IPO in June 2020.
Net loss: Net loss was $28.3 million, or $0.70 per share and $106.9 million, or $2.83 per share, in the three and twelve month periods ended December 31, 2021, respectively, and $15.3 million, or $0.41 per share and $53.4 million, or $2.66 per share in the three and twelve month periods ended December 31, 2020, respectively.
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 patients most likely to achieve clinical benefit from resulting product candidates.

Avidity Biosciences Reports Fourth Quarter and Year-End 2021 Financial Results and Recent Highlights

On March 1, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the fourth quarter and year ended December 31, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAR 1, 2022, View Source [SID1234609281]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"2021 was a pivotal year for Avidity, the field of RNA therapeutics and for the DM1 community as the first patient was dosed with an AOC as part of the AOC 1001 MARINA trial," said Sarah Boyce, president and chief executive officer. "This milestone transitioned us into a clinical-stage biopharmaceutical company and we continue to make great strides with our advancing and expanding pipeline and AOC platform technology. By the end of 2022, we plan to have three programs in three different rare diseases in the clinic with AOC 1001, AOC 1020 and AOC 1044."

"We are in a strong financial position with $406 million at year-end bolstered through approximately $175 million raised in 2021, inclusive of $155 million of net proceeds from our first follow on financing. We are well funded into 2024 which we expect will allow us to complete the MARINA trial, advance the AOC 1044 and AOC 1020 programs into clinical development and to continue to invest in expanding our pipeline and the AOC platform in muscle and beyond," said Mike MacLean, chief financial officer.

2021 Key Highlights

AOC 1001 Achievements

The Company initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).
The FDA and the European Medicines Agency (EMA) granted AOC 1001 Orphan Designation and the FDA granted AOC 1001 Fast Track Designation.
The MARINA trial is on track for a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the trial participants in Q4 2022.
Pipeline Advancements

AOC 1044 was named as the clinical development candidate for the Duchenne Muscular Dystrophy (DMD) program targeting Exon 44. AOC 1044 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
AOC 1020 was named as the clinical development candidate for the facioscapulohumeral muscular dystrophy (FSHD) program. AOC 1020 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
Avidity also entered a collaboration with the FSHD Clinical Trial Research Network (FSHD CTRN) to support a natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can potentially accelerate and support future clinical trial design.
Organizational Highlights

Avidity recently appointed Steve Hughes, M.D. as chief medical officer. Dr. Hughes brings over 20 years of experience in the biotechnology industry and has extensive experience in RNA-based treatments and rare diseases.
Fourth Quarter and Year-End 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $405.5 million as of December 31, 2021, which reflects $174.7 million raised in 2021, inclusive of $155.1 million of net proceeds from our first follow on financing.

Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $1.9 million for the fourth quarter of 2021 compared with $2.1 million for the fourth quarter of 2020, and $9.3 million for the full year 2021 compared with $6.8 million for the full year 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $33.0 million for the fourth quarter of 2021 compared with $13.6 million for the fourth quarter of 2020, and $101.2 million for the full year 2021 compared with $37.6 million for the full year 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $7.4 million for the fourth quarter of 2021 compared with $4.8 million for the fourth quarter of 2020, and $26.2 million for the full year 2021 compared with $13.5 million for the full year 2020. The increases were primarily due to higher personnel costs. The full year increase was also due to higher professional fees and insurance costs.