On February 25, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported operational highlights and financial results for the fourth quarter and year ended December 31, 2020 (Press release, Intellia Therapeutics, FEB 25, 2021, View Source [SID1234575597]).

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"Intellia’s achievements in 2020 reflect important progress on both our full-spectrum strategy and our mission to deliver curative genome editing treatments for people with severe diseases. Dosing our first patient with NTLA-2001, the first-ever systemically delivered CRISPR-based therapy, was a major milestone for our team, as we completed our transition to a clinical-stage company in the fourth quarter," said Intellia President and Chief Executive Officer, John Leonard, M.D. "Looking ahead, we are focused on our three core priorities for 2021: clinical validation of our approach with NTLA-2001, advancement and expansion of our in vivo and ex vivo pipeline, and continued platform innovation. We have made steady progress in our global Phase 1 study of NTLA-2001 and look forward to sharing our first clinical data this year. Additionally, we are on track to submit first-in-human regulatory applications to begin clinical studies of NTLA-5001 for AML and NTLA-2002 for HAE, and we plan to nominate at least one new development candidate from our research portfolio."

Fourth Quarter 2020 and Recent Operational Highlights

NTLA-2001 for ATTR: NTLA-2001 is the first systemically delivered CRISPR-based therapy dosed in a patient and could potentially be the first curative treatment for ATTR. By applying the Company’s in vivo lipid nanoparticle (LNP) delivery technology, NTLA-2001 offers the possibility of halting and reversing the disease with potent, lifelong transthyretin (TTR) protein reduction after a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead party, and Regeneron Pharmaceuticals, Inc. (Regeneron).

The Company anticipates reporting interim clinical data from the Phase 1 study this year. These results are expected to characterize the emerging safety and activity profile of NTLA-2001 at the initial dose levels.
In November, Intellia dosed the first patient in its global Phase 1 study evaluating NTLA-2001 in adults with hereditary ATTR with polyneuropathy (hATTR-PN). The Company continues to enroll patients in the study and is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy.
Intellia intends to evaluate NTLA-2001 in a broader ATTR population of both polyneuropathy and cardiomyopathy patients following its Phase 1 safety assessment and dose optimization.
NTLA-5001 for AML: NTLA-5001 is a potential best-in-class engineered T cell therapy designed to treat all genetic subtypes of AML. This investigational candidate is an autologous T cell receptor (TCR)-T cell therapy targeting the Wilms’ Tumor 1 (WT1) antigen utilizing Intellia’s proprietary cell engineering process.

Intellia plans to submit an Investigational New Drug (IND) application or equivalent regulatory application for NTLA-5001 in mid-2021. This first-in-human trial is expected to evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies.
NTLA-5001 shows high anti-tumor activity in proof-of-concept mouse models of acute leukemias. The preclinical data presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December highlighted faster expansion and superior function of T cells manufactured by Intellia’s proprietary approach, compared to standard T cell engineering approaches currently in use.
The Company is also evaluating the potential use of NTLA-5001 to treat WT1-positive solid tumors in preclinical studies.
NTLA-2002 for HAE: NTLA-2002 aims to prevent attacks for people living with HAE after a single course of treatment. Intellia is applying its modular LNP delivery system to develop NTLA-2002 to knock out the KLKB1 gene in the liver to permanently reduce plasma kallikrein activity. This approach is expected to provide continuous suppression of kallikrein activity and eliminate the significant treatment burden associated with currently available therapies for HAE patients.

Intellia commenced clinical manufacturing activities to support the Company’s plans to submit an IND or equivalent regulatory application in the second half of 2021.
The Company is applying insights gained from NTLA-2001 to expedite clinical development of NTLA-2002. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE.
The Company plans to present additional preclinical results in support of NTLA-2002 at the upcoming American Academy of Allergy, Asthma & Immunology (AAAAI) 2021 Annual Meeting, taking place virtually from February 26 – March 1, 2021.
Modular Platform and Pipeline Expansion: Intellia is advancing its modular platform technologies to broaden the in vivo and ex vivo applications of genome editing. This includes developing capabilities for innovative CRISPR/Cas9-mediated in vivo editing in multiple tissue types, targeted transgene insertion and an allogeneic approach for the development of "off-the-shelf" T cell therapies. These efforts will support new therapeutic candidates for genetic diseases requiring removal and/or restoration of a protein, and next-generation engineered cell therapies for cancers and auto-immune diseases.

Intellia plans to nominate at least one additional development candidate in 2021 and expects to present preclinical data at upcoming scientific conferences highlighting research advancements and platform innovations.
Intellia demonstrated the modularity of its targeted insertion approach for a second target, in non-human primates, showing insertion of the SERPINA1 gene produced normal levels of human alpha-1 antitrypsin (AAT) after a single administration. In December, these results were presented at the Alpha-1 Foundation’s 20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency. Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene.
Intellia is advancing preclinical validation of in vivo hematopoietic stem cell (HSC) genome editing using the Company’s proprietary non-viral delivery systems and CRISPR/Cas9 technology to potentially cure sickle cell disease. This research is supported by a grant from the Bill & Melinda Gates Foundation.
Financing: In December, Intellia closed an underwritten public offering of 5,513,699 shares of common stock, including the exercise in full of the underwriters’ option to purchase additional shares, at the public offering price of $36.50 per share. Intellia received aggregate gross proceeds of approximately $201 million, before underwriting discounts and commissions and offering expenses.
Upcoming Events

The Company will participate in the following events during the first quarter of 2021:

AAAAI Annual Meeting, February 26–March 1, Virtual
Keystone Symposium: Precision Engineering of the Genome, Epigenome and Transcriptome, March 8-10, Virtual
Barclays Capital Global Healthcare Conference, March 8, Virtual
Oppenheimer’s 31st Annual Healthcare Conference, March 16, Virtual
Cold Spring Harbor Laboratories meeting on Nucleic Acid Therapies, March 24 – 26, Virtual
Upcoming Milestones

The Company has set forth the following for pipeline progression:

ATTR: Report initial clinical data from Phase 1 study of NTLA-2001 in 2021
AML: Submit an IND or IND-equivalent for NTLA-5001 in mid-2021
HAE: Submit an IND or IND-equivalent for NTLA-2002 in 2H 2021
Nominate at least one new development candidate in 2021
Fourth Quarter and Full-Year 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $597.4 million as of December 31, 2020, compared to $284.5 million as of December 31, 2019. The increase was driven by net proceeds of $296.6 million from our follow-on public offerings, $100.0 million upfront payment from the Regeneron collaboration expansion, which included a $30.0 million equity investment, $49.5 million of net equity proceeds raised from the Company’s "At the Market" (ATM) agreement, $18.2 million from the Regeneron and Novartis Institutes for BioMedical Research, Inc. (Novartis) collaborations and $13.2 million in proceeds from employee-based stock plans. These increases were offset in part by cash used to fund operations of approximately $164.6 million.
Collaboration Revenue: Collaboration revenue decreased by $4.3 million to $6.6 million during the fourth quarter of 2020, compared to $10.9 million during the fourth quarter of 2019. The decrease was primarily driven by a decrease in Novartis revenue as the research portion of the collaboration ended in December 2019.
R&D Expenses: Research and development expenses increased by $6.5 million to $38.2 million during the fourth quarter of 2020, compared to $31.7 million during the fourth quarter of 2019. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs and expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $1.8 million to $10.8 million during the fourth quarter of 2020, compared to $9.0 million during the fourth quarter of 2019. This increase was primarily related to employee related expenses, including stock-based compensation of $1.3 million.
Net Loss: The Company’s net loss was $42.2 million for the fourth quarter of 2020, compared to $28.3 million during the fourth quarter of 2019.
Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of December 31, 2020 will enable the Company to fund its robust R&D plans, anticipated operating expenses and capital expenditure requirements at least through the next 24 months. This expectation excludes any strategic use of capital not currently in the Company’s base-case planning assumptions.

Conference Call to Discuss Fourth Quarter and Full-Year 2020 Earnings

The Company will discuss these results on a conference call today, February 25, 2021, at 8 a.m. ET.

To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call.
All participants should ask to be connected to the Intellia Therapeutics conference call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, beginning on February 25, 2021 at 12 p.m. ET.

On February 25, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2020, and provided a corporate update (Press release, BioCryst Pharmaceuticals, FEB 25, 2021, View Source [SID1234575596]).

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"With two approvals for ORLADEYO, our launch in the U.S., proof of concept data for BCX9930 in complement-mediated diseases, a successful phase 1 trial of BCX9250 for FOP and the addition of more than $425 million through our May and December financings, 2020 was a transformational year for BioCryst," said Jon Stonehouse, president and chief executive officer of BioCryst.

"Using our strong balance sheet as a foundation, we expect to continue this transformation in 2021 with ORLADEYO generating revenue in the U.S., Japan and Europe, and the opportunity to significantly advance the 9930 program as an oral monotherapy for both PNH, and renal complement-mediated diseases," Stonehouse added.

Program Updates and Key Milestones

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

BioCryst launched ORLADEYO in the United States following U.S. Food and Drug Administration (FDA) approval on December 3, 2020, and product shipments began on December 16, 2020.

On January 22, 2021, the company announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan had granted marketing and manufacturing approval for oral, once-daily ORLADEYO 150 mg for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

ORLADEYO is the first and only prophylactic HAE medication approved in Japan and will be commercialized in Japan by BioCryst’s partner, Torii Pharmaceutical Co., Ltd. OrphanPacific, Inc. is BioCryst’s representative partner in Japan and holds the marketing authorization.

Torii will launch ORLADEYO in Japan following the successful completion of BioCryst’s pricing negotiations with the Japanese National Health Insurance System (NHI).

BioCryst is eligible to receive an additional milestone payment of $15 million from Torii upon receipt of a reimbursement price from the NHI in excess of the threshold specified in the agreement with Torii. In addition, BioCryst will receive tiered royalties ranging from 20 percent to potentially 40 percent of Japanese net sales.
In Europe, the Committee for Medicinal Products for Human Use (CHMP) is scheduled to review the ORLADEYO marketing authorization application this week. The company expects approval from the European Commission (EC) approximately 60 days following a positive opinion from the CHMP.

On October 30, 2020, the company announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) had granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE patients in the UK aged 12 years and older can gain access to ORLADEYO for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the EC.

In the fourth quarter of 2020, the company presented data in several manuscripts and abstracts.

On October 22, 2020, the company announced that data from the first 24 weeks of the Phase 3 APeX-2 trial of ORLADEYO in 121 HAE patients ages 12 years or older had been published online by the Journal of Allergy and Clinical Immunology.

On November 13, 2020, the company presented data in several abstracts at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies.

On November 30, 2020, the company announced that the journal Allergy had published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating ORLADEYO for the prophylactic treatment of HAE.
Complement Oral Factor D Inhibitor Program – BCX9930

The company has completed the enrollment of its ongoing dose ranging trial in treatment-naïve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients, and PNH patients with an inadequate response to C5 inhibitors. The company plans to present data from the 16 enrolled PNH patients (10 treatment naïve and six inadequate C5 responders) at its upcoming R&D day on March 22.

In previously reported data from treatment-naïve (no prior treatment with C5 inhibitors) PNH patients receiving doses of oral BCX9930 through 400 mg bid, oral BCX9930 drove rapid and dose-dependent reductions in key biomarkers, including LDH, and increasing hemoglobin levels in all PNH patients in the trial. Increases in hemoglobin levels were maintained without transfusions. BCX9930 has been safe and well tolerated at all doses in the trial. No drug-related serious adverse events have been reported.
Additional Updates

On December 7, 2020, the company announced transactions totaling $325 million in funding for BioCryst, with $250 million available at closing, to support the launch of ORLADEYO in HAE and the development of BCX9930. Royalty Pharma provided BioCryst with an upfront cash payment of $125 million and will receive royalties on direct annual net sales of ORLADEYO up to $550 million, and a tiered percentage of sublicense revenue for ORLADEYO in certain territories. In addition, Royalty Pharma will receive a 1.0% royalty on global net sales of BCX9930, if approved. A fund managed by Athyrium Capital Management provided BioCryst with a $200 million credit facility, of which BioCryst drew $125 million at closing. The additional capital will be available in two tranches at BioCryst’s option, upon reaching defined revenue milestones. The credit facility bears interest at LIBOR + 8.25% (with a LIBOR floor of 1.75%) and is interest-only for the entire five-year term, with all outstanding principal due at maturity.

On December 21, 2020, the company announced that in a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting once-daily dosing.

On February 3, 2021, the company announced that the FDA had approved a supplemental new drug application for RAPIVAB (peramivir injection) expanding the patient population of RAPIVAB for the treatment of acute uncomplicated influenza to include patients six months and older who have been symptomatic for no more than two days. Prior to this approval, RAPIVAB had been indicated for patients two years and older.
Fourth Quarter 2020 Financial Results

For the three months ended December 31, 2020, total revenues were $4.0 million, compared to $39.7 million in the fourth quarter of 2019. In the fourth quarter of 2019, we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement. Additionally, in that period we recognized $13.9 million of RAPIVAB product sales under our U.S. Department of Health and Human Services (HHS) contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract. ORLADEYO revenues in the fourth quarter of 2020 were $0.1 million.

Research and development (R&D) expenses for the fourth quarter of 2020 increased to $35.4 million from $26.8 million in the fourth quarter of 2019, primarily due to increased investment in our Factor D and galidesivir programs, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2020 increased to $21.0 million, compared to $10.5 million in the fourth quarter of 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.

Interest expense was $5.6 million in the fourth quarter of 2020, compared to $3.1 million in the fourth quarter of 2019. This increase was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.

Net loss for the fourth quarter of 2020 was $60.5 million, or $0.34 per share, compared to a net loss of $2.6 million, or $0.02 per share, for the fourth quarter of 2019.

Cash, cash equivalents, restricted cash and investments totaled $302.6 million at December 31, 2020, and reflect an increase from $137.8 million at December 31, 2019. Cash, cash equivalents, restricted cash and investments include $250 million in cash received through transactions with Royalty Pharma and Athyrium Capital Management in December 2020. Operating cash use for the fourth quarter of 2020 was $49.9 million, and for the full year of 2020 was $147.9 million.

Full Year 2020 Financial Results

For the full year ended December 31, 2020, total revenues were $17.8 million, compared to $48.8 million in the full year ended December 31, 2019. In the fourth quarter of 2019 we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement, with the remaining amount of $1.9 million recognized in 2020. Additionally, in the fourth quarter of 2019 we recognized $13.9 million of RAPIVAB product sales under our HHS contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract.

R&D expenses in full year 2020 increased to $123.0 million from $107.1 million in full year 2019, primarily due to increased investment in our Factor D and galidesivir programs, and an increase in other research, preclinical and development activities, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.

SG&A expenses in full year 2020 increased to $67.9 million, compared to $37.1 million in full year 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.

Interest and other income was $9.4 million in full year 2020, compared to $1.9 million in full year 2019. The increase was primarily due to the settlement of arbitration proceedings related to our Seqirus dispute in the first quarter of 2020.

Interest expense was $14.5 million in full year 2020, compared to $11.9 million in full year 2019. This was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.

Net loss for full year 2020 was $182.8 million, or $1.09 per share, compared to a net loss of $108.9 million, or $0.94 per share, for full year 2019.

Financial Outlook for 2021

In the launch period for ORLADEYO, the company is not providing specific revenue or operating expense guidance. Based on our expectations for revenue, operating expenses, and our option to access an additional $75 million from our existing credit facility, we believe our current cash runway takes us into 2023.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 6779206. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 6779206.

On February 25, 2021 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the fourth quarter and full year ended December 31, 2020, and provided a corporate update (Press release, BioCryst Pharmaceuticals, FEB 25, 2021, View Source [SID1234575594]).

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!

"With two approvals for ORLADEYO, our launch in the U.S., proof of concept data for BCX9930 in complement-mediated diseases, a successful phase 1 trial of BCX9250 for FOP and the addition of more than $425 million through our May and December financings, 2020 was a transformational year for BioCryst," said Jon Stonehouse, president and chief executive officer of BioCryst.

"Using our strong balance sheet as a foundation, we expect to continue this transformation in 2021 with ORLADEYO generating revenue in the U.S., Japan and Europe, and the opportunity to significantly advance the 9930 program as an oral monotherapy for both PNH, and renal complement-mediated diseases," Stonehouse added.

Program Updates and Key Milestones

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

BioCryst launched ORLADEYO in the United States following U.S. Food and Drug Administration (FDA) approval on December 3, 2020, and product shipments began on December 16, 2020.

On January 22, 2021, the company announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan had granted marketing and manufacturing approval for oral, once-daily ORLADEYO 150 mg for prophylactic treatment of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

ORLADEYO is the first and only prophylactic HAE medication approved in Japan and will be commercialized in Japan by BioCryst’s partner, Torii Pharmaceutical Co., Ltd. OrphanPacific, Inc. is BioCryst’s representative partner in Japan and holds the marketing authorization.

Torii will launch ORLADEYO in Japan following the successful completion of BioCryst’s pricing negotiations with the Japanese National Health Insurance System (NHI).

BioCryst is eligible to receive an additional milestone payment of $15 million from Torii upon receipt of a reimbursement price from the NHI in excess of the threshold specified in the agreement with Torii. In addition, BioCryst will receive tiered royalties ranging from 20 percent to potentially 40 percent of Japanese net sales.
In Europe, the Committee for Medicinal Products for Human Use (CHMP) is scheduled to review the ORLADEYO marketing authorization application this week. The company expects approval from the European Commission (EC) approximately 60 days following a positive opinion from the CHMP.

On October 30, 2020, the company announced that the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) had granted ORLADEYO a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE patients in the UK aged 12 years and older can gain access to ORLADEYO for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the EC.

In the fourth quarter of 2020, the company presented data in several manuscripts and abstracts.

On October 22, 2020, the company announced that data from the first 24 weeks of the Phase 3 APeX-2 trial of ORLADEYO in 121 HAE patients ages 12 years or older had been published online by the Journal of Allergy and Clinical Immunology.

On November 13, 2020, the company presented data in several abstracts at the 2020 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology across HAE patients, caregivers and treating physicians showing many patients experience a significant treatment burden associated with current prophylactic HAE therapies.

On November 30, 2020, the company announced that the journal Allergy had published data from the APeX-J trial, a randomized, placebo-controlled trial conducted in Japan evaluating ORLADEYO for the prophylactic treatment of HAE.
Complement Oral Factor D Inhibitor Program – BCX9930

The company has completed the enrollment of its ongoing dose ranging trial in treatment-naïve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients, and PNH patients with an inadequate response to C5 inhibitors. The company plans to present data from the 16 enrolled PNH patients (10 treatment naïve and six inadequate C5 responders) at its upcoming R&D day on March 22.

In previously reported data from treatment-naïve (no prior treatment with C5 inhibitors) PNH patients receiving doses of oral BCX9930 through 400 mg bid, oral BCX9930 drove rapid and dose-dependent reductions in key biomarkers, including LDH, and increasing hemoglobin levels in all PNH patients in the trial. Increases in hemoglobin levels were maintained without transfusions. BCX9930 has been safe and well tolerated at all doses in the trial. No drug-related serious adverse events have been reported.
Additional Updates

On December 7, 2020, the company announced transactions totaling $325 million in funding for BioCryst, with $250 million available at closing, to support the launch of ORLADEYO in HAE and the development of BCX9930. Royalty Pharma provided BioCryst with an upfront cash payment of $125 million and will receive royalties on direct annual net sales of ORLADEYO up to $550 million, and a tiered percentage of sublicense revenue for ORLADEYO in certain territories. In addition, Royalty Pharma will receive a 1.0% royalty on global net sales of BCX9930, if approved. A fund managed by Athyrium Capital Management provided BioCryst with a $200 million credit facility, of which BioCryst drew $125 million at closing. The additional capital will be available in two tranches at BioCryst’s option, upon reaching defined revenue milestones. The credit facility bears interest at LIBOR + 8.25% (with a LIBOR floor of 1.75%) and is interest-only for the entire five-year term, with all outstanding principal due at maturity.

On December 21, 2020, the company announced that in a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting once-daily dosing.

On February 3, 2021, the company announced that the FDA had approved a supplemental new drug application for RAPIVAB (peramivir injection) expanding the patient population of RAPIVAB for the treatment of acute uncomplicated influenza to include patients six months and older who have been symptomatic for no more than two days. Prior to this approval, RAPIVAB had been indicated for patients two years and older.
Fourth Quarter 2020 Financial Results

For the three months ended December 31, 2020, total revenues were $4.0 million, compared to $39.7 million in the fourth quarter of 2019. In the fourth quarter of 2019, we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement. Additionally, in that period we recognized $13.9 million of RAPIVAB product sales under our U.S. Department of Health and Human Services (HHS) contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract. ORLADEYO revenues in the fourth quarter of 2020 were $0.1 million.

Research and development (R&D) expenses for the fourth quarter of 2020 increased to $35.4 million from $26.8 million in the fourth quarter of 2019, primarily due to increased investment in our Factor D and galidesivir programs, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2020 increased to $21.0 million, compared to $10.5 million in the fourth quarter of 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.

Interest expense was $5.6 million in the fourth quarter of 2020, compared to $3.1 million in the fourth quarter of 2019. This increase was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.

Net loss for the fourth quarter of 2020 was $60.5 million, or $0.34 per share, compared to a net loss of $2.6 million, or $0.02 per share, for the fourth quarter of 2019.

Cash, cash equivalents, restricted cash and investments totaled $302.6 million at December 31, 2020, and reflect an increase from $137.8 million at December 31, 2019. Cash, cash equivalents, restricted cash and investments include $250 million in cash received through transactions with Royalty Pharma and Athyrium Capital Management in December 2020. Operating cash use for the fourth quarter of 2020 was $49.9 million, and for the full year of 2020 was $147.9 million.

Full Year 2020 Financial Results

For the full year ended December 31, 2020, total revenues were $17.8 million, compared to $48.8 million in the full year ended December 31, 2019. In the fourth quarter of 2019 we recognized revenue of $20.1 related to a one-time upfront milestone per the Torii agreement, with the remaining amount of $1.9 million recognized in 2020. Additionally, in the fourth quarter of 2019 we recognized $13.9 million of RAPIVAB product sales under our HHS contract, while in the fourth quarter 2020 we had no RAPIVAB product sales under our HHS contract.

R&D expenses in full year 2020 increased to $123.0 million from $107.1 million in full year 2019, primarily due to increased investment in our Factor D and galidesivir programs, and an increase in other research, preclinical and development activities, partially offset by a ramp down of clinical investment related to ORLADEYO, which launched commercially in the U.S. during December 2020.

SG&A expenses in full year 2020 increased to $67.9 million, compared to $37.1 million in full year 2019. The increase was primarily due to increased investment in commercial activities to support the U.S. launch of ORLADEYO.

Interest and other income was $9.4 million in full year 2020, compared to $1.9 million in full year 2019. The increase was primarily due to the settlement of arbitration proceedings related to our Seqirus dispute in the first quarter of 2020.

Interest expense was $14.5 million in full year 2020, compared to $11.9 million in full year 2019. This was due to service on the royalty and debt financings which were completed in December 2020. As part of those financings, there was also a loss on debt extinguishing related to the closing of our secured credit facility with MidCap Financial.

Net loss for full year 2020 was $182.8 million, or $1.09 per share, compared to a net loss of $108.9 million, or $0.94 per share, for full year 2019.

Financial Outlook for 2021

In the launch period for ORLADEYO, the company is not providing specific revenue or operating expense guidance. Based on our expectations for revenue, operating expenses, and our option to access an additional $75 million from our existing credit facility, we believe our current cash runway takes us into 2023.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 6779206. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 6779206.

On February 25, 2021 CTI BioPharma Corp. (NASDAQ: CTIC) reported that management will provide a corporate overview at the Cowen 41st Annual Health Care Conference at 12:10 p.m. EST (Press release, CTI BioPharma, FEB 25, 2021, https://cbc.gcs-web.com/news-releases/news-release-details/cti-biopharma-present-cowen-41st-annual-health-care-conference [SID1234575593]). The conference will be held in a virtual meeting format.

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!

Presentation details:

Event: Cowen 41st Annual Health Care Conference
Date: Thursday, Mar. 4, 2021
Time: 12:10 p.m. EST

The presentation will be webcast live and available for replay from the Investors section of CTI BioPharma’s website at www.ctibiopharma.com.

On February 25, 2021 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported financial results for the fourth quarter and year ended December 31, 2020 and provided a corporate update (Press release, Jounce Therapeutics, FEB 25, 2021, View Source [SID1234575592]).

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!

"2020 proved to be a year of important pipeline execution and corporate development at Jounce despite the challenges presented by the COVID-19 pandemic. As we enter 2021, we are strongly positioned to execute on our two proof of concept studies, INNATE and SELECT, and continue to advance our sustainable discovery pipeline. Our potential first-in-class programs and biomarker approaches are aimed at bringing meaningful clinical benefit to the growing population of PD-(L)1 inhibitor naïve and experienced patients," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "The need for novel approaches targeting different immune cells in the tumor microenvironment highlights the importance of our translational science platform and our productive discovery engine. This approach has allowed us to generate multiple targets beyond T-cells, most notably our highest priority program, JTX-8064, targeting LILRB2, also known as ILT4. As we enter 2021, Jounce is poised to further our goal of bringing the right immunotherapies to the right patients."

Pipeline Update and Highlights:

JTX-8064 (LILRB2 / ILT4)

Initiated Phase 1 INNATE trial of JTX-8064: In January 2021, Jounce enrolled the first dose cohort in INNATE, a Phase 1 clinical trial of JTX-8064 alone and in combination with its PD-1 inhibitor, JTX-4014, or pembrolizumab. The trial is designed to progress quickly through dose escalation and demonstrate proof of concept in tumor specific expansion cohorts.

Presented JTX-8064 preclinical data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 35th Annual Meeting: In November 2020 at SITC (Free SITC Whitepaper), Jounce presented preclinical data for JTX-8064 that informed the indication selection and biomarker strategies for JTX-8064 to maximize potential therapeutic benefit for patients with solid tumor malignancies.
Vopratelimab (ICOS) and JTX-4014 (PD-1)

Initiated enrollment in the Phase 2 SELECT trial of vopratelimab: In October 2020, Jounce initiated enrollment in the randomized Phase 2 SELECT trial to evaluate vopratelimab in combination with JTX-4014 versus JTX-4014 alone in immunotherapy naïve TISvopra biomarker-selected, second line non-small cell lung cancer patients. COVID-19 related delays are currently impacting patient enrollment, and Jounce now anticipates reporting data from the SELECT trial in 2022.

Continued to advance JTX-4014 as a combination agent: JTX-4014 is a PD-1 inhibitor intended for combination with Jounce’s broad pipeline beginning with its two ongoing proof of concept studies, the INNATE trial and the SELECT trial. The SELECT trial will also provide additional important single agent data for JTX-4014 in a new biomarker selection paradigm..
JTX-1811 (CCR8)

Established exclusive license agreement with Gilead for the development and commercialization of JTX-1811: In October 2020, Jounce licensed to Gilead Sciences, Inc. ("Gilead") the worldwide rights to JTX-1811, a potential first-in-class antibody designed to bind to CCR8 and selectively deplete immunosuppressive tumor-infiltrating T regulatory cells. Upon clearance of an investigational new drug application ("IND"), JTX-1811 will transition to Gilead for clinical development and potential commercialization. In addition to an $85.0 million upfront and a $35.0 million equity investment, Jounce has the potential to earn up to $685.0 million in milestones as well as royalties on worldwide sales. Jounce continues to progress JTX-1811 to IND clearance and remains on track for an IND filing in the first half of 2021.
Discovery Pipeline

Productive discovery engine with IND every 12 to 18 months: Jounce continues to invest in and advance its growing immuno-oncology pipeline. Its discovery engine is built upon the capability to thoroughly investigate different cell types in the tumor microenvironment, including T cells, myeloid cells and stromal cells.
Fourth Quarter and Full Year 2020 Financial Results:

Cash position: As of December 31, 2020, cash, cash equivalents and investments were $213.2 million, compared to $170.4 million as of December 31, 2019. The increase in cash, cash equivalents and investments was primarily due to receipt of $120.0 million in proceeds from the license and stock purchase agreements with Gilead and $14.5 million received during 2020 under Jounce’s at-the-market offering program ("ATM"), offset by operating expenses incurred during the year. In January 2021, the Company completed the sale of all available amounts under the existing ATM with the sale of 3,156,200 shares for net proceeds of $30.2 million.
License and collaboration revenue: $62.3 million of license and collaboration revenue was recognized during the fourth quarter of 2020. Jounce did not recognize any license and collaboration revenue for the same period in 2019. License and collaboration revenue was $62.3 million for the full year 2020, compared to $147.9 million for the full year 2019. Revenue recognized during 2020 was related to Jounce’s license agreement with Gilead. Revenue recognized during 2019 was comprised of $50.0 million of cash revenue related to Jounce’s JTX-8064 license agreement with Celgene and $97.9 million of non-cash revenue recognition related to the $225.0 million upfront payment received in July 2016 under the Celgene collaboration agreement.
Research and development expenses: Research and development expenses were $20.0 million for the fourth quarter of 2020, compared to $16.6 million for the same period in 2019. Research and development expenses were $78.7 million for the full year 2020, compared to $67.1 million for the full year 2019. The increase in research and development expenses for the full year 2020 was primarily due to $7.9 million of increased clinical and regulatory expense primarily attributable to the SELECT clinical trial, $3.2 million of increased manufacturing and IND-enabling expenses and $2.9 million of increased employee compensation costs. These increases were partially offset by $0.9 million and $0.8 million of decreased other research costs, primarily related to reduced travel, and lab consumable costs, respectively.
General and administrative expenses: General and administrative expenses were $6.9 million for both the fourth quarter of 2020 and 2019. General and administrative expenses were $28.8 million for the full year 2020, compared to $27.9 million for the full year 2019. The increase in general and administrative expenses for full year 2020 was primarily attributable to $1.5 million of increased employee compensation costs.
Net income (loss): Net income was $35.5 million for the fourth quarter of 2020, resulting in basic net income per share of $0.90 and diluted net income per share of $0.86. Net loss was $22.7 million for the same period in 2019, resulting in basic and diluted net loss per share of $0.68. Net loss was $43.8 million for the full year 2020, resulting in basic and diluted net loss per share of $1.24. Net income was $56.8 million for the full year 2019, resulting in basic net income per share of $1.72 and diluted net income per share of $1.66. Net loss for the full year 2020 was attributable to increased operating expenses, offset by $62.3 million of license revenue recognized under Jounce’s agreement with Gilead. Net income for the full year 2019 was primarily attributable to $147.9 million of revenue recognized under the Celgene license and collaboration agreements in the year.
Financial Guidance:

Based on its current operating and development plans, Jounce reiterates its financial guidance for 2021. Gross cash burn on operating expenses and capital expenditures for the full year 2021 is expected to be approximately $95.0 million to $110.0 million. Given the strength of its balance sheet, Jounce expects its existing cash, cash equivalents and investments to be sufficient to enable the funding of its operating expenses and capital expenditure requirements through the second quarter of 2023.

Conference Call and Webcast Information:

Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 4698355. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. The webcast will be archived and made available for replay on Jounce’s website approximately two hours after the call and will be available for 30 days.