On November 9, 2021 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a biopharmaceutical company developing a broad pipeline of novel immunotherapies by applying its proprietary technology to its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms, reported financial results for the quarter ended September 30, 2021 (Press release, Janux Therapeutics, NOV 9, 2021, View Source [SID1234594899]).

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"In the third quarter, we continued to utilize our novel TRACTr and TRACIr platforms to advance our pipeline of next generation immunotherapies. We remain on-track to execute key milestones, including two IND submissions next year for our PSMA-TRACTr and EGFR-TRACTr candidates," said David Campbell, Ph.D., President and CEO of Janux. "Further, we welcomed Dr. Ron Barrett and Alana McNulty to Janux’s Board of Directors. These two key leadership additions bring important expertise to the company as we work to advance our programs towards the clinic."

RECENT BUSINESS HIGHLIGHTS AND FUTURE MILESTONES:

TRACTr product candidates advancing as planned. Janux’s lead TRACTr programs of next-generation T cell engagers remain on-track.

In the first half of 2022, Janux expects to submit an Investigational New Drug (IND) application for its PSMA-TRACTr candidate, targeting prostate-specific membrane antigen (PSMA).
In the second half of 2022, Janux expects to submit an IND application for its EGFR-TRACTr candidate, targeting epidermal growth factor receptor (EGFR).
In 2023, Janux expects to submit an IND application for its TROP2-TRACTr, targeting trophoblast cell surface antigen 2 (TROP2).
On track for selection of TRACIr development candidate in 2022 as planned. Janux is applying its TRACIr technology to develop a costimulatory bispecific product candidate against programmed death-ligand 1 (PD-L1) and Cluster of Differentiation 28 (CD28) to further enhance the anti-tumor activity of T cells. This will be Janux’s first program derived from its TRACIr platform.
Strengthened Board of Directors with appointment of Ron Barrett, Ph.D., and Alana McNulty. Dr. Barrett is a scientist entrepreneur with more than 30 years of experience in the biopharmaceutical industry as a co-founder and leader of biopharmaceutical companies and has been responsible for advancing research that led to FDA approval of three drugs. Ms. McNulty has more than 30 years of experience in finance and business development for private and publicly traded biopharmaceutical companies, playing a key role over her career in a breadth of strategic transactions.
THIRD QUARTER 2021 FINANCIAL HIGHLIGHTS:

Cash and cash equivalents and short-term investments: As of September 30, 2021, Janux reported cash and cash equivalents and short-term investments of $387.5 million, compared to $7.8 million at December 31, 2020.
Research and development expenses: Research and development expenses for the quarter ended September 30, 2021 were $8.4 million, compared to $0.8 million for the comparable period in 2020. The increase in research and development expenses in 2021 was primarily attributable to the development of Janux’s platform technologies and programs. Janux also incurred additional personnel-related expenses, including stock-based compensation, as operations grew in support of program advances.
General and administrative expenses: General and administrative expenses for the quarter ended September 30, 2021 were $3.6 million, compared to $0.4 million for the same period in 2020. The increase in general and administrative expenses was primarily attributable to an increase in personnel-related expenses, including stock-based compensation, due to increased headcount in 2021. The increase in general and administrative expenses were also due to an increase in legal fees, professional fees, and other various general and administrative expenses, as Janux now operates as a public company.
Net loss: For the quarter ended September 30, 2021, Janux reported a net loss of $10.8 million, compared to a net loss of $1.3 million for the comparable period in 2020.

On November 9, 2021 Acorda Therapeutics, Inc. (Nasdaq: ACOR) reported its financial results for the third quarter 2021 and changes to its leadership team (Press release, Acorda Therapeutics, NOV 9, 2021, View Source [SID1234594898]).

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"Acorda made significant progress this quarter. We saw a 34% increase in INBRIJA net sales over the same quarter in 2020, despite the continuing impact of the pandemic on our business. Today we announced an agreement with Esteve to commercialize Inbrija in Germany, the largest pharmaceutical market in Europe and fourth largest in the world. Esteve expects to launch INBRIJA there in mid-2022," said Ron Cohen, M.D., Acorda’s President and Chief Executive Officer. "We have also added two seasoned executives to Acorda’s leadership team. Mike Gesser is a highly experienced CFO who will enhance our efforts to maintain fiscal discipline and increase the efficiency of our organization. Neil Belloff has a long track record of success as a General Counsel for biotech companies and at the Securities and Exchange Commission. We thank Dr. Blank for his years of service to Acorda; under his leadership, Acorda secured marketing authorizations for INBRIJA from both the FDA and the European Medicines Agency. We are pleased that Dr. Blank will continue to provide the benefit of his expertise to Acorda as a consultant."

"We are making excellent progress on our top corporate priorities: accelerating Inbrija’s sales trajectory, maintaining our Ampyra brand in the face of generic competition, commercializing Inbrija outside the US, which provides a significant additional revenue stream to Acorda, and aligning our operating expenses to our revenue. Our goal is to be cash flow positive on a run rate basis by the end of 2022."

Third Quarter 2021 Financial Results

For the quarter ended September 30, 2021, the Company reported INBRIJA net revenue of $7.8 million, compared to $5.8 million for the same quarter in 2020.

For the quarter ended September 30, 2021, the Company reported AMPYRA net revenue of $20.0 million compared to $27.3 million for the same quarter in 2020. In September 2018, AMPYRA lost its exclusivity and generics entered the market. Consequently, the Company expects AMPYRA revenue to continue to decline.

Research and development (R&D) expenses for the quarter ended September 30, 2021 were $1.9 million, including $0.2 million of share-based compensation compared to $5.7 million, including $0.6 million of share-based compensation for the same quarter in 2020.

Sales, general and administrative (SG&A) expenses for the quarter ended September 30, 2021 were $29.6 million, including $0.6 million of share-based compensation, compared to $39.9 million, including $1.8 million of share-based compensation for the same quarter in 2020.

Change in fair value of derivative liability for the quarter ended September 30, 2021 was $(0.3) million compared to $(4.9) million for the same quarter in 2020.

Benefit from income taxes for the quarter ended September 30, 2021 was $3.1 million compared to a provision for income taxes of $1.5 million for the same quarter in 2020.

The Company reported a GAAP net loss of $27.1 million for the quarter ended September 30, 2021, or $2.43 per diluted share. GAAP net income in the same quarter of 2020 was $7.3 million, or $0.32 per diluted share.

Non-GAAP net loss for the quarter ended September 30, 2021 was $15.9 million, or $1.43 per diluted share. Non-GAAP net loss in the same quarter of 2020 was $10.9 million, or $1.36 per diluted share. This quarterly non-GAAP net loss measure, more fully described below under "Non-GAAP Financial Measures," excludes share-based compensation charges, non-cash interest charges on our debt, changes in the fair value of acquired contingent consideration, changes in the fair value of derivative liability related to our 2024 convertible senior secured notes, and expenses that pertain to non-routine corporate restructurings. A reconciliation of the GAAP financial results to non-GAAP financial results is included with the attached financial statements.

At September 30, 2021, the Company had cash, cash equivalents, and restricted cash of $62 million, compared to $103 million at year end 2020. Restricted cash includes $25 million in escrow related to the 6% semi-annual interest portion of the 2024 convertible senior secured notes, which is payable in cash or stock. If the Company elects to pay interest due in stock, a corresponding amount of restricted cash will be released from escrow.

For the full-year 2021, Acorda continues to expect AMPYRA net revenue to be $75 – $85 million, and operating expenses to be $130 – $140 million. The operating expense guidance is a non-GAAP projection that excludes restructuring costs and share-based compensation as more fully described below under "Non-GAAP Financial Measures."

INBRIJA Ex-US

Acorda announced that it has entered into distribution and supply agreements with Esteve Pharmaceuticals GmbH for the commercialization of INBRJIA in Germany. Acorda will receive a €5 million upfront signing fee, and will receive additional sales-based milestones. Acorda will also receive a significant double-digit percent of the selling price of INBRIJA in Germany in exchange for supply of the product. Esteve expects to launch INBRIJA in Germany in mid-2022.

Leadership Team

Michael Gesser has joined Acorda as Chief Financial Officer (CFO). Mr. Gesser was most recently the CFO of Tergus Pharma and has also held CFO positions at BioMedomics, Inc., HAP Innovations, LLC, Suntech Medical, Inc., and Osmotica Pharmaceutical Corp. Previous to those roles, he held several senior-level financial positions at Allergan Pharmaceuticals. Mr. Gesser received his M.B.A. from the Belk School of Business at the University of North Carolina at Charlotte and his B.S. in Finance at the Cameron School of Business at the University of North Carolina at Wilmington.

Neil Belloff has joined the Company as General Counsel. Mr. Belloff has over 30 years of business and legal experience and was formerly the Chief Operating Officer, General Counsel and Corporate Secretary of Eloxx Pharmaceuticals and held senior level positions at Celgene Corporation, Deutsche Telekom, AG, and the United States Securities and Exchange Commission. Mr. Belloff received his J.D. from the University of Bridgeport School of Law, M.A. from New York University, his B.A. from Queens College of the City University of New York, and completed post-graduate studies in the LL.M Program in Securities Regulation at Georgetown University Law Center. Andrew Mayer will remain Deputy General Counsel and Corporate Secretary.

Burkhard Blank, M.D., the Company’s Chief Medical Officer, will leave his position at the end of this year. He is expected to serve as a consultant to Acorda following his departure.

Mr. Gesser and Mr. Belloff were each granted options to purchase 85,000 shares of Acorda’s common stock (for an aggregate of 170,000 shares). In accordance with Nasdaq Listing Rule 5635(c)(4), these stock options were granted to Mr. Gesser and Mr. Belloff under the company’s 2016 Inducement Plan as a material inducement to their accepting employment with the company as Chief Financial Officer and General Counsel, respectively.

The stock options have an exercise price of $3.74 per share, equal to the closing price of the company’s common stock on the grant date of November 8, 2021, which is the date Mr. Gesser and Mr. Belloff commenced employment. The stock options will vest over four years, with 25% vesting on the one-year anniversary of the commencement of employment, and the remaining 75% vesting on a quarterly basis over the remaining three years thereafter, subject to continuing employment. The stock options have a 10-year term and are subject to the terms and conditions of the 2016 Inducement Plan.

Webcast

The Company will host a webcast in conjunction with its third quarter 2021 update and financial results today at 4:30 p.m. ET.

To participate in the Webcast, please use the following pre-registration link:

View Source
Once you have registered, you will receive a confirmation email with Webcast details. You will receive an email 2 hours prior to the start of the call with the link to join. The presentation will be available in the Investors section of www.acorda.com.

A replay of the audio portion will be available from 7:30 p.m. ET on November 9, 2021 until 11:59 p.m. ET on December 9, 2021. To access the replay, please dial (866) 813 9403 (domestic) or +44 204 525 0658 (international); access code 602929. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On November 9, 2021 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three and nine months ended September 30, 2021 and provided an operating forecast and program updates (Press release, Ligand, NOV 9, 2021, View Source [SID1234594897]). Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

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"Our business has performed exceptionally well over the past few months, and we are pleased to be reporting excellent third quarter financial results," said John Higgins, CEO of Ligand. "Our core royalties tied to Kyprolis and Evomela continue to grow nicely, and we have seen five approvals during the last year for new products or major markets that are now launching and are expected to fuel royalty growth next year and beyond."

"As announced separately today, we are pursuing plans to split Ligand into two separate, publicly traded companies with one featuring the OmniAb business, and the other featuring Ligand’s existing collection of core royalties and the technologies, pipeline and contracts associated with the Pelican protein expression platform and the Captisol business," Higgins added. "Along with outside advisors we have concluded the time is right to pursue this strategic plan and accelerate investment into the OmniAb platform and technologies to further drive value."

Third Quarter 2021 Financial Results

Total revenues for the third quarter of 2021 were $64.8 million, compared with $41.8 million for the same period in 2020. Royalties for the third quarter of 2021 were $15.6 million, compared with $9.0 million for the same period in 2020. Captisol sales were $35.1 million for the third quarter of 2021, compared with $23.4 million for the same period in 2020, with the increase primarily due to higher sales of Captisol for use with remdesivir, a treatment for COVID-19. Contract revenue was $14.1 million for the third quarter of 2021, compared with $9.5 million for the same period in 2020, with the increase primarily due to the additional revenue from Pfenex, which was acquired in October 2020.

Cost of Captisol was $11.4 million for the third quarter of 2021, compared with $6.4 million for the same period in 2020, with the increase primarily due to higher sales of Captisol. Amortization of intangibles was $11.8 million for the third quarter of 2021, compared with $3.9 million for the same period in 2020, with the increase primarily due to amortization of contractual relationships and technologies gained through the Pfenex acquisition. Research and development expense was $16.9 million for the third quarter of 2021, compared with $12.9 million for the same period of 2020, with the increase primarily due to the addition of Pfenex expenses. General and administrative expense was $12.7 million for the third quarter of 2021, compared with $15.0 million for the same period in 2020, with the decrease primarily due to $4.9 million of acquisition and integration costs in the prior-year period.

Other operating income was $3.8 million for the third quarter of 2021, which represented a non-cash valuation adjustment related to eliminating the remaining Pfenex CVR liability. There was no other operating income for the same period in 2020.

Net income for the third quarter of 2021 was $13.7 million, or $0.80 per diluted share, compared with net loss of $(6.7) million, or $(0.42) per share, for the same period in 2020. Net income for the third quarter of 2021 included a $1.6 million net non-cash gain from the value of Ligand’s short-term investments, while net loss for the third quarter of 2020 included a $(11.7) million net non-cash loss from the value of Ligand’s short-term investments. Adjusted net income for the third quarter of 2021 was $27.1 million, or $1.58 per diluted share, compared with $17.5 million, or $1.04 per diluted share, for the same period in 2020. Please see the table below for a reconciliation of net income/(loss) to adjusted net income.

As of September 30, 2021, Ligand had cash, cash equivalents and short-term investments of $323.2 million.

Year-to-Date Financial Results

Total revenues for the nine months ended September 30, 2021 were $204.7 million, compared with $116.4 million for the same period in 2020. Royalties for the nine months ended September 30, 2021 were $31.4 million, compared with $22.8 million for the same period in 2020. Captisol sales were $128.9 million for the nine months ended September 30, 2021, compared with $69.0 million for the same period in 2020, with the increase primarily due to higher sales of Captisol for use with remdesivir. Contract revenue was $44.4 million for the nine months ended September 30, 2021, compared with $24.7 million for the same period in 2020, with the increase primarily due to the additional revenue from the acquisitions of Icagen in April 2020 and Pfenex in October 2020.

Cost of goods sold was $50.2 million for the nine months ended September 30, 2021, compared with $18.7 million for the same period in 2020, with the increase primarily attributable to higher sales of Captisol. Amortization of intangibles for the nine months ended September 30, 2021 was $35.4 million, compared with $11.3 million for the same period in 2020, with the increase primarily due to amortization of contractual relationships and technologies gained through the Icagen and Pfenex acquisitions. Research and development expense was $50.8 million for the nine months ended September 30, 2021, compared with $37.5 million for the same period of 2020, with the increase primarily due to additional expenses following the Icagen and Pfenex acquisitions. General and administrative expense was $39.7 million for the nine months ended September 30, 2021, compared with $34.4 million for the same period in 2020, with the increase primarily due to additional expenses following the Icagen and Pfenex acquisitions, partially offset by acquisition and integration costs in the prior-year period.

Other operating income was $37.6 million for the nine months ended September 30, 2021, which represented a non-cash valuation adjustment related to eliminating the Pfenex CVR liability. There was no other operating income for the same period in 2020.

Net income for the nine months ended September 30, 2021 was $62.6 million, or $3.64 per diluted share, compared with net loss of $(8.7) million, or $(0.54) per share, for the same period in 2020. Net income for the nine months ended September 30, 2021 included a $2.4 million net non-cash gain from the value of Ligand’s short-term investments, while net loss for the same period in 2020 included a net non-cash loss in the value of Ligand’s short-term investments of $(17.9) million. Adjusted net income for the nine months ended September 30, 2021 was $79.4 million, or $4.62 per diluted share, compared with $49.4 million, or $2.93 per diluted share, for the same period in 2020. Please see the table below for a reconciliation of net income/(loss) to adjusted net income.

2021 Financial Guidance

Ligand is reaffirming 2021 financial guidance. Ligand expects full-year 2021 total revenues to be between $265 million and $275 million, and adjusted earnings per diluted share to be between $5.80 and $6.05.

Third Quarter 2021 and Recent Business Highlights

OmniAb Platform Updates

OmniAb is Ligand’s industry-leading BI- (Biological Intelligence) powered multi-species antibody platform for the discovery of monospecific and bispecific therapeutic human antibodies. As of September 30, 2021, 19 different OmniAb-derived antibodies have been studied in approximately 84 active or completed clinical trials.

Gloria Biosciences received approval from China’s National Medical Products Administration (NMPA) for zimberelimab (GLS-010), an OmniAb-derived anti-PD-1 monoclonal antibody for the treatment of recurrent or refractory classical Hodgkin’s lymphoma. Zimberelimab is the first OmniAb-derived antibody to receive regulatory approval.

CStone Pharmaceuticals presented data at ESMO (Free ESMO Whitepaper) Congress 2021 from the GEMSTONE-301 trial, a registrational study of OmniAb-derived sugemalimab in the treatment of patients with stage III non-small cell lung cancer (NSCLC). The data for sugemalimab as a consolidation therapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS). Sugemalimab was well-tolerated with no new safety signals. CStone also presented updated data from the registrational study of sugemalimab in patients with stage IV NSCLC in an oral presentation at the IASLC 2021 World Conference on Lung Cancer. The final analysis confirmed the efficacy and safety demonstrated in the interim analysis, showing that sugemalimab plus chemotherapy was associated with a significant improvement of PFS as first-line treatment in patients with both squamous and non-squamous metastatic NSCLC. Additionally, the estimated 2-year overall survival rate was nearly 50%. New drug applications for sugemalimab in patients with metastatic stage IV NSCLC and in patients with locally advanced/unresectable stage III NSCLC have been accepted by China’s NMPA and are currently under review.

Aptevo Therapeutics announced positive Phase 1 data showing some patients with relapsed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) achieved a remission with APVO436 after failing 1-8 lines of prior therapies. Data was published in the peer-reviewed journal, Cancers, showing the risk of cytokine release syndrome is low for blood cancer patients treated with APVO436. APVO436 is an OmniAb-derived bispecific antibody targeting CD123 and CD3 for the treatment of hematological malignancies.

Harbour BioMed announced the initiation of a Phase 3 trial with batoclimab (HBM9161), its OmniAb-derived anti-FcRn monoclonal antibody, for the treatment of generalized myasthenia gravis (gMG). This study aims to assess the efficacy and safety of batoclimab in patients with gMG in China. Harbour BioMed also announced the start of a Phase 2 trial in China of batoclimab for the treatment of thyroid eye disease. Harbour BioMed licensed batoclimab from HanAll Biopharma and has the right to develop, manufacture and commercialize in Greater China (including Hong Kong, Macau and Taiwan).

OmniAb partnered with LandingAI to incorporate an industry leading LandingLens visual inspection software platform to strengthen the xPloration deep screening platform using AI and computer vision.

During the third quarter, Ligand entered into an OmniAb licensing agreement with Pierre Fabre.

Pelican Platform Updates

The Pelican Expression Technology is Ligand’s proprietary Pseudomonas fluorescens protein expression technology that has major collaborations with Jazz Pharmaceuticals, Merck, Serum Institute of India and Alvogen, each of which has potential to contribute meaningfully to Ligand’s royalty revenue.

Merck announced VAXNEUVANCE met key immunogenicity and safety endpoints in a Phase 3 pivotal trial evaluating use in infants. The FDA approved VAXNEUVANCE for adults 18 years of age and older in July and Merck has submitted a supplemental regulatory licensure application to the FDA for use in children. On October 20, the Center for Disease Control’s committee on immunization practices provisionally recommend vaccination either with a sequential regimen of VAXNEUVANCE followed by PNEUMOVAX23, or with a single dose of 20-valent pneumococcal conjugate vaccine for adults 65 years and older, and for adults ages 19 to 64 with certain underlying medical conditions or other disease risk factors.

Jazz Pharmaceuticals announced the National Comprehensive Cancer Network added Rylaze to its Clinical Practice Guidelines in Oncology as a treatment option for both pediatric and adult acute lymphoblastic leukemia patients with hypersensitivity to E. coli asparaginase products as a component of the multi-agent chemotherapeutic regimen.

Other

Travere Therapeutics announced positive topline interim results from the ongoing Phase 3 PROTECT study of sparsentan in IgA nephropathy. Sparsentan treatment demonstrated a statistically significant mean reduction of proteinuria from baseline after 36 weeks, more than threefold the reduction of active comparator irbesartan (p<0.0001). Travere met with the FDA for sparsentan in focal segmental glomerulosclerosis (FSGS) confirming plans to submit additional data in the first half of 2022 as part of an accelerated approval submission. Additionally, Travere and Vifor Pharma entered into a licensing agreement for the commercialization of sparsentan in Europe, Australia and New Zealand.

Ligand entered into a collaboration agreement with China Resources Double-Crane for exclusive Asia rights to develop a novel oral COVID-19 antiviral treatment using Ligand’s BEPro technology. BEPro is a proprietary prodrug technology for the development of compounds with improved product profiles. Ligand had generated preclinical pharmacokinetics data showing its oral BEPro-enabled COVID-19 antivirals have favorable blood concentration profiles and generated lower levels of active nucleotide in the kidney, a potential site for toxicity, compared with other oral and intravenous compounds.

Sermonix Pharmaceuticals announced completion of enrollment in the Phase 2 ELAINE 1 randomized trial assessing oral lasofoxifene versus intramuscular fulvestrant for the treatment of ER+/HER2- breast cancer in patients with an ESR1 mutation. Sermonix expects data from the trial to be reported in the first half of 2022. Lasofoxifene is also being studied in a separate fully-enrolled trial, ELAINE 2, in combination with Eli Lilly and Company’s CDK4 and 6 inhibitor Verzenio (abemaciclib). Topline data are also expected in the first half of 2022.

Icagen Ion Channel Technology’s Dr. Anil Nair presented at the 3rd Annual Drug Discovery & Development Summit and gave an oral presentation entitled "In Silico Drug Discovery: Application of Computer-Aided Drug Design in an Industrial Environment".

In July, Ligand announced the appointment of Jennifer Cochran, Ph.D. to the Company’s Board of Directors. Dr. Cochran is the Shriram Chair of the Department of Bioengineering at Stanford University, where she also is a professor of bioengineering and (by courtesy) of chemical engineering and a member of the cancer biology, biophysics and immunology programs and was a Founder and former CEO of xCella Biosciences.

Ligand provides regular updates on individual partner events through its Twitter account, @Ligand_LGND.

Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, other than with respect to total revenues, the Company only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and the effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 540-1167 from the U.S. or (929) 517-0358 from outside the U.S., using the conference ID 5486177. To participate via live or replay webcast, a link is available at www.ligand.com.

About OmniAb

The OmniAb discovery platform provides Ligand’s pharmaceutical industry partners access to the diverse antibody repertoires and high-throughput screening technologies to enable discovery of next-generation therapeutics. At the heart of the OmniAb platform is the Biological Intelligence of our proprietary transgenic animals, including OmniRat, OmniChicken and OmniMouse that have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. OmniFlic (transgenic rat) and OmniClic (transgenic chicken) address industry needs for bispecific antibody applications though a common light chain approach, and OmniTaur features unique structural attributes of cow antibodies for complex targets. OmniAb animals comprise the most diverse host systems available in the industry and they are optimally leveraged through computational antigen design and immunization methods, paired with high-throughput microfluidic-based single B cell screening and deep computational analysis of next-generation sequencing datasets to identify fully human antibodies with superior performance and developability characteristics. An established core competency focused on ion channels and transporters further differentiates our technology and creates opportunities to further leverage across modalities, including antibody-drug conjugates and others. The OmniAb suite of technologies and differentiating computational capabilities and BI features are combined to offer a highly efficient and customizable end-to-end solution for the growing discovery needs of the global pharmaceutical industry.

About the Pelican Expression Technology

Pelican is a robust, validated, cost-effective and scalable platform for recombinant protein production that is especially well-suited for complex, large-scale protein production where traditional systems are not. Multiple global manufacturers have demonstrated consistent success with the platform and the technology is currently out-licensed for numerous commercial and development-stage programs. The versatility of the platform has been demonstrated in the production of enzymes, peptides, antibody derivatives and engineered non-natural proteins. Partners seek the platform as it can contribute significant value to biopharmaceutical development programs by reducing development timelines and costs for manufacturing therapeutics and vaccines. Given pharmaceutical industry trends toward large molecules with increasing structural complexities, Pelican is well positioned to meet these growing needs as the most comprehensive broadly available protein production platform in the industry.

About Captisol

Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Captisol was invented and initially developed by scientists in the laboratories of Dr. Valentino Stella, University Distinguished Professor at the University of Kansas’ Higuchi Biosciences Center for specific use in drug development and formulation. This unique technology has enabled several FDA-approved products, including Gilead’s VEKLURY, Amgen’s KYPROLIS, Baxter International’s NEXTERONE, Acrotech Biopharma L.L.C.’s and CASI Pharmaceuticals’ EVOMELA, Melinta Therapeutics’ BAXDELA and Sage Therapeutics’ ZULRESSO. There are many Captisol-enabled products currently in various stages of development. Ligand maintains a broad global patent portfolio for Captisol with more than 400 issued patents worldwide relating to the technology (including over 40 in the U.S.) and with the latest expiration date in 2033. Other patent applications covering methods of making Captisol, if issued, extend to 2040.

On November 9, 2021 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Celldex Therapeutics, NOV 9, 2021, View Source [SID1234594896]).

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"During the third quarter we reported compelling data from our ongoing Phase 1b study of CDX-0159 in chronic inducible urticaria, including a rapid, profound and durable 95% complete response rate to provocation testing after just a single dose," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "Last month, we added to these positive results, reporting additional patient-reported outcome measures that demonstrated rapid and sustained improvement in urticaria disease control and improvements in quality of life. We were also pleased to recently initiate a Phase 1 study of the subcutaneous formulation of CDX-0159 and recently opened enrollment in the Phase 1b study in prurigo nodularis."

Mr. Marucci continued, "We continue to make significant progress across our clinical pipeline including our bispecific platform, which is exploring important pathways in inflammatory diseases, auto-immune disorders and oncology. We look forward to updating you on these programs over the coming months."

Recent Program Highlights

CDX-0159 – KIT Inhibitor Program

CDX-0159 is a humanized monoclonal antibody developed by Celldex that binds the KIT receptor with high specificity and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells, which mediate inflammatory responses such as hypersensitivity and allergic reactions. KIT signaling controls the differentiation, tissue recruitment, survival and activity of mast cells.

In July, Celldex reported interim data from the CDX-0159 single dose Phase 1b open label study in inducible urticaria, which were presented in a late-breaking poster discussion session as part of the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2021.

All 19 patients experienced a clinical response as assessed by provocation threshold testing; 18/19 (95%) experienced a complete response and 1/19 (5%) experienced a partial response.

Rapid onset of responses after dosing and sustained durability were observed and most patients with cold urticaria and symptomatic dermographism experienced a complete response by week 1 and by week 4, respectively. The median duration of response for patients was 77+ days (11+ weeks) for cold urticaria and 57+ days (8+ weeks) for symptomatic dermographism.

A single 3 mg/kg dose of CDX-0159 resulted in rapid, marked and durable suppression of serum tryptase and depletion of skin mast cells (87% depletion) as measured through biopsy. The kinetics of serum tryptase and skin mast cell depletion mirrored clinical activity which confirmed that serum tryptase level is a robust pharmacodynamic biomarker for assessing mast cell burden and clinical activity in inducible urticaria and potentially in other diseases with mast cell driven involvement.

CDX-0159 was generally well tolerated. The most common adverse events were hair color changes, mild infusion reactions, and transient changes in taste perception.

In September, Celldex reported symptom control & quality of life measurements data from the CDX-0159 single dose Phase 1b open label study in inducible urticaria, which were presented in an e-poster session as part of the European Academy of Dermatology and Venereology (EADV) 2021 Virtual 30th Congress.

A single 3 mg/kg dose of CDX-0159 resulted in a rapid and sustained improvement in urticaria control and greatly reduced disease impact on quality of life, as measured by the Urticaria Control Test (UCT) and Dermatology Life Quality Index (DLQI). These data support and build on the previously reported 95% complete response rate to provocation testing.

Additional Phase 1b single dose data from the cholinergic cohort of this study are planned to be submitted for presentation at EAACI 2022.

Celldex continues to enroll patients in the Phase 1b multi-center, randomized, double-blind, placebo-controlled study of CDX-0159 in chronic spontaneous urticaria (CSU). This study is designed to assess the safety and treatment effects of multiple ascending doses of CDX-0159 in up to 40 patients with CSU who remain symptomatic despite treatment with antihistamines. Treatment results from this study are planned to be submitted for presentation at EAACI 2022.

In September, Celldex initiated and has since completed dosing in a randomized, double-blind, placebo-controlled, Phase 1 study designed to evaluate the safety of single ascending doses of the subcutaneous formulation of CDX-0159 in healthy volunteers. Celldex intends to utilize the subcutaneous formulation in its Phase 2 program in chronic urticarias.

In September, enrollment opened in the Phase 1b multi-center, randomized, double-blind, placebo-controlled study of CDX-0159 in patients with prurigo nodularis (PN), a chronic skin disease characterized by the development of hard, intensely itchy (pruritic) nodules on the skin. This study is designed to assess the safety and treatment effects across multiple dosing cohorts of CDX-0159 in up to 40 patients with PN.
CDX-1140 – CD40 Agonist Program

CDX-1140 is a potent CD40 human agonist antibody developed by Celldex that the Company believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 study of CDX-1140 in up to ~260 patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas, the monotherapy cohort, the combination cohort with CDX-301 and the safety run-in combination cohort with gemcitabine/nab-paclitaxel have been completed. Expansion cohorts including CDX-1140 with KEYTRUDA (pembrolizumab) in patients with squamous cell head and neck cancer and non-small cell lung cancer who have progressed on checkpoint therapy are ongoing.

The combination of CDX-1140 with pembrolizumab has completed the safety run-in phase. Expansion cohorts in patients with checkpoint-refractory/resistant squamous cell head and neck cancer and non-small cell lung cancer are enrolling patients. Of the six patients with squamous cell head and neck cancer treated with CDX-1140 at 1.5 mg/kg in combination with pembrolizumab, encouraging preliminary results have been observed including a confirmed partial response and durable stable disease. Of the six evaluable patients with non-small cell lung cancer, four have had stable disease as their best response. Adverse events, such as arthralgia, myalgia, and fatigue, have occurred more frequently in combination with pembrolizumab relative to CDX-1140 monotherapy and the protocol has been amended to allow CDX-1140 dose reduction, if necessary, to help manage these toxicities. Enrollment to the study is ongoing.

Emerging data from the safety run-in cohort of CDX-1140 with gemcitabine/nab-paclitaxel in patients with previously untreated metastatic pancreatic adenocarcinoma and external CD40 agonist data recently reported using the same regimen, suggest that simultaneous treatment with chemotherapy and CD40 activation may not be optimal. Alternative strategies for investigating CDX-1140 in pancreatic cancer in other regimens are being explored, including through investigator sponsored studies.
CDX-527 – Bispecific Antibody Program

CDX-527 is the first candidate developed by Celldex from its bispecific platform which utilizes the Company’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway.

In June, Celldex reported initial data from the Phase 1 dose-escalation study in up to ~40 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy to be followed by tumor-specific expansion cohorts, which were presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting. A good safety profile was observed along with promising pharmacodynamic and pharmacokinetic activity, which are important key hurdles for the development of bispecific antibodies. The study is designed to determine the MTD during a dose-escalation phase and to recommend a dose level for further study in the subsequent expansion phase. The expansion is designed to further evaluate the tolerability, and biologic and anti-tumor effects of selected dose level(s) of CDX-527 in specific tumor types. Enrollment to the dose escalation portion of the study has been completed and an expansion cohort in ovarian cancer is currently enrolling patients.
Recent Operational Highlights

In July, Celldex closed an underwritten public offering of common stock, including the full exercise of the underwriters’ option to purchase additional shares, for gross proceeds of $287.5 million. Celldex believes that the proceeds from this offering, together with current reserves, provide the cash runway to fund key clinical, regulatory and operational activities through 2025.
In September, Marc Rothenberg, MD, PhD was appointed to the Celldex Scientific Advisory Board. Dr. Rothenberg is currently Director of the Allergy and Immunology Division and Director of the Cincinnati Center for Eosinophilic Disorders at Cincinnati Children’s Hospital Medical Center. His clinical and research interests have focused on developing innovative therapies for allergic inflammatory diseases, with a focus on eosinophilic gastrointestinal disorders (EGIDs).
To date, the Company has managed delays and disruptions related to the COVID-19 pandemic without significant impact in planned and ongoing preclinical and clinical trials, manufacturing or shipping. The Company continues to carefully monitor the evolving situation closely across all development programs and work to minimize potential impact/disruptions.

Third Quarter 2021 Financial Highlights and 2021 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2021 were $423.1 million compared to $164.0 million as of June 30, 2021. The increase was primarily driven by net proceeds of $269.9 million from our July 2021 underwritten public offering, partially offset by third quarter cash used in operating activities of $16.4 million. At September 30, 2021, Celldex had 46.7 million shares outstanding.

Revenues: Total revenue was $0.2 million in the third quarter of 2021 and $4.3 million for the nine months ended September 30, 2021, compared to $0.7 million and $3.6 million for the comparable periods in 2020. The increase in revenue for the nine months ended September 30, 2021 compared to the nine months ended September 30, 2020 was primarily due to an increase in services performed under our contract manufacturing and research and development agreements with Rockefeller University and Gilead Sciences, partially offset by a decrease in revenue from product development and licensing agreements as a result of the $1.8 million milestone payment received from Rockefeller University in the first quarter of 2020 related to Celldex’s manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $13.6 million in the third quarter of 2021 and $38.6 million for the nine months ended September 30, 2021, compared to $10.7 million and $32.1 million for the comparable periods in 2020. The increase in R&D expenses was primarily due to an increase in clinical trial, contract research, and personnel expenses.

G&A Expenses: General and administrative (G&A) expenses were $5.8 million in the third quarter of 2021 and $14.2 million for the nine months ended September 30, 2021, compared to $3.6 million and $10.8 million for the comparable periods in 2020. The increase in G&A expenses was primarily due to higher personnel and legal expenses.

Intangible Asset Impairment: The Company recorded a non-cash impairment charge of $3.5 million related to the TAM program IPR&D asset in the third quarter of 2021 as a result of a lack of interest in the program from third parties. The Company recorded a non-cash impairment charge of $3.5 million during the second quarter of 2020 due to the discontinuation of the CDX-3379 program.

Changes in Fair Value Remeasurement of Contingent Consideration: The gain on fair value remeasurement of contingent consideration was $1.9 million for the third quarter of 2021 and $1.2 million for the nine months ended September 30, 2021, primarily due to updated assumptions for the TAM program, changes in discount rates and the passage of time.

Net Loss: Net loss was $20.5 million, or ($0.45) per share, for the third quarter of 2021, and $50.4 million, or ($1.21) per share, for the nine months ended September 30, 2021, compared to a net loss of $14.2 million, or ($0.36) per share, for the third quarter of 2020 and $37.9 million, or ($1.44) per share, for the nine months ended September 30, 2020.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at September 30, 2021 are sufficient to meet estimated working capital requirements and fund planned operations through 2025.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA.

On November 9, 2021 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that members of the management team will participate at the following investor conferences (Press release, Neurocrine Biosciences, NOV 9, 2021, View Source [SID1234594895]):

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Kevin Gorman, Chief Executive Officer, and Matt Abernethy, Chief Financial Officer will present at the Jefferies London Healthcare Conference at 1:00 p.m. Greenwich Mean Time (8:00 a.m. Eastern Time) on Tuesday, Nov. 16, 2021 in London.
Matt Abernethy, Chief Financial Officer, and Eiry Roberts, Chief Medical Officer, will present at the Evercore ISI 4th Annual HealthCONx Virtual Conference at 10:55 a.m. Eastern Time on Tuesday, Nov. 30, 2021.
The live presentations will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentations will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.