On February 1, 2021 Coherus BioSciences, Inc. ("Coherus", Nasdaq: CHRS) reported a collaboration with Shanghai Junshi Biosciences, Co., Ltd ("Junshi Biosciences", HKEX: 1877; SSE: 688180) for the development and commercialization of toripalimab, Junshi Biosciences’ anti-PD-1 antibody, in the United States and Canada (Press release, Coherus Biosciences, FEB 1, 2021, View Source [SID1234574456]). Upon satisfaction of closing conditions, Coherus and Junshi Biosciences will co-develop toripalimab, and Coherus will be responsible for all commercial activities in the licensed territory. Under the terms of the agreement, Coherus will also be granted options to Junshi Biosciences’ TIGIT-targeted antibody and next generation engineered IL-2 cytokine for evaluation as potential combination therapies with toripalimab, as well as certain negotiation rights to two early-stage checkpoint inhibitor antibodies.

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"Toripalimab has a compelling late-stage clinical profile and will be the cornerstone of our immuno-oncology franchise. This transaction expands our late-stage pipeline into the rapidly growing checkpoint inhibitor market, which is expected to exceed $25 billion in the United States by 2025, and provides us a PD-1 backbone for potential long-term growth with next-generation immuno-oncology combinations," said Denny Lanfear, CEO of Coherus. "Junshi Biosciences is an innovation-driven biopharmaceutical company with global clinical research capabilities, and we are excited to collaborate with them as we build on our success in oncology with biosimilars and broaden our mission of expanding patient access and delivering health care system savings to larger markets."

"We believe Coherus is the right partner for us in North America. Their commercial team has demonstrated remarkable ability to gain significant share of the oncology market against entrenched large competitors," said Dr. Ning LI, CEO of Junshi Biosciences. "Toripalimab could be the first marketed Chinese anti-PD-1 antibody in the overseas market. The collaboration with Coherus will be a critical step to build up our global commercial network. We look forward to working closely with Coherus to establish toripalimab’s position in the United States and Canadian markets in order to provide patients with affordable high-quality innovative care."

More than 2,100 patients have received toripalimab treatment in clinical trials, and toripalimab is approved for second-line treatment of unresectable or metastatic melanoma in China where it is marketed by Junshi Biosciences and is included on the National Reimbursement Drug List ("NRDL") for the treatment of melanoma. Over the next three years, significant data are expected to read out from the extensive registrational development program, which includes 15 ongoing and completed pivotal clinical trials evaluating toripalimab in multiple treatment settings for a broad range of solid tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

The United States Food and Drug Administration ("FDA") has granted breakthrough therapy designation to toripalimab for 3rd line nasopharyngeal carcinoma ("NPC"), and Coherus expects the first toripalimab biologics license application ("BLA") to be filed with the FDA for this indication later this year. Additionally, FDA has granted Fast Track status for the development of toripalimab for the treatment of mucosal melanoma, and orphan drug designation for NPC, mucosal melanoma and soft tissue sarcoma. Coherus and Junshi Biosciences plan to file additional toripalimab BLAs with the FDA over the next three years for multiple rare and highly prevalent cancers, including non-small cell lung cancer ("NSCLC").

As part of the collaboration, Coherus will also be granted options and certain negotiation rights to four of Junshi Biosciences’ novel oncology molecules for potential development in combination with toripalimab:

JS006, an antibody targeting TIGIT, a clinically validated immune inhibitory checkpoint. Anti-TIGIT antibodies have demonstrated synergistic anti-tumor activity in combination with anti-PD-1 antibodies. Coherus expects this compound to enter clinical development later this year. The option to JS006 is exercisable prior to initiation of Phase 2 clinical development.
JS018, a next-generation engineered IL-2 cytokine designed to inhibit stimulation of regulatory T cells while retaining stimulatory activity on effector T cells and natural killer ("NK") cells. The option to JS018 is exercisable prior to initiation of Phase 2 clinical development.
Two additional undisclosed early-stage novel immuno-oncology drug candidates to which Coherus will also be granted certain negotiation rights.
Mr. Lanfear provided further comment on Coherus’ corporate strategy: "We plan to invest the cash generated by our biosimilar commercial business to build a focused immuno-oncology franchise, which will leverage our development and commercial capabilities into large and growing markets. We will prudently allocate our R&D resources to realize the exciting potential of toripalimab in both the monotherapy and combination settings. With respect to biosimilars, our focus will be commercialization, as we continue to pursue UDENYCA market share growth and prepare for projected launches through 2023 of biosimilars of Humira, Avastin and Lucentis, if approved."

Coherus is discontinuing the development of CHS-2020 (Eylea biosimilar candidate) and will direct those capital and development resources to the toripalimab monotherapy and combinations program.

Terms of the Coherus – Junshi Biosciences collaboration
Under the terms of the agreement, Coherus will pay $150 million upfront for exclusive rights to toripalimab in the United States and Canada, options in these territories to Junshi Biosciences’ anti-TIGIT antibody and next-generation engineered IL-2 cytokine, and certain negotiation rights to two undisclosed preclinical immuno-oncology drug candidates. Coherus will also pay Junshi Biosciences a 20% royalty on net sales of toripalimab and up to an aggregate $380 million in one-time payments for the achievement of various milestones, including up to $290 million for attainment of certain sales thresholds. The option exercise fee for each of the anti-TIGIT antibody and the IL-2 cytokine is $35 million per program. Additionally, for each option program, Coherus will pay Junshi Biosciences an 18% royalty on net sales and up to an aggregate $255 million for the achievement of various milestones, including up to $170 million for attainment of certain sales thresholds. The Companies will collaborate in the development of toripalimab and the other licensed compounds, and Coherus will pay for a portion of these co-development activities up to a maximum of $25 million per licensed compound per year.

Closing of the collaboration agreement is subject to clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Jonathan Lanfear of Lanfear Advisors LLC led negotiations on behalf of Coherus, and Ropes & Gray LLP provided licensee’s legal counsel.

Conference call at 8:00 am Eastern Time
Webcast: The conference call will be broadcast live in listen-only mode on the Company’s investor relations website at View Source If you would like to ask a question, the dial in number for the conference call is (844) 452-6826 (Toll Free U.S. and Canada) or (765) 507-2587 (International).
Conference ID: 7784059
Please dial-in 15 minutes early to ensure a timely connection to the call.

A link to the conference call replay will be available on the Coherus website for two weeks following the call.

About toripalimab (approved in China as TUOYI)
Toripalimab is the first Chinese domestic anti-PD-1 monoclonal antibody to obtain a marketing approval in China. More than thirty company-sponsored toripalimab clinical studies covering more than 15 indications have been conducted globally, including in China and the United States. In December 2018, toripalimab obtained a conditional approval from China’s National Medical Products Administration ("NMPA") for the second-line treatment of patients with unresectable or metastatic melanoma. Supplemental NDAs of toripalimab for the third-line treatment of recurrent/metastatic nasopharyngeal carcinoma and second-line treatment of metastatic urothelial carcinoma were accepted by the NMPA in April and May 2020, respectively. Both supplemental NDAs received priority review designations from the NMPA in July 2020. In December 2020, toripalimab was included in the NRDL for the treatment of melanoma by the China National Healthcare Security Administration (NHSA).

In the United States, the FDA has granted toripalimab breakthrough therapy designation for the treatment of recurrent/metastatic nasopharyngeal carcinoma, Fast Track designation for mucosal melanoma, and orphan drug designation for the treatment of nasopharyngeal carcinoma, mucosal melanoma and soft tissue sarcoma.

About the toripalimab clinical development program
Toripalimab is being evaluated in an extensive registrational clinical development program for a broad range of solid tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

Safety and Efficacy of Recombinant Humanized Anti-PD-1 mAb for Patients With Locally Advanced or Metastatic Melanoma (POLARIS-01) – NCT03013101
Phase 1b/2 trial Evaluating Toripalimab in Patients with Advanced Gastric Adenocarcinoma, ESCC, NPC and Head and Neck Squamous Cell Carcinoma (POLARIS-02) – NCT02915432
Safety and Efficacy of Toripalimab for Patients With Locally Advanced or Metastatic Bladder Urothelial Carcinoma (POLARIS-03) – NCT03113266
A Study to Evaluate the Efficacy and Safety of Toripalimab Injection in the Treatment of Recurrent or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma Who Have Failed at Least Two Prior Lines of Therapy and Are Positive for Specific Markers – NCT04603040
Phase III Study of Comparing TORIPALIMAB INJECTION Versus Placebo Combined With Chemotherapy for Recurrent or Metastatic Nasopharyngeal Cancer – NCT03581786
Toripalimab or Placebo as Adjuvant Therapy in Hepatocellular Carcinoma After Curative Hepatic Resection (JUPITER-04) – NCT03859128
A Phase III Study to Investigate Toripalimab Versus Dacarbazine as the First Line Therapy for Unresectable or Metastatic Melanoma (JS001) – NCT03430297
A Study to Evaluate the Efficacy and Safety of Toripalimab or Placebo Combined With Chemotherapy in Treatment-naive Advanced NSCLC (CHOICE-01) – NCT03856411
Toripalimab or Placebo With Paclitaxel and Cisplatin in Esophageal Squamous Cell Carcinoma (JUPITER-06) – NCT03829969
Toripalimab Plus Pemetrexed+Platinus in Advanced Non-small-cell Lung cancer Patients Previously Treated with EGFR-TKI – NCT03924050
Toripalimab in Combination With Nab-Paclitaxel For Patients With Metastatic or Recurrent Triple-Negative Breast Cancer (TNBC) With or Without Systemic Treatment (TORCHLIGHT) – NCT04085276
Phase 3 Trial Comparing Toripalimab + Lenvatinib vs. Lenvatinib Alone as a 1st line Treatment for Patients with Advanced HCC – NCT04523493
Toripalimab in Combination With Platinum Plus Etoposide in Patients With Extensive-Stage Small Cell Lung Cancer – NCT04012606
A Study of Toripalimab or Placebo Plus Chemotherapy as Treatment in Early Stage NSCLC – NCT04158440
Study to Evaluate the Efficacy and Safety of Toripalimab in Combination With Axitinib Versus Sunitinib Monotherapy in Advanced Renal Cell Cancer – NCT04394975
A Study Evaluating Toripalimab Injection Combined With Standard Chemotherapy as a First-line Treatment for Locally Advanced or Metastatic Urothelial Carcinoma – NCT04568304
About JS006
JS006 is a recombinant humanized IgG4κ monoclonal antibody against human TIGIT specifically, developed independently by Junshi Biosciences. According to the results of preclinical studies, JS006 can specifically block the TIGIT-PVR pathway.

About JS018
JS018 is a next-generation engineered IL-2 cytokine designed to inhibit stimulation of regulatory T cells while retaining stimulatory activity on effector T cells and natural killer ("NK") cells.

On February 1, 2021, Black Diamond Therapeutics, Inc. (the "Company") reported that it entered into an Open Market Sale AgreementSM (the "Sales Agreement") with Jefferies LLC ("Jefferies"), pursuant to which the Company may issue and sell shares of its common stock, $0.0001 par value per share (the "Common Stock"), having an aggregate offering price of up to $150,000,000 (the "Shares") from time to time through Jefferies as its sales agent (Filing, 8-K, Black Diamond Therapeutics, FEB 1, 2021, View Source [SID1234574455]). The Shares to be sold under the Sales Agreement, if any, will be offered and sold pursuant to the Company’s automatic shelf registration statement on Form S-3ASR and related prospectus, filed with the Securities and Exchange Commission on February 1, 2021, which became effective upon filing (the "Registration Statement").

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Upon delivery of a placement notice and subject to the terms and conditions of the Sales Agreement, Jefferies may sell the Shares by any method permitted by law deemed to be an "at-the-market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended. The Company may sell the Shares in amounts and at times to be determined by the Company from time to time subject to the terms and conditions of the Sales Agreement, but it has no obligation to sell any of the Shares under the Sales Agreement.

The Company or Jefferies may suspend or terminate the offering of Shares upon notice to the other party and subject to other conditions. Jefferies will act as sales agent on a commercially reasonable efforts basis consistent with its normal trading and sales practices and applicable state and federal law, rules and regulations and the rules of Nasdaq.

The Company will pay Jefferies a cash commission equal to three percent (3.0%) of gross proceeds from the sale of the Shares pursuant to the Sales Agreement. The Company has also agreed to provide Jefferies with customary indemnification and contribution rights.

The offering of Shares pursuant to the Sales Agreement will terminate upon the earlier of (i) the sale of all Shares subject to the Agreement or (ii) termination of the Sales Agreement in accordance with its terms.

The foregoing description of the Sales Agreement is not complete and is qualified in its entirety by reference to the full text of the Sales Agreement, a copy of which was filed as Exhibit 1.1 to the Registration Statement.

This Current Report on Form 8-K shall not constitute an offer to sell or solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of such state or jurisdiction.

On February 1, 2021 Cancer Genetics, Inc. (the "Company") (Nasdaq: CGIX), a leader in drug discovery and preclinical oncology and immuno-oncology services, reported the closing of its previously announced private placement of approximately $10.0 million through the issuance of an aggregate 2,758,624 shares of its common stock and warrants to purchase up to an aggregate 2,758,624 shares of common stock, at a combined purchase price of $3.625 per share of common stock and associated warrant in a private placement priced at-the-market under Nasdaq rules (Press release, Cancer Genetics, FEB 1, 2021, View Source [SID1234574454]).

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The warrants have an exercise price of $3.50 per share, are exercisable immediately and have a term of five and one-half years.

The Company currently intends to use the net proceeds from the offering for general corporate purposes, including working capital and capital expenditures. The net proceeds are also expected to be available to the combined company once the previously announced merger with StemoniX closes, which is subject to stockholder approval.

The securities described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act") and Regulation D promulgated thereunder and in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

Under an agreement with the investors, the Company is required to file an initial registration statement with the Securities and Exchange Commission covering the resale of the shares of common stock to be issued to the investors by 9:30 a.m., Eastern Time, on February 2, 2021 and to use its best efforts to have the registration statement declared effective as promptly as practical thereafter, and in any event no later than 90 days after today in the event of a "full review" by the Securities and Exchange Commission.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state.

On February 1, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance (Press release, Vertex Pharmaceuticals, FEB 1, 2021, View Source [SID1234574453]).

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"Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including small molecules for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases, gene editing for sickle cell disease and beta thalassemia, and cell therapy for type 1 diabetes," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "As we enter 2021, we look forward to treating more CF patients and reaching important R&D milestones in multiple additional diseases which will fuel our growth this year and for many years to come."

GAAP and Non-GAAP product revenues increased 49% and 55%, respectively, compared to 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements. Net product revenues in 2020 were $4.8 billion in the U.S. and $1.4 billion outside the U.S.

GAAP and Non-GAAP net income increased compared to 2019, largely driven by strong growth in product revenues.

Cash, cash equivalents and marketable securities as of December 31, 2020 were $6.7 billion, an increase of approximately $2.9 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other disease areas.

GAAP and Non-GAAP income taxes increased compared to 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

GAAP and Non-GAAP product revenues increased 15% and 29%, respectively, compared to the fourth quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements.

GAAP and non-GAAP net income increased compared to the fourth quarter of 2019, largely driven by strong growth in total product revenues.

Combined GAAP R&D and SG&A expenses were similar to the fourth quarter of 2019.

Combined Non-GAAP R&D and SG&A expenses increased compared to the fourth quarter of 2019, primarily due to the incremental investment to support the global use of Vertex’s medicines and the expansion of Vertex’s pipeline in CF and other disease areas.

GAAP and Non-GAAP income taxes increased compared to the fourth quarter of 2019 primarily due to Vertex’s increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex’s provision for income taxes.

Full-Year 2021 Financial Guidance

Vertex today provided its full-year 2021 financial guidance. Product revenue guidance is primarily based on:

The continued strong performance of TRIKAFTA in the U.S. and KAFTRIO in certain European countries
The launch of medicines in the U.S. for rare mutations following approval in December 2020 and the approval of TRIKAFTA for children with CF ages 6-11 in the U.S. expected mid-year
Countries where patients currently have access or reimbursement
Key Business Highlights

Cystic Fibrosis (CF) R&D pipeline

Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in increasing the number of eligible patients and expanding approval and access for our medicines to additional geographies and age groups.

TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)

The U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA to include people with CF ages 12 and older with certain mutations that are responsive to TRIKAFTA based on in vitro data. SYMDEKO and KALYDECO also received approvals to include additional responsive mutations in people with CF ages 6 and older and ages 4 months and older, respectively.
Swissmedic, the Swiss Agency for Therapeutic Products, granted marketing authorization and a reimbursement agreement was reached for TRIKAFTA in Switzerland in people with CF ages 12 and older with two F508del mutations or one F508del mutation and one minimal function mutation.
The U.S. FDA accepted a supplemental New Drug Application (sNDA) for TRIKAFTA for the treatment of children with CF ages 6 to 11 who have at least one F508del mutation or have certain mutations that are responsive to TRIKAFTA based on in vitro data. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021.
Health Canada accepted a New Drug Submission for Priority Review for TRIKAFTA for the treatment of people with CF ages 12 years and older.
SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)

The European Commission (EC) granted approval of the label extension for SYMKEVI to include people with CF ages 6 years and older with two copies of the F508del mutation or one copy of the F508del mutation and certain residual function mutations.
KALYDECO (ivacaftor)

The EC granted approval of the label extension for KALYDECO to include the treatment of infants with CF ages 4 months and older who have R117H or certain gating mutations.
R&D pipeline outside of CF

Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Beta Thalassemia and Sickle Cell Disease

Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease.
Enrollment and dosing are ongoing in the clinical studies for CTX001. More than 20 patients have been dosed with CTX001 across both studies to date. Completion of enrollment in both studies is expected in 2021.
Alpha-1 Antitrypsin (AAT) Deficiency

Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.
APOL1-mediated Kidney Diseases

Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.
Type 1 Diabetes (T1D)

Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
The U.S. FDA cleared the Investigational New Drug Application (IND) for VX-880, the islet cells alone program. Vertex expects to initiate a Phase 1/2 clinical trial in the first half of 2021.
Investments in External Innovation

Skyhawk Therapeutics and Vertex established a strategic collaboration to discover and develop novel small molecules that modulate RNA splicing for the treatment of serious diseases.
Non-GAAP Financial Measures

In this press release, Vertex’s financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex’s pre-tax income (i) stock-based compensation expense, (ii) an adjustment to product revenues and related cost of sales to reflect the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019, (iii) revenues and expenses related to collaboration agreements, (iv) gains or losses related to the fair value of the company’s strategic investments, (v) increases or decreases in the fair value of contingent consideration, (vi) acquisition-related costs and (vii) other adjustments. The company’s non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company’s GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company’s business, are important in comparing current results with prior period results and provide additional information regarding the company’s financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company’s business and to evaluate its performance. The company adjusts, where appropriate, for both revenues and expenses in order to reflect the company’s operations. The company’s calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities. The company does not provide a GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

Notes and Explanations

1: "ORKAMBI adjustment" in the company’s Reconciliation of GAAP to Non-GAAP Net Income in the three and twelve months ended December 31, 2019 included an adjustment to net product revenues and cost of sales related to the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019. The company had previously recognized a portion of net product revenues related to ORKAMBI distributed through the early access program in France. As a result, the company recognized an adjustment to increase net product revenues by $155.8 million and cost of sales by $14.9 million, which related to prior period shipments of ORKAMBI distributed through the early access program in France. The company excluded the adjustment to net product revenues and cost of sales from its Non-GAAP measures for the three and twelve months ended December 31, 2019.

2: In the three and twelve months ended December 31, 2020 and 2019, "Tax adjustments" primarily related to the estimated income taxes related to non-GAAP adjustments to pre-tax income including (i) stock-based compensation (including an adjustment for excess tax benefits related to stock-based compensation), (ii) increases or decreases in the fair value of the company’s strategic investments and (iii) collaborative payments. In the twelve months ended December 31, 2020, "Tax adjustments" also included non-recurring discrete benefits to the company’s provision for income taxes, such as the transfer of intellectual property rights to the company’s U.K. entity, of approximately $287.6 million that the company excluded from its Non-GAAP measures.

3: The difference between the company’s full-year 2021 combined GAAP R&D and SG&A expenses and combined non-GAAP R&D and SG&A expenses guidance relates primarily to $430 million to $500 million of stock-based compensation expense and $200 million to $250 million of R&D expenses related to existing collaboration agreements. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities.

4: "Other income, net" includes gains related to changes in the fair value of the company’s strategic investments and from sales of certain investments.

5: During the three and twelve months ended December 31, 2020 and 2019, the increase in the fair value of contingent consideration relates to potential payments to Exonics Therapeutics’ former equity holders.

6: "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2020 and 2019 primarily related to collaborative upfront and milestone payments.

7: "Acquisition-related costs" in the three and twelve months ended December 31, 2020 and 2019 related to costs associated with the company’s acquisitions of Semma Therapeutics and Exonics.

8: The company records a provision for income taxes on its pre-tax income using an effective tax rate approximating statutory rates. Since the company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of December 31, 2018, its tax provision has included a significant non-cash charge due to the company’s ability to offset its pre-tax income against previously benefited net operating losses and credits. As of December 31, 2019, the company had federal net operating losses and credits that were available to offset future pre-tax income. The company utilized substantially all of its remaining federal net operating losses in 2020. As a result, a larger portion of the company’s tax provision will represent a cash expense in future periods, subject to continued utilization of certain tax credits.

On February 1, 2021 Corcept Therapeutics Incorporated (NASDAQ: CORT) reported it will report its preliminary 2020 fourth quarter and full-year financial results and provide a corporate update on February 8, 2021 (Press release, Corcept Therapeutics, FEB 1, 2021, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announce-preliminary-selected-financial [SID1234574452]). The company will host a conference call that day at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time).

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Conference Call Information

To participate, dial 1-833-693-0540 (United States) or 1-661-407-1581 (internationally) approximately 15 minutes before the start of the call. The conference ID number is 9289307.

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