On November 9, 2021 Mersana Therapeutics, Inc. (NASDAQ:MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Mersana Therapeutics, NOV 9, 2021, View Source [SID1234595039]).

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"We have made significant progress towards our vision of building UpRi into a foundational medicine in ovarian cancer and advancing a diverse pipeline addressing areas of high unmet medical need. Over the course of next year, we expect UPLIFT to be fully enrolled, UP-NEXT to be initiated and enrolling, UPGRADE to be charting the path in combinations and to have multiple assets in the clinic addressing a diverse set of targets," said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. "Our promising pipeline assets are generated by our innovative platforms Dolaflexin, Dolasynthen and Immunosynthen, which act as efficient product engines creating value now and into the future."

Recent Highlights and Anticipated Milestones

Upifitamab Rilsodotin (UpRi), first-in-class Dolaflexin ADC targeting NaPi2b:

Reported updated interim data in almost 100 patients from the UpRi ovarian cancer expansion cohort. Data continued to demonstrate a clinically meaningful and consistent profile for UpRi with a confirmed objective response rate (ORR) of 34% in the two-thirds of patients with NaPi2b high ovarian cancer. Data continued to show a differentiated tolerability profile for UpRi without the severe neutropenia, neuropathy and ocular toxicities seen with other ADC platforms. These data support the potential for UpRi to demonstrate a clinically meaningful benefit in the ongoing registration-enabling UPLIFT study in platinum-resistant ovarian cancer, a setting in which the single-agent chemotherapy standard of care has an ORR of no more than 12% and carries substantial toxicities.

Introduced UP-NEXT, a Phase 3 study of UpRi monotherapy maintenance, informed by FDA feedback. UP-NEXT is designed to establish UpRi as a maintenance agent following treatment with platinum doublets in platinum-sensitive ovarian cancer. Watch-and-wait remains the standard of care for patients who have previously received or are poorly served by existing maintenance agents, as well as patients who achieve only stable disease to platinum doublets. UP-NEXT is designed to provide data to address these unmet needs. The Company plans to provide more details on the final design of UP-NEXT and expects to initiate the study in 2022.

UPLIFT, a single-arm registration study in platinum-resistant ovarian cancer, continues to enroll. UPLIFT is enrolling a broader population of patients with platinum-resistant ovarian cancer than other studies in this indication through more flexible inclusion criteria with respect to lines of therapy and underlying comorbidities, the use of a robust diagnostic assay capturing two-thirds of the patients as NaPi2b high for the primary endpoint, as well as a secondary endpoint in the overall population. The Company plans to enroll approximately 100 patients with high NaPi2b expression and up to 180 patients overall and expects the study to be substantially enrolled during the summer of 2022.

Enrollment continues in UPGRADE, the Company’s Phase 1 combination umbrella study. UPGRADE is a Phase 1 dose-escalation study evaluating the combination of carboplatin with UpRi followed by UpRi continued until progression. The dose escalation portion of the study is intended to determine the recommended Phase 2 dose in combination with carboplatin. The expansion portion of the study is intended to provide proof of concept for a potential new standard of care for platinum-sensitive ovarian cancer earlier in the disease by demonstrating that the combination of platinum and UpRi followed by UpRi continuation could result in improved efficacy and tolerability with the ultimate goal of improved clinical benefit for patients. The study will inform further development of UpRi in this broader and earlier line patient population.

Completed enrollment and evaluation of UpRi in the NSCLC adenocarcinoma expansion cohort. The Company observed modest single-agent activity that did not meet its internal threshold for advancement and has decided to deprioritize further clinical evaluation of UpRi in this indication. The safety profile of UpRi in lung adenocarcinoma was generally consistent with the favorable profile observed with UpRi at the 36 mg/m2 dose level selected for further advancement in ovarian cancer.
XMT-1592, first Dolasynthen ADC targeting NaPi2b:

Phase 1 dose escalation study of XMT-1592 is ongoing. The Company continues dose exploration in order to determine the recommended Phase 2 dose and expects this to continue into 2022. The Company plans to provide further details of the XMT-1592 development plan alongside its 2022 milestones and goals early next year.
XMT-1660, first-in-class Dolasynthen ADC targeting B7-H4:

Investigational New Drug (IND)-enabling studies of XMT-1660 ongoing with Phase 1 studies expected to start in early 2022. B7-H4 is expressed in high unmet need tumors such as breast, endometrial and ovarian. B7-H4 is expressed on both tumor cells and immunosuppressive tumor-associated macrophages (TAMs). This provides the potential for both a direct, cytotoxic antitumor effect as well as for additional payload delivery to the tumor microenvironment that could further contribute to immunogenic cell death, dendritic cell activation, and stimulation of an immune response consistent with the features of the Company’s unique DolaLock payload.
XMT-2056, first-in-class HER2-targeted Immunosynthen STING-agonist ADC:

Presented new preclinical data for XMT-2056 at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) (Triple Meeting) in October 2021. In vitro and in vivo studies demonstrate that Immunosynthen STING-agonist ADCs activate the STING pathway in both tumor-resident immune cells and tumor cells, offering the potential for an increased therapeutic index and an advantage over other innate immune activating pathways. The Company developed XMT-2056 based on a differentiated anti-HER2 antibody that binds a novel epitope, providing the opportunity for combinations with well-established anti-HER2 therapies. In preclinical models, XMT-2056 showed efficacy in combination with trastuzumab. In both high and low HER2 models, XMT-2056 demonstrated increased efficacy in comparison to benchmark agents such as a trastuzumab-TLR7/8 agonist ADC as well as a small molecule systemically-administered STING agonist. XMT-2056 was generally well-tolerated in non-human primate studies with no clinical signs and no adverse findings in clinical pathology or histopathology after single and repeat IV doses. The Company plans to initiate a Phase 1 study of XMT-2056 in early 2022.
Corporate:

Increased financial flexibility with additional access to capital. The Company entered into a new credit facility at favorable terms for up to $100 million with Oxford and Silicon Valley Bank of which $60 million is available immediately, with remaining balance primarily available upon achievement of certain pipeline and UpRi related milestones. In connection with this new facility, the Company retired the prior debt financing agreement with Silicon Valley Bank.
Strengthened leadership team with key appointments. In August 2021, the Company announced the appointment of Tushar Misra, Ph.D., as Chief Manufacturing Officer. Dr. Misra was most recently EVP, Head of Technical Development & Manufacturing at Laronde and has held senior leadership positions at Wave Life Sciences, Takeda Pharmaceuticals and Sunovion Pharmaceuticals. In October, the Company appointed Mohan Bala, Ph.D., as SVP, Strategic Product Planning & Program Leadership. Dr. Bala was most recently Chief Operating Officer at Constellation Pharmaceuticals and has over 20 years of clinical development and commercialization experience.
Upcoming Events

Mersana plans to present a poster entitled, "STING-agonist ADCs targeting tumor-associated antigens coordinate immune-mediated killing of antigen-negative cancer cells," at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) during the live poster display session available from November 12 – 13, 2021.
Mersana plans to give a corporate presentation at the 2021 Stifel Virtual Healthcare Conference scheduled for November 17, 2021.
Mersana plans to participate in a fireside chat at the Evercore ISI 4th Annual HealthconX scheduled for December 1, 2021.
Third Quarter 2021 Financial Results

Cash and cash equivalents as of September 30, 2021, were $191.7 million, compared to $255.1 million in cash and cash equivalents as of December 31, 2020. Net cash used in operating activities in the third quarter of 2021 was $36.1 million.

The Company expects that its available funds will be sufficient to support its operating plan commitments into the first half of 2023.

Research and development expenses for the third quarter of 2021 were $35.3 million, compared to $16.5 million for the same period in 2020. The difference was primarily due to an increase in manufacturing, clinical and regulatory expenses, an increase in headcount and advancement of diagnostic development efforts for the NaPi2b biomarker. Non-cash stock-based compensation expense included in these research and development expenses increased by $1.7 million.

General and administrative expenses for the third quarter of 2021 were $10.1 million, compared to $5.9 million during the same period in 2020 primarily due to an increase in headcount and consulting and professional fees. Non-cash stock-based compensation expense included in these general and administrative expenses increased by $1.4 million.
Net loss for the third quarter of 2021 was $45.5 million, or $0.63 per share, compared to net loss of $22.5 million, or $0.33 per share, for the same period in 2020. Weighted average common shares outstanding for the quarters ended September 30, 2021 and September 30, 2020 were 71,753,004 and 68,419,192, respectively.
Conference Call Details
Mersana Therapeutics will host a conference call today at 8:00 a.m. ET to report financial results for the third quarter 2021 and provide certain business updates. To access the call, please dial 877-303-9226 (domestic) or 409-981-0870 (international) and provide the Conference ID 2116349. A live webcast of the presentation will be available on the Investors & Media section of the Mersana website at www.mersana.com.

On November 9, 2021 Geron Corporation (Nasdaq: GERN), a late-stage biopharmaceutical company focused on the development and commercialization of treatments for hematologic malignancies, reported that key highlights from its Investor Event held on November 9 (Press release, Geron, NOV 9, 2021, View Source [SID1234595038]). An archive of the presentation webcast is available through the Investor Relations section of Geron’s website under Events. The Company also reported its financial results for the third quarter and year to date 2021 periods. As of September 30, 2021, the Company had $215.8 million in cash and marketable securities. These financial resources, combined with expected future non-dilutive funding under a current debt facility, are expected to be sufficient to fund current operations, including the new programs presented at the Investor Event, through the end of the first quarter of 2023.

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"By targeting telomerase, imetelstat inhibits the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies, resulting in malignant cell apoptosis and potential disease-modifying activity," said John A. Scarlett, M.D., Chairman and Chief Executive Officer. "The compelling efficacy, molecular and cytogenetic data from our Phase 2 trials in lower risk MDS and myelofibrosis patients indicate the potential modification of those underlying diseases, which gives us further confidence in the transformational potential of imetelstat and that the Phase 2 results will be confirmed in our ongoing Phase 3 trials. Our vision aims to bring this same disease modification potential to patients across multiple hematologic malignancies."

Investor Event Highlights

At the Investor Event, key opinion leaders (KOLs) in the treatment of hematologic malignancies presented their views on the current unmet medical needs in these diseases. In addition, the KOLs described the meaningful clinical benefits observed in patients from Geron’s Phase 2 trials in lower risk myelodysplastic syndromes (LR MDS) and myelofibrosis (MF), as well as their involvement in the new programs exploring the use of imetelstat in other indications and in combination with current standard of care treatments. Following is a summary of their perspectives.

Continuing Unmet Needs in LR MDS and MF Potentially Addressed with Imetelstat Treatment

In LR MDS, treatment options for ringed sideroblast negative patients, as well as potential durable transfusion independence, remain areas of unmet need.
In MF, patients have limited treatment options post-JAK inhibitors resulting in shortened survival.
Data from Geron’s Phase 2 trials provide strong evidence that imetelstat can potentially meet the needs in LR MDS and MF patients, which differentiate it from treatments currently available and in development today.
Disease Modification Potential Uniquely Positions Imetelstat in the Treatment of LR MDS and MF

As a first in class telomerase inhibitor, imetelstat inhibits telomerase activity, resulting in apoptosis of malignant cells, limiting the uncontrolled proliferation and survival of malignant clones that drive disease progression.
Clinical benefits observed from Geron’s Phase 2 trials would not have been expected in the absence of imetelstat-associated disease modification.
Correlation of cytogenetic data and mutational changes to clinical benefits further strengthens the evidence of disease-modifying activity of imetelstat.
Imetelstat’s disease modification potential could transform current treatment approaches in LR MDS and MF patients.
Programs to Explore New Indications and Combinations for Imetelstat Expand Telomerase Inhibition Potential

The Company unveiled three new clinical programs and one preclinical program for imetelstat. The clinical programs explore the use of imetelstat as a single agent and in combination with current standard of care treatments to expand the potential application of imetelstat.
The first new clinical program will evaluate imetelstat in combination with ruxolitinib in frontline MF patients. Named IMproveMF, the Geron-sponsored Phase 1 trial will begin as a dose-finding study in approximately 20 patients with a primary endpoint of safety. Upon finding a recommended dose of the combination therapy, the next portion of the trial will confirm the safety of the recommended dose and evaluate the efficacy of the combination therapy. Approximately 20 patients will be enrolled into the second part of the trial and the endpoints include safety, symptom response, spleen response and change in fibrosis. The Company expects the first clinical site for IMproveMF to be open in the first half of 2022.
The second new clinical program will evaluate imetelstat as a single agent in higher risk MDS and acute myeloid leukemia (AML) patients after failing hypomethylating agent (HMA) treatment. Named IMpress, this investigator-sponsored trial has been designed to enroll approximately 45 patients with overall response rate as the primary endpoint. The Company expects IMpress to begin in the first half of 2022.
The third new clinical program will evaluate imetelstat in combination with venetoclax or azacitidine in relapsed/refractory AML patients. Named TELOMERE, this investigator-sponsored trial has been designed to be conducted in two parts. The first part will be a dose finding study in approximately 20 patients with a primary endpoint of safety. Upon finding a recommended dose of the combination therapy, the next portion of the trial will confirm the safety of the recommended dose and evaluate the efficacy of the combination therapy. Approximately 50 patients will be enrolled into the second part of the trial and the primary endpoint is overall response rate. The Company expects TELOMERE to begin in the first half of 2022.
The preclinical program is being conducted at MD Anderson Cancer Center to define the role of imetelstat in lymphoid malignancies. In vitro and in vivo experiments are planned, and the Company expects preliminary results to be available by the end of 2022.
The Company also reviewed the commercial potential for imetelstat in lower risk MDS and refractory MF patients. Based on internal analyses of these two indications, the Company estimates potential peak revenue of more than $3 billion for imetelstat from the United States and the five largest countries in the European Union.

Third Quarter and Year-to-Date 2021 Results

For the third quarter of 2021, the Company reported a net loss of $26.7 million, or $0.08 per share, compared to $19.7 million, or $0.06 per share, for the comparable 2020 period. Net loss for the first nine months of 2021 was $84.1 million, or $0.26 per share, compared to $51.8 million, or $0.20 per share, for the comparable 2020 period.

Revenues for the three and nine months ended September 30, 2021 were $109,000 and $353,000, respectively, compared to $108,000 and $203,000 for the comparable 2020 periods. Revenues in 2021 and 2020 primarily reflect estimated royalties from sales of cell-based research products from the Company’s divested stem cell assets. In connection with the divestiture of Geron’s human embryonic stem cell assets, including intellectual property and proprietary technology, to Lineage Cell Therapeutics, Inc. (formerly BioTime, Inc., which acquired Asterias Biotherapeutics, Inc.) in 2013, Geron is entitled to receive royalties on sales from certain research or commercial products utilizing Geron’s divested intellectual property.

Total operating expenses for the three and nine months ended September 30, 2021 were $25.8 million and $83.4 million, respectively, compared to $20.1 million and $53.9 million for the comparable 2020 periods.

Research and development expenses for the three and nine months ended September 30, 2021 were $18.5 million and $61.6 million, respectively, compared to $13.6 million and $35.3 million for the comparable 2020 periods. The increase in research and development expenses for the three and nine months ended September 30, 2021, compared to the same periods in 2020, primarily reflects increased clinical development costs associated with conducting two Phase 3 clinical trials, higher imetelstat manufacturing costs for producing validation batches at contract manufacturers to enable future production of imetelstat for clinical and commercial purposes and higher personnel-related costs for additional headcount. Under current planning assumptions, the Company expects top-line results from the IMerge Phase 3 trial in lower risk MDS to be available the beginning of January 2023, and for the IMpactMF Phase 3 trial in refractory MF, expects the interim analysis in 2024 and the final analysis in 2025.

General and administrative expenses for the three and nine months ended September 30, 2021 were $7.3 million and $21.8 million, respectively, compared to $6.5 million and $18.6 million for the comparable 2020 periods. The increase in general and administrative expenses for the three and nine months ended September 30, 2021, compared to the same periods in 2020, primarily reflects new costs in connection with pre-commercial activities, including modernizing the internal infrastructure to support a commercial launch, and higher legal costs.

Interest income for the three and nine months ended September 30, 2021 was $112,000 and $421,000, respectively, compared to $322,000 and $1.6 million for the comparable 2020 periods. The decrease in interest income for the three and nine months ended September 30, 2021, compared to the same periods in 2020, primarily reflects lower yields on the Company’s marketable securities portfolio.

Interest expense for the three and nine months ended September 30, 2021 was $1.1 million and $2.6 million, respectively and reflects the Company’s debt facility secured in September 2020 for up to $75 million. As of September 30, 2021, a total of $35.0 million has been drawn down under the facility.

Financial Resources

As of September 30, 2021, the Company had $215.8 million in cash and marketable securities. These financial resources, combined with expected future non-dilutive funding under the current debt facility, are expected to fund current operations, including the new programs presented at the Investor Event, through the end of the first quarter of 2023.

As of September 30, 2021, the Company had 70 employees. The Company plans to grow to a total of approximately 80 to 85 employees by year-end 2021, of which the majority will be development and manufacturing personnel.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Data from Phase 2 clinical trials provide strong evidence that imetelstat targets telomerase to inhibit the uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies resulting in malignant cell apoptosis and potential disease-modifying activity. Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment.

About IMerge Phase 3

IMerge Phase 3 is a double-blind, randomized, placebo-controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 170 transfusion dependent patients with Low or Intermediate-1 risk myelodysplastic syndromes (MDS), also referred to as lower risk MDS, who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). The primary endpoint is the rate of red blood cell (RBC) transfusion independence (TI) for any consecutive period of eight weeks or longer, or 8-week RBC-TI rate. Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks, or 24-week RBC-TI rate, and the rate of hematologic improvement-erythroid (HI-E), defined as a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden.

IMerge Phase 3 is fully enrolled and patient enrollment has been closed. For additional information about IMerge Phase 3, visit ClinicalTrials.gov/NCT02598661.

About IMpactMF

IMpactMF is an open label, randomized, controlled Phase 3 clinical trial with registrational intent. The trial is designed to enroll approximately 320 patients with Intermediate-2 or High-risk myelofibrosis who are refractory to prior treatment with a JAK inhibitor, also referred to as refractory MF. Patients will be randomized to receive either imetelstat or best available therapy. The primary endpoint is overall survival (OS). Key secondary endpoints include symptom response, spleen response, progression free survival, complete response, partial response, clinical improvement, duration of response, safety, pharmacokinetics, and patient reported outcomes.

IMpactMF is currently enrolling patients. For further information about IMpactMF, including enrollment criteria, locations and current status, visit ClinicalTrials.gov/NCT04576156.

On November 9, 2021 Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) and Pfizer Inc. (NYSE: PFE), reported a strategic commercialization arrangement for rimegepant in markets outside of the United States upon approval (Press release, Biohaven Pharmaceutical, NOV 9, 2021, View Source [SID1234595037]). Rimegepant is commercialized as Nurtec ODT in the U.S., and is indicated for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine in adults. An application for the approval of rimegepant is currently under review by the European Medicines Agency and several additional regulatory authorities outside of the U.S.

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"We believe this collaboration, which brings together the winning combination of Biohaven’s Neuroscience R&D with Pfizer’s industry-leading expertise and large global footprint will help accelerate access to rimegepant for patients around the world," said Vlad Coric MD, Chief Executive Officer of Biohaven. "With this alliance, Biohaven Pharmaceutical and Pfizer believe there is an opportunity to change the paradigm in migraine treatment and potentially establish a new standard of care."

Under the terms of the arrangement, Biohaven would remain primarily responsible for further clinical development of rimegepant and the parties will cooperate in regulatory activities to secure approval for the product. Biohaven will continue to solely commercialize Nurtec ODT in the U.S and Pfizer would commercialize rimegepant, upon approval, in all regions outside the U.S. Additionally, per the arrangement, Pfizer gains rights outside of the U.S. to zavegepant, a third generation, high affinity, selective and structurally unique, small molecule CGRP receptor antagonist, currently being studied in an intranasal delivery and a soft-gel formulation in Phase 3 clinical trials for migraine indications.
"We are excited to join forces with Biohaven in the fight against migraine and help those patients impacted by this debilitating neurological disease," said Nick Lagunowich, Global President, Pfizer Internal Medicine. "We believe our legacy in pain and Women’s Health combined with our experienced customer-facing colleagues, will enable us to maximize this opportunity with Biohaven, potentially bringing a valuable new treatment option to patients living with migraine pain."

Terms of the Arrangement
Biohaven and Pfizer are entering into a collaboration and license agreement and related sublicense agreement pursuant to which Pfizer will acquire rights to commercialize rimegepant and zavegepant outside of the U.S. Biohaven will continue to lead research and development globally and Pfizer would execute commercialization globally, outside of the U.S. Under the financial terms of all transaction agreements, Pfizer will make an upfront payment of $500 million, consisting of $150 million cash and $350 million in the purchase of Biohaven equity at a 25 percent market premium. Biohaven is also eligible to receive up to $740 million in milestones. In addition to the tiered double-digit royalties owed to Biohaven on net sales outside of the U.S., Pfizer will compensate Biohaven for the related royalties on net sales outside of the U.S. owed under the Company’s license and funding agreements with Bristol-Myers Squibb Company and Royalty Pharma.

As noted above, in connection with the transaction, Pfizer will make a $350 million investment in the common shares of Biohaven.
Closing of the license agreements and equity purchase are contingent on completion of review under applicable antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S. and equivalents outside the U.S., and other customary closing conditions.
Biohaven and Pfizer global collaboration will be discussed on Biohaven 3Q Earnings Investor Call 8:00AM ET on November 9, 2021. To access the call, please dial 877-407-9120 (domestic) or 412-902-1009 (international). The conference call webcast and accompanying slide presentation can be accessed through the "Investors" section of Biohaven’s website at www.biohavenpharma.com.

About Migraine
More than one billion people suffer from migraine worldwide, of which 75 percent are women. The World Health Organization classifies migraine as one of the 10 most disabling medical illnesses. Migraine is characterized by debilitating headache attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity that can be associated with nausea or vomiting, and/or sensitivity to sound (phonophobia) and sensitivity to light (photophobia). There is a large unmet need for new acute and preventive treatments, as a significant portion of migraine patients are unsatisfied with current standard of care migraine treatments due to a lack of efficacy or safety or tolerability burden.

About Rimegepant
Rimegepant targets a root cause of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) under the trade name Nurtec ODT for the acute treatment of migraine in February 2020 and for the preventive treatment of episodic migraine in May 2021. Nurtec ODT is the #1 prescribed migraine treatment in its class with a cumulative launch to date of U.S. net revenue of approximately $336 million and with more than one million prescriptions. A single dose of 75 mg Nurtec ODT provides fast pain relief, significant pain reduction and return to normal function, and has a lasting effect of up to 48 hours in some patients. Nurtec ODT is taken orally as needed, up to 18 doses/month to stop migraine attacks or taken every other day to help prevent migraine attacks and reduce the number of monthly migraine days. Nurtec ODT does not have addiction potential and is not associated with medication overuse headache or rebound headache.

NURTEC ODT U.S. IMPORTANT SAFETY INFORMATION
Nurtec ODT (orally disintegrating tablet) is a prescription medicine that is used to treat migraine in adults. It is for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine. It is not known if Nurtec ODT is safe and effective in children.
Do not take Nurtec ODT if you are allergic to Nurtec ODT (rimegepant) or any of its ingredients.
Before you take Nurtec ODT, tell your healthcare provider (HCP) about all your medical conditions,
including if you:

•have liver problems,
•have kidney problems,
•are pregnant or plan to become pregnant,
•breastfeeding or plan to breastfeed.

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Nurtec ODT may cause serious side effects including allergic reactions, including trouble breathing and rash. This can happen days after you take Nurtec ODT. Call your HCP or get emergency help right away if you have swelling of the face, mouth, tongue, or throat or trouble breathing. This occurred in less than 1% of patients treated with Nurtec ODT.
The most common side effects of Nurtec ODT were nausea (2.7%) and stomach pain/indigestion (2.4%). These are not the only possible side effects of Nurtec ODT. Tell your HCP if you have any side effects.

On November 9, 2021 Pliant Therapeutics, Inc. (Nasdaq: PLRX) ("the Company"), a clinical stage biotechnology company focused on discovering and developing novel therapeutics for the treatment of fibrosis, reported third quarter 2021 financial results (Press release, Pliant Therapeutics, NOV 9, 2021, View Source [SID1234595036]).

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"The third quarter was productive for the company, highlighted by the interim data readout from our PET trial which exceeded our expectations, showing a dose response and achieving near target saturation of PLN-74809 at the higher doses," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "In addition to the positive data readout, our Phase 2a INTEGRIS-IPF and INTEGRIS-PSC programs are making great progress in enrollment, and, with the additional Phase 1b BAL doses, we continue to methodically de-risk the current and future clinical development of PLN-74809."

Third Quarter and Recent Highlights

Positive interim results from Phase 2a PET imaging clinical trial show αvβ6 target engagement up to 98% in the lungs of patients with idiopathic pulmonary fibrosis (IPF). The ongoing Phase 2a open-label PET imaging clinical trial is evaluating αvβ6 target engagement levels achieved by PLN-74809 across single-doses of 60, 120, 240 or 320 mg in up to 12 IPF patients. Interim results showed a dose response and a greater than 50% target engagement across all doses and established a model of the dose and plasma concentration response. Furthermore, these data support potential anti-fibrotic activity of PLN-74809 at the doses being evaluated in the ongoing Phase 2a INTEGRIS-IPF trial.

PLN-74809 Phase 2a trial in idiopathic pulmonary fibrosis (IPF) enrollment on track with topline data anticipated by mid-2022. INTEGRIS-IPF is a 12-week randomized, dose-ranging, double-blind, placebo-controlled trial evaluating the safety, tolerability and pharmacokinetics of PLN-74809 at doses of 40, 80 or 160 mg in IPF patients. Exploratory endpoints include quantitative lung fibrosis (QLF) imaging, pulmonary function tests as well as biomarkers. Enrollment is currently on track to be completed by the end of 2021 with topline data anticipated by mid-2022.

Data anticipated in the first quarter of 2022 from additional doses in the PLN-74809 Phase 1b proof-of-mechanism study evaluating inhibition of TGF-β signaling in the lungs of healthy volunteers. In a previously reported Phase 1b proof-of-mechanism study, 40 mg once-daily dosing of PLN-74809 inhibited activation of TGF-β, a key driver of fibrosis. Based on additional safety and pharmacokinetic data from the Phase 1a study, Pliant initiated an extension of the Phase 1b study. This study extension evaluates inhibition of TGF-b signaling at doses above 40 mg of PLN-74809. These data, coupled with the recently reported positive interim Phase 2a PET target engagement data, will provide important information about the relationship between PLN-74809 plasma exposure, target engagement, TGF-β signaling inhibition and potential antifibrotic activity across the dose range currently being tested in the INTEGRIS-IPF trial. Data from this Phase 1b study is anticipated in the first quarter of 2022.

PLN-74809 Phase 2a trial in primary sclerosing cholangitis (PSC) enrollment on track. INTEGRIS-PSC is a 12-week randomized, dose-ranging, double-blind, placebo-controlled trial evaluating the safety, tolerability, and pharmacokinetics PLN-74809 at doses of 40, 80 or 160 mg in PSC patients. Exploratory endpoints include fibrosis

biomarkers such as Pro-C3 and ELF, changes in ALP and liver imaging. Enrollment is currently anticipated to be completed by mid-2022.

Muscular Dystrophy and Oncology programs advancing towards IND. Early-stage integrin targeting programs focused on developing novel therapies in oncology and muscular dystrophies continue to advance towards IND. The muscular dystrophy program utilizes an antibody to improve muscle function through activation of an integrin compensatory mechanism. The oncology program focuses on increasing tumor sensitivity to checkpoint inhibitors through small molecule inhibition of αvβ8.

COVID-19 Preparedness
The Company continues to develop and maintain policies and procedures to enable us to operate safely and productively during the COVID-19 pandemic. The Company has experienced delays in clinical trial operations which have impacted and may further impact the expected timing of data readouts. The Company continues to work closely with clinical sites to continue site initiation and operation activities in compliance with study protocols while observing government and institutional guidelines.

Third Quarter 2021 Financial Results
•Research and development expenses were $21.1 million, as compared to $16.9 million for the prior-year quarter. The increase was due primarily to employee related expenses and higher costs related to the advancement of several programs and ongoing Phase 1/2 clinical trials.
•General and administrative expenses were $7.7 million, as compared to $4.6 million for the prior-year quarter. The increase was due to higher personnel-related and professional services expenses.
•Net loss of $27.0 million as compared to $16.5 million for the prior-year quarter due an overall increase in expense associated with our research and development programs as well as personnel-related costs.
•As of September 30, 2021, the Company had cash, cash equivalents and short-term investments of $221.0 million. The Company believes it has sufficient funds to meet its operating and capital requirements into 2023.

On November 9, 2021 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported it is 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 (Press release, Ligand, NOV 9, 2021, View Source [SID1234595035]). The spin-off is intended to create two companies with dedicated operational focus, business-specific capital allocation, agility to meet partner needs, and compelling focused investment profiles.

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"Our OmniAb business continues to have an outstanding year with the first regulatory approval for an OmniAb-derived antibody received during the third quarter and a second approval anticipated by year-end," said John Higgins, CEO of Ligand. "We believe more than ever that OmniAb offers one of the industry’s leading antibody discovery platforms and that the business is primed for success for years to come. After significant planning and analysis, we have concluded we are operating two distinct, high-growth companies within Ligand. Along with outside advisors we have determined the time is right to pursue a strategic plan to create two independent companies and accelerate investment into the OmniAb platform and technologies to further drive value."

Based on initial management and advisor review, an IPO and eventual distribution of OmniAb shares to Ligand shareholders is the leading option under consideration at this time. The IPO would be of newly issued shares of OmniAb, Inc., which would include the Ab Initio computational antigen design technology, Icagen’s ion channel technology, the xPloration high-throughput screening technology, and the suite of OmniAb transgenic animals used for antibody discovery. In an IPO, Ligand expects OmniAb to issue less than 20% of its common stock, with Ligand retaining the remaining interest, which would eventually be distributed to Ligand stockholders in a manner generally intended to qualify as a tax-free transaction. Ligand’s OmniAb strategic review is in response to the ongoing success of OmniAb, as well as the opportunity to unlock the value of this business. Ligand’s Board of Directors has not approved a specific course of action, and Ligand will continue to evaluate other options to optimize value and ensure flexibility to invest in growth. There can be no assurance that this process will result in Ligand pursuing a particular transaction or consummating any such transaction.

This press release does not constitute an offer to sell or the solicitation of an offer to buy securities, and shall not constitute an offer, solicitation, or sale in any jurisdiction in which such offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

The latest corporate presentation can be accessed at investor.ligand.com.

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 third quarter financial results as well as this announcement, and to 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.