On March 9, 2021 Foundation Medicine, Inc. and InformedDNA, the nation’s largest independent provider of genetics services, reported a collaboration that will enable physicians to refer U.S. patients with possible inherited cancer gene mutations to genetic counseling services and confirmatory genetic testing based on the results of Foundation Medicine’s tumor testing (Press release, Foundation Medicine, MAR 9, 2021, View Source [SID1234576614]).

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For patients who undergo Foundation Medicine’s comprehensive genomic profiling tests and receive a result showing a potential inherited cancer gene variant, genetic counseling and follow up germline (genetic) testing aligns with clinical guidelines and industry best practices, and provides oncologists and their patients with more data to inform treatment decisions.

Cancer can be influenced by many different genetic changes; some are acquired over time, known as somatic mutations, and others are inherited, known as germline mutations. Foundation Medicine’s tests evaluate more than 300 cancer-related genes, including genes with variants that may have potential germline implications. If a potential germline variant is identified via tissue or liquid-based genomic testing, Foundation Medicine’s test report highlights this to the patient’s provider, so that the provider may consider follow up with genetic counseling and confirmatory germline testing.

Through this new collaboration, Foundation Medicine and InformedDNA are helping advanced cancer patients and their physicians navigate the appropriate genetic counseling and confirmatory testing resources available to them should they choose to leverage them to inform treatment plans.

"Both somatic and germline drivers of disease are critical to inform treatment decisions and assess future risk, yet many physicians and health systems do not have a referral process in place or access to internal genetic counseling services and confirmatory germline testing. Further, given the broader implications of this information, responsible communication from a genetic counselor is critical," said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. "We’re pleased to partner with InformedDNA to create a unique option that enables access to genetic counseling and guidance on confirmatory testing, thereby assisting care teams with obtaining additional information to develop holistic treatment plans for patients and their families."

InformedDNA is a leader in telegenetic counseling, offering services with board-certified genetic counselors across all 50 states. If confirmatory genetic testing is desired and deemed necessary following a potential germline variant finding on one of Foundation Medicine’s reports, InformedDNA’s genetic counselors will guide patients in determining the appropriate test and understanding the benefits, risks, and implications of confirmatory germline testing.

"Together, we’re improving access to an important aspect of tumor testing for cancer patients – the ability to identify patients at risk of carrying inherited genetic mutations," said Rebecca Sutphen, M.D., FACMGG, co-founder and chief medical officer at InformedDNA. "This collaboration aims to make genetic counseling and confirmatory testing more accessible to U.S. patients with potential germline mutations, helping to ensure that physicians have the information needed to develop personalized cancer treatment plans for their patients and implement preventive measures for their families."

Approximately five to 10 percent of all cancers are hereditary.1 In addition to playing a role in diagnosing and treating an individual with cancer, genetic information can be used to help inform health decisions for a patient’s family members.

"We believe it is essential for all advanced cancer patients to have access to the tools needed to understand the hereditary implications of their cancer, if interested," said Sue Friedman, executive director and founder of Facing Hereditary Cancers Empowered (FORCE). "We applaud Foundation Medicine and InformedDNA for embarking on this effort to fill an important gap in cancer care, ensuring people affected by hereditary cancer have the information, support and guidance they need to navigate treatment and prevention."

Foundation Medicine’s reporting of tumor variants does not distinguish between somatic or germline variants and is not intended to replace germline testing or provide information specific to cancer predisposition.

On March 9, 2021 ANI Pharmaceuticals, Inc. ("ANI" or the "Company") (Nasdaq: ANIP) reported that it has signed a definitive agreement to acquire Novitium Pharma, a privately held, New Jersey-based pharmaceutical company with development, manufacturing, and commercialization capabilities for $163.5 million, including $89.5 million in cash and $74 million in equity plus two potential future cash earn-outs of up to $46.5 million (Press release, ANI Pharmaceuticals, MAR 9, 2021, View Source [SID1234576613]). The transaction has been approved by the ANI Board of Directors and is expected to close in the second half of 2021, subject to regulatory approvals and approval by ANI shareholders as required by Nasdaq listing standards as described below. Equity holders of Novitium have approved the transaction.

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"Strengthening our well-established Generics business with enhanced development capabilities and increased focus on niche opportunities is a key pillar of ANI’s growth strategy. The acquisition of Novitium aligns with this strategy, adding a best-in-class R&D engine that delivers sustainable growth through new product launches and offering upside through niche 505(b)(2) opportunities. The addition of a U.S. based, state-of-the-art manufacturing facility and enhanced scale to our CDMO business are other attractive advantages of this acquisition. We also look forward to welcoming Samy, Chad, Vijay and the entire Novitium team to ANI," stated Nikhil Lalwani, President and CEO.

"We expect the transaction will be immediately accretive to our adjusted EBITDA. Upon closing of the transaction, our strengthened pro forma capital structure, including the new senior credit facilities underwritten by Truist Securities and the PIPE from Ampersand Capital Partners will enable us to facilitate long-term growth," concluded Lalwani.

Samy Shanmugam, Co-Founder and President, and Chad Gassert, Co-Founder and CEO, commented, "We are delighted to join ANI as we enter this next phase of exciting growth at Novitium Pharma. We look forward to supporting ANI to execute on their strategy of growing their business, expanding and diversifying their product portfolio, and creating long-term value for their investors."

Transaction Strongly Positions ANI for Future Growth

Proven R&D Engine Fuels Sustainable Growth
Novitium anticipates launching 25+ additional products in 2021 and 2022, including products with U.S. Food and Drug Administration ("FDA") Competitive Generic Therapy ("CGT") designation. The Company currently has 21 abbreviated new drug applications ("ANDAs") filed with the FDA and more than 30 additional products under development. Novitium’s leadership of Samy Shanmugam, Chad Gassert, Vijay Thorappadi and the entire team has an excellent track record of execution and efficiency from filing to launch. Integration of Novitium’s R&D operations will establish a center of excellence for ANI’s generics product development program.
Expands ANI’s R&D Pipeline Focused on Niche Opportunities
Novitium is currently advancing three 505(b)(2) candidates in Oncology and Hypertension. These 505(b)(2) products will build on ANI’s increasing focus on niche opportunities, including its first Paragraph IV filing in 2020 and the filing of injectables ANDAs in 2021.
Enhances Scale of CDMO Business and U.S.-Based Manufacturing Capacity
Novitium adds nine new customers to ANI’s growing CDMO business. Additionally, Novitium brings with it a 50,000 sq ft East Windsor, NJ facility, including 27 manufacturing suites and eight clinical suites supporting R&D, commercial manufacturing and packaging. An ongoing 20,000 sq ft building expansion will add 18 new manufacturing suites. Novitium’s annual production capacity is approximately two billion units in aggregate across tablets, capsules, liquid suspensions and solutions, powders and powder for oral suspension, controlled release and potent compounds.
Compelling Financial Profile
The combination is expected to be accretive to ANI’s adjusted non-GAAP EPS in the first 12 months after closing. The transaction is expected to add approximately $15 million to adjusted EBITDA in the second half of 2021, assuming a mid-year 2021 close. The acquisition diversifies ANI’s revenue base by contributing to each of its reporting segments: Generics, Contract Manufacturing, Royalties/Other and, following the launch of Novitium’s 505(b)(2) pipeline products, the Brand segment.
Terms of the Transaction & Financing

Under the terms of the transaction, the Purchase Price of $163.5 Million is comprised of (i) a cash payment of $89.5 million and (ii) $74 million in equity. Novitium is also eligible to receive (i) $25 million in contingent payments upon the achievement of financial targets related to Generics products and filing of certain ANDAs and (ii) $21.5 million in contingent payments upon the achievement of financial targets from the 505(b)(2) products.

ANI will finance the transaction with a new $300 million Term Loan B, issuance of $74 million in equity to the sellers, and a $25 million PIPE investment by Ampersand Capital Partners. The facility also includes a new $40 million revolving credit facility. The new debt financing will be secured by substantially all the assets of ANI and its subsidiaries and used for the cash portion of the acquisition and to refinance ANI’s existing senior credit facilities. The proposed capital structure further facilitates sustainable growth for ANI.

Approvals

The transaction is expected to be completed in the second half of 2021, subject to the satisfaction of customary closing conditions, l approval from relevant regulatory agencies, including clearance under the Hart-Scott Rodino Antitrust Improvements Act, and approval by ANI shareholders under Nasdaq listing standards as the result of the equity portion of the Novitum transaction and the shares issuable in the PIPE transaction.

Advisors

Bourne Partners, Truist Securities and Houlihan Lokey acted as financial advisors to ANI Pharmaceuticals. SVB Leerink acted as financial advisor to Novitium Pharma and its shareholders. Hughes Hubbard & Reed were ANI’s legal advisors and Orrick, Herrington & Sutcliffe acted as legal advisors to Novitium and its shareholders.

On March 9, 2021 AstraZeneca reported that The UK’s National Institute for Health and Care Excellence’s (NICE) appraisal committee has chosen not to recommend PARP inhibitor Lynparza for BRCA-positive metastatic prostate cancer (Press release, AstraZeneca, MAR 9, 2021, View Source [SID1234576608]).

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Lynparza (olaparib) is intended for the treatment of hormone-relapsed metastatic prostate cancer with BRCA1/2 mutations that has progressed after abiraterone or enzalutamide treatment in adults.

The current treatment for these patients includes docetaxel, cabazitaxel, or radium-223. In its draft guidance, NICE said that there is no direct cimparision of Lynparza to this current standard of care.

It added that although clinical trial evidence shows people receiving Lynparza treatment experienced improved progression-free survival and overall survival compared to people having re-treatment with abiraterone or enzalutamide, the evidence is uncertain.

This is because re-treatment with abiraterone or enzalutamide is not considered effective and is not the standard of care in the NHS.

NICE also said that it is uncertain how effective Lynparza is compared to docetaxel, cabazitaxel, or radium-223 as there is no direct comparison.

An indirect comparison suggests that Lynparza increases overall survival compared to cabazitaxel, but NICE reiterated that this is uncertain.

In addition, the cost-effectiveness estimates for Lynparza are uncertain because of the above ‘limitations’ in the clinical evidence and economic model.

This means that the estimates are higher than what NICE would usually consider an acceptable use of NHS resources.

The draft guidance is open to consultation until 26 March 2021, after which NICE will make its final recommendations for Lynparza in this indication.

On March 9, 2021 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel immunotherapy treatments for cancer, infectious disease and autoimmune disease, reported the grant of patent number 10,940,181 entitled "Combined Preparations for the Treatment of Cancer or Infection" by the United States Patent & Trade Mark Office (Press release, Immutep, MAR 9, 2021, View Source [SID1234576377]).

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This United States patent was filed as a divisional application and follows the grant of the United States parent patent announced on 30 December 2020.

The claims of this new patent build on the protection provided by the parent patent, and are directed to methods of treating cancer by administering Immutep‘s lead active immunotherapy candidate eftilagimod alpha ("efti" or "IMP321") and a PD-1 pathway inhibitor. Again, according to the claims, the PD-1 pathway inhibitor is either pembrolizumab or nivolumab. The expiry date of the patent is 20 January 2036 (including a patent term adjustment of 12 days).

"We are very pleased to add another United States patent to our expanding patent portfolio, especially in this case, because of its direct relevance to our clinical development programs. These patent grants are important as they underpin ongoing investment in clinical development of efti and allow us to confidently engage in business development discussions," said Marc Voigt, CEO of Immutep.

A further divisonal application has been filed to pursue other aspects of the invention, including combinations where the PD-1 pathway inhibtor is a PD-L1 inhibitor.

On March 9, 2021 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases,reported financial results for the year ended December 31, 2020 and provided an update on clinical activities and financial guidance for 2021 (Press release, NewLink Genetics, MAR 9, 2021, View Source [SID1234576364]).

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"The fourth quarter and full year 2020 were marked by significant achievements for Lumos Pharma," commented Rick Hawkins, Chairman, CEO and President of Lumos Pharma. "From the completion of our merger last spring, the subsequent sale of our PRV providing significant non-dilutive funds, to the initiation of our Phase 2b OraGrowtH210 Trial evaluating our orally administered therapeutic for PGHD, Lumos Pharma has built a solid foundation to advance our clinical and corporate strategy targeting PGHD, diseases of growth hormone deficiency, and other rare diseases in the coming year."

Clinical Updates

LUM-201 Data Published in Journal of Endocrine Society (JES) – The manuscript, "Development of a Predictive Enrichment Marker for Oral GH Secretagogue LUM-201 in Children with Growth Hormone Deficiency," by Bright, G., MD, et al, was published in the Journal of Endocrine Society late February. This peer-reviewed analysis of data from prior studies of LUM-201 in pediatric growth hormone deficiency (PGHD) supports the utility of specific predictive enrichment markers (PEMs) in identifying PGHD patients likely to respond to LUM-201. The two PEMs identified after a single dose of LUM-201, baseline IGF-1 cut-off level > 30 ng/ml and peak GH level ≥ 5 ng/mL, are being used to qualify PGHD patients for enrollment in our OraGrowtH210 Trial.
Predictive Enrichment Markers Define Subset of Moderate Growth Hormone Deficiency – The manuscript, "Corroboration Between Prediction Enrichment Markers for Height Velocity to rhGH and an Oral GH Secretagogue Treatment in Children with Moderate GHD," by Blum, W., PhD, et al, was published in the Journal of Endocrine Society late February. This peer-reviewed analysis of data on children with growth hormone deficiency (GHD) in a large legacy database (GeNeSIS data) corroborates that approximately 60% of the total pediatric GHD patient population meet the definition of moderately GHD deficient (PEM-positive) and are likely to respond to a growth hormone secretagogue.
Poster to be Presented at ENDO 2021 – The poster entitled, "LUM-201 Elicits Greater GH Response than Standard GH Secretagogues in Pediatric Growth Hormone Deficiency," (abstract 7102) will be presented at the Endocrine Society 2021 Annual Meeting, March 20th-23rd.
Phase 2b OraGrowtH210 Trial Advances – The Phase 2b OraGrowtH210 Trial initiated in Q4 2020 continues to advance. This global Phase 2b trial will evaluate orally administered LUM-201 in approximately 80 patients diagnosed with PGHD. The purpose of this trial will be to prospectively confirm our Predictive Enrichment Marker (PEM) strategy and to identify the optimal dose of LUM-201 to be used in a Phase 3 registration trial. The Company continues to anticipate data read-out for the OraGrowtH210 Trial mid-year 2022.
OraGrowtH212 Trial (PK/PD study) of LUM-201 in PGHD Initiation Anticipated Q2 2021 – This study will evaluate the pharmacokinetic and pharmacodynamic (PK/PD) effects of two dose levels of LUM-201 (1.6 and 3.2 mg/kg/day) in approximately 24 PGHD patients at a single specialized clinical site. The purpose of this study will be to confirm prior preclinical and clinical data supporting the increased pulsatile release of endogenous growth hormone peaks that characterizes the unique mechanism of action of LUM-201. Recently we were informed of a fire at the San Borja Arriaran Hospital in Santiago, Chile, which is the location of the OraGrowtH212 trial. While we had originally expected to initiate this trial in Q1 2021, we now anticipate the initiation of the OraGrowtH212 Trial to occur in Q2 2021 due to potential delays as the hospital addresses this incident. Our investigator’s clinic was not directly involved in the fire, and we continue to work with our local contacts to advance the trial. As we have previously stated, this trial is not on the critical path for regulatory approval of LUM-201, and we do not anticipate the fire will cause any delays to our previously stated regulatory approval timeline. We are exploring alternate sites to conduct the trial in the event that the original site is unable to proceed in a timely manner.
OraGrowtH211 Trial, a Long-Term Extension Study, is Announced – Lumos Pharma announced the OraGrowtH211 Trial, an extension study to determine the long-term safety, PK/PD markers and growth outcomes attributable to LUM-201 administered to children with growth hormone deficiency. The OraGrowtH211 Trial will be open to all eligible PGHD patients who have completed OraGrowtH210, OraGrowtH212 or other subsequent LUM-201 trials.
Business Development – The Company continues to pursue opportunities to expand our rare disease pipeline through the in-licensure or acquisition of another novel therapeutic candidate for those suffering from rare diseases.
Corporate Updates

Received Final Tranche of Funds from PRV Sale – In January 2021, Lumos received the second and final tranche of $26.0 million from the total $60.0 million due to the Company from the PRV sale. We anticipate these funds will serve as additional capital to support the expansion of the Company’s pipeline through its business development efforts.
Financial Guidance for 2021 – The Company anticipates average cash use of approximately $8.0 to $9.0 million per quarter through 2021.
Management Changes – As previously disclosed, Eugene Kennedy, MD, Chief Medical Officer (CMO), departed Lumos Pharma March 4, 2021 to join a privately held company focused on developing therapeutics for patients suffering from cancer. Lumos Pharma will conduct a search for his replacement. John McKew, PhD, COO and CSO together with George Bright, MD, VP, Clinical Development and a pediatric endocrinologist, will cover all CMO responsibilities in the interim.
Appointed New Board Member with Rare Disease Background – February 16, 2021, Lumos Pharma announced the appointment of new Board member, An van Es-Johansson, M.D., with a wealth of experience in rare diseases. Dr. van Es-Johansson recently served as the Chief Medical Officer and Head of Development for AlzeCure Pharma, a Swedish pharmaceutical company with a primary focus on Alzheimer’s disease, where she now serves as a senior advisor. Dr. van Es-Johansson’s early work in the life science industry focused on the clinical development of recombinant human growth hormone (rhGH) therapeutics for Turner Syndrome and other endocrine disorders at both Eli Lily and Pharmacia Upjohn. Since then, Dr. van Es-Johansson has held leadership roles at several large and small biopharmaceutical companies and currently serves on the Board of Directors at Medivir AB, Savara Pharmaceuticals, PLUS Therapeutics, and Agendia BV. Dr. van Es-Johansson received a M.D. from Erasmus University, Rotterdam, The Netherlands.
Financial Results for the Year Ended December 31, 2020

Cash Position – Lumos Pharma ended the year on December 31, 2020, with cash and cash equivalents totaling $98.7 million compared to $5.0 million on December 31, 2019 and pro forma December 31, 2019 cash of $95.5 million, inclusive of NewLink Genetics. The Company expects its cash on hand is sufficient to fund current operations through the read-out of our Phase 2b OraGrowtH210 Trial and completion of the OraGrowtH212 Trial.
R&D Expenses – Research and development expenses for the year ended December 31, 2020 were $9.2 million, an increase of $3.5 million from $5.7 million for the same period in 2019. The increase is primarily due to increases of $2.4 million in clinical trial expenses, $1.4 million in personnel-related and stock compensation expenses, $0.8 million in supplies and other expenses and $0.4 million in write-off of the acquired NewLink’s in-process research and development costs, offset by a decrease of $1.5 million in contract manufacturing expense.
G&A Expenses – General and administrative expenses for the year ended December 31, 2020 were $17.3 million, an increase of $13.1 million from $4.2 million for the same period in 2019. The increase was due primarily to increases of $7.0 million in personnel-related and stock compensation expenses, $4.2 million in operating expenses for insurance, rent, supplies, and depreciation expenses, $1.6 million due to the Merger related expenses and $0.6 million in legal and consulting expenses, offset by a decrease of $0.3 million in travel expenses.
Net Loss – The net loss for the year ended December 31, 2020 was $5.7 million compared to a net loss of $9.7 million for the same period in 2019.
Lumos Pharma ended Q4 2020 with 8,305,269 shares outstanding.
Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 4:30 p.m. ET today to discuss its financial results and to give an update on clinical and business development activities. There will also be a question-and-answer session following management’s prepared remarks.

Access to the live conference call is available five minutes prior to the start of the call by dialing (855) 469-0612 (U.S.) or (484) 756-4268 (international). The conference call will be webcast live and a link to the webcast can be accessed through the Lumos Pharma website at View Source in the "Investors & Media" section under "Events and Presentations" or through this link: View Source To ensure a timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and using the passcode 3735869. The replay will be available for two weeks from the date of the call.