On August 11, 2020 BridgeBio Pharma, Inc. (Nasdaq: BBIO), a clinical-stage biopharmaceutical company focused on genetic diseases and cancers with clear genetic drivers, reported its financial results for the second quarter ending June 30, 2020 and provided an update on the company’s operations (Press release, BridgeBio, AUG 11, 2020, View Source [SID1234563437]).

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Since the beginning of 2020, BridgeBio has initiated four company-sponsored clinical trials, progressed its additional 11 ongoing clinical trials, submitted three Investigational New Drug (IND) applications to the U.S. Food and Drug Administration (FDA), and completed the rolling submission of its first New Drug Application (NDA) with the FDA. During the quarter, BridgeBio strengthened its corporate governance by adding three world-class independent directors to its board. In addition, it recently entered into a partnership with Perceptive Advisors-founded company LianBio, expanding BridgeBio’s global reach into China.

BridgeBio remains on track with each of its four core value drivers – acoramidis (formerly AG10, TTR stabilizer) for ATTR cardiomyopathy, low-dose infigratinib (FGFRi) for achondroplasia, AAV5 gene therapy for congenital adrenal hyperplasia (CAH), and encaleret (CaSRi) for autosomal dominant hypocalcemia type 1 (ADH1) – and the company believes it is adequately financed through key readouts for each of these programs. Notably, BridgeBio dosed the first child in its Phase 2 clinical trial of infigratinib in achondroplasia in July.

The strategic collaboration with LianBio will initially focus on targeted oncology. BridgeBio’s near-term economics includes a total of $26.5 million in upfront and milestone payments. BridgeBio will additionally receive up to $505 million in future milestone payments, tiered royalty payments ranging from single- to double-digits and increase its equity interest via investment in LianBio. BridgeBio CEO Neil Kumar has also been appointed to the LianBio board of directors.

Across the company, BridgeBio’s drug engineering platform continues to deliver. Strengthening its ability to discover new targets, it established collaboration agreements with Johns Hopkins University and University of Florida and continues to assess a wide variety of new programs in the genetic disease space. BridgeBio’s pre-clinical platform has expanded to include additional modalities such as antisense oligonucleotides and deepened expertise in critical areas such as molecular modeling and novel statistical approaches to genetics. Its clinical platform has grown and now encompasses more than 350 trial sites in over 25 countries.

"On a risk-adjusted basis we are in a great position to produce meaningful medicines for patients and meaningful returns to investors over the next 18 to 24 months. This is exemplified by our disease-modifying, first or best-in-class therapeutic candidates for ATTR, achondroplasia, ADH1, and CAH. We intend to deliver on this goal by expanding our industry-leading target identification and research engine and global clinical development infrastructure, and look forward to delivering our medicines, once approved, to patients through our growing commercial organization," said BridgeBio CEO and founder Neil Kumar, Ph.D.

Recent pipeline progress and corporate updates:

Low-dose infigratinib – Selective FGFR inhibitor for achondroplasia: Dosed first child in the Phase 2 clinical program (PROPEL 2) (NCT04265651).
BBP-418 – Glycosylation substrate pro-drug for LGMD2i: Dosed first subject in Phase 1 clinical trial in healthy volunteers.
Expansion into China through partnership with LianBio: BridgeBio entered into a strategic collaboration with Perceptive Advisors-founded LianBio, expanding its reach into China and other major Asian markets. The initial focus of the collaboration will be targeted oncology. Under the terms of the agreements, LianBio receives commercial rights in China and selected Asian markets and will participate in clinical development activities for BridgeBio’s Phase 3 FGFR inhibitor infigratinib and Phase 1-ready SHP2 inhibitor BBP-398. BridgeBio’s near-term economics includes a total of $26.5 million in upfront and milestone payments. BridgeBio will additionally receive up to $505 million in future milestone payments, tiered royalty payments ranging from single- to double-digits and increase its equity interest via investment in LianBio. BridgeBio CEO Neil Kumar has also been appointed to the LianBio board of directors.
Three new independent directors added to BridgeBio’s board:
○ Brent Saunders, former Allergan CEO and biopharma deal-maker
○ Randy Scott, Ph.D., genomics pioneer and entrepreneur
○ Andrew Lo, Ph.D., renowned economist and BridgeBio co-founder
New academic partnerships: Established collaboration agreements with Johns Hopkins University and University of Florida to accelerate the development of new medicines in genetically driven diseases.
BridgeBio Pharma R&D Day: BridgeBio will hold a virtual R&D Day on Tuesday, Sept. 29, 2020 from 8:30 am ET – noon. The event will be webcast, with a link available on the event calendar at View Source
Major milestones anticipated over the next 18-24 months for BridgeBio’s four core value drivers:

Acoramidis (formerly AG10) – TTR stabilizer for ATTR: Remain on track to complete enrollment in the Phase 3 ATTRibute-CM study in ATTR cardiomyopathy (ATTR-CM) in the first half of 2021, with topline data expected in the first half of 2022. Acoramidis is a potentially best in class TTR stabilizer for ATTR-CM, a large and growing disease affecting >400K patients globally, and one of the first drug candidates to arise from BridgeBio’s drug engineering platform.
Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia: Remain on track to report initial data from the ongoing Phase 2 dose ranging study by YE2021. Achondroplasia is the most common form of genetic short stature and one of the most commonly-known genetic diseases, with >55K cases in the US and EU. Low-dose infigratinib is the only known therapy in development for achondroplasia that targets the disease at its genetic source and the only orally administered product candidate in clinical stage development.
Encaleret – CaSR antagonist for Autosomal Dominant Hypocalcemia Type 1 (ADH1): Remain on track to initiate the planned Phase 2 study in 2020, with potential proof-of-concept data available in 2021. If the development program is successful, encaleret would be the first approved therapy for ADH1, a condition caused by gain of function variants in the calcium-sensing receptor gene estimated to be carried by 12k individuals in the US.
BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): IND-enabling studies for AAV gene therapy proceeding. Remain on track to initiate a first in human Phase 1/2 study and report initial data in 2021. CAH is one of the most prevalent genetic diseases thought to be addressable with AAV gene therapy, with >75K cases in the US and EU.
Second quarter 2020 financial results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $840.9 million as of June 30, 2020 compared to $577.1 million at December 31, 2019. The net change in cash balance of $263.8 million reflects $537.0 million in net proceeds received from the issuance of our 2.50% Convertible Senior Notes due 2027, $24.1 million in net proceeds received from Eidos’ at-the-market issuance of shares, offset by payment of $75.0 million to repurchase BridgeBio shares, $49.3 million payment related to capped call option and the remaining payment of $173.0 million primarily related to operating expenses.

Operating Expenses

Operating expenses for the second quarter and first half of 2020 were $124.6 million and $227.1 million, as compared to $69.3 million and $133.1 million, respectively, for the same periods in the prior year. The increases in operating expenses of $55.2 million and $94.0 million during the periods were attributable to the increase in external-related costs and increase in headcount to support the progression in our research and development programs, including our increasing research pipelines, and overall growth of our operations.

Our research and development expenses have not been significantly impacted by the global outbreak of COVID-19 for the periods presented. While we have experienced some initial delays in certain of our clinical enrollment and trial commencement activities, we continue to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at home drug delivery, as well as mitigation strategies with our contract manufacturing organizations. The longer-term impact of COVID-19 on our operating expenses is currently unknown.

On August 11, 2020 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company focused on hospital acute care and gastroenterology reported second quarter 2020 financial results (Press release, Cumberland Pharmaceuticals, AUG 11, 2020, View Source [SID1234563436]). Net revenues from continuing operations during the quarter were $9.6 million. Combined revenues from continued and discontinued operations were $10.3 million, as the company recorded an additional $750,000 in revenue in the second quarter associated with divested product rights.
The Company’s financial position included $98 million in total assets, $49 million of total liabilities, and over $48 million of shareholders’ equity at the end of the quarter.
"As the world continues to adapt to a new way of life during the pandemic, we are working hard to manage our business, secure our supply chain and ensure the ongoing delivery of our products to support patient care during these unprecedented times," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "Our facilities have remained open, and we have continued to operate our business, while working to maintain compliance with the many laws and regulations we fall under as a publicly traded, pharmaceutical company. We continue to make progress toward our goal of building a specialty pharmaceutical business that delivers sustained growth, profitable operations, and long-term value."
RECENT COMPANY DEVELOPMENTS:
Special Acute Care Product Supply Arrangements
During the second quarter, Cumberland implemented three national initiatives to help medical facilities treat patients with conditions associated with COVID-19 infections including high fevers, electrolyte imbalances and pneumonia. These initiatives included the availability of special supply and financial arrangements for the three acute care brands – Caldolor, Vaprisol, and Vibativ during this healthcare emergency.
In addition, the Company also sponsored a national program with infectious disease experts to provide information on the management of complicated respiratory infections resulting from COVID-19.
Vibativ Clinical Manuscripts
During the second quarter, the Company announced a new study published in Drugs – Real World Outcomes, detailing the positive clinical outcomes that resulted from treating patients with bacteremia or endocarditis with Vibativ. This publication is a sub analysis of the Telavancin Observational Use Registry (TOURTM), a study conducted to record population characteristics, prescription information, and real-world clinical outcomes of patients with Gram-positive infections treated with Vibativ. The analysis suggests Vibativ is a promising and viable option for patients with bacteremia or endocarditis, including those with MRSA or another S. aureus pathogen.

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Additionally, Cumberland announced the publication of two studies confirming the continued in vitro potency of telavancin. Both publications were part of continued surveillance of telavancin activity since 2011. The first publication tested a global collection of 24,408 Gram-positive clinical isolates, and the second publication tested a U.S. collection of 15,882 S. aureus isolates. Both studies documented the sustained in vitro antimicrobial activity and spectrum of telavancin-many years after its clinical approval-against Gram-positive clinical isolates collected worldwide over 7 years, from 2011 through 2017.
Vibativ is a patented, FDA-approved injectable anti-infective for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia and complicated skin and skin structure infections. It addresses a range of Gram-positive bacterial pathogens, including those that are considered difficult-to-treat and multidrug-resistant.
Environmental, Social and Governance (ESG) Activities
In April 2020, Cumberland released its inaugural Sustainability Report. This report describes the Company’s activities pertaining to Environmental, Social and Governance matters, otherwise known as corporate sustainability. It includes details about Cumberland’s community involvement, ethical marketing, and drug safety.
The report notes that, during 2019, Cumberland provided nearly 4 million patient doses of products, safely disposed of over 9,700 pounds of expired and damaged products and had no product recalls. The Company also had no product listings on the FDA’s Safety Alerts Database and no products identified in the FDA Adverse Event Reporting System during 2019.
Cumberland’s board appointed Caroline R. Young, former president of the Nashville Health Care Council, as the company’s first ESG board director.
Paycheck Protection Program
At the beginning of the second quarter, Cumberland received the funding of a loan from Pinnacle Bank pursuant to the Paycheck Protection Program (the "PPP") under the Federal Coronavirus Aid, Relief, and Economic Security Act ("CARES Act").
Cumberland applied for this loan after carefully considering the eligibility criteria to participate in this program and determining that the Company met these criteria. Under the terms of the PPP, certain amounts of the loan may be forgiven if they are used for qualifying expenses as described in the CARES Act. The Company intends to use the loan amount for such qualifying expenses.
Cumberland has not laid off or furloughed any employees as a result of the COVID-19 pandemic and, due to assistance from the PPP loan, the Company does not foresee doing so. Cumberland will continue to monitor and evaluate changes to this program as they emerge and will take appropriate action, if necessary.
Ifetroban Phase II Clinical Programs
Enrollment in Cumberland’s clinical studies declined during the second quarter due to the COVID-19 pandemic. While enrollment of new patients is currently limited, the Company is working to ensure that patients already entered into a trial continue to receive their study drug.
Cumberland has completed a pilot Phase II study involving ifetroban in patients suffering from aspirin-exacerbated respiratory disease, a severe form of asthma. A follow-up Phase II study is currently underway for this asthma indication.
The Company is also currently evaluating ifetroban in two pilot Phase II studies in 1) patients with systemic sclerosis or scleroderma, a debilitating autoimmune disorder characterized by diffuse fibrosis of the skin and
internal organs and 2) patients with cardiomyopathy associated with Duchenne Muscular Dystrophy. This rare, fatal, genetic neuromuscular disease results in deterioration of the skeletal, heart and lung muscles.
Cumberland is awaiting further study results before deciding on the best path for approval for ifetroban, its first new chemical entity.
FINANCIAL RESULTS:
Net Revenues: For the three months ended June 30, 2020, net revenues from ongoing operations were $9.6 million, compared to $9.4 million for the prior year period. Combined revenues from continued and discontinued operations during the quarter were $10.3 million as another $750,000 in revenue was recorded associated with product rights that have been divested.
Net revenue by product for the three months ended June 30, 2020, included $3.5 million for Kristalose, $3.3 million for Vibativ, $1.2 million for Caldolor, $0.6 million for Acetadote (including the brand and Company’s Authorized Generic), $0.2 million for Vaprisol, and $0.01 million for Omeclamox-Pak.
Operating Expenses: Total operating expenses for the three months ended June 30, 2020 were $11.2 million, compared to $10.8 million during the prior year period.
Earnings: Net income (loss) for the second quarter 2020 was $(0.9) million or $(0.06) a share, compared to $(0.5) million or $(0.04) a share for the prior year period.
Adjusted Earnings for the second quarter were $1.2 million or $0.08 per diluted share, compared to $0.8 million or $0.05 per diluted share for the prior year period. The definition and reconciliation of Adjusted Earnings to net income is provided in this release.
Balance Sheet: At June 30, 2020, Cumberland had $97.5 million in total assets including $27.4 million in cash and marketable securities. Total liabilities were $49.1 million, including $17.0 million outstanding on the Company’s revolving line of credit, resulting in total shareholder’s equity of $48.5 million.

Conference Call and Webcast
A conference call and live Internet webcast will be held on Tuesday, August 11, at 4:30 p.m. Eastern Time to discuss the results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 2247089. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On August 11, 2020, Replimune Group, Inc. (the "Company") reported that it entered into a Sales Agreement (the "Sales Agreement") with SVB Leerink LLC (the "Agent"), pursuant to which the Company may sell, from time to time, at its option, up to an aggregate of $75,000,000 of shares of the Company’s common stock, $0.001 par value per share (the "Shares"), through the Agent, as the Company’s sales agent (Filing, 8-K, Replimune, AUG 11, 2020, View Source [SID1234563435]).

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Any Shares to be offered and sold under the Sales Agreement will be issued and sold (i) by methods deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended or in negotiated transactions, if authorized by the Company and (ii) pursuant to, and only upon the effectiveness of, a registration statement on Form S-3 filed by the Company with the Securities and Exchange Commission on August 11, 2020 for an offering of up to $350,000,000 of various securities, including shares of the Company’s common stock, preferred stock, debt securities, warrants and/or units for sale to the public in one or more public offerings.

Subject to the terms of the Sales Agreement, the Agent will use reasonable efforts to sell the Shares from time to time, based upon the Company’s instructions (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company cannot provide any assurances that it will issue any Shares pursuant to the Sales Agreement. The Company will pay the Agent a commission of up to 3.0% of the gross proceeds from the sale of the Shares, if any. The Company has also agreed to provide the Agent with customary indemnification rights.

Pursuant to the Sales Agreement, the parties mutually agreed to terminate that certain sales agreement, dated August 8, 2019, by and between the Company and the Agent (as amended, the "2019 Sales Agreement") with respect to the Company’s previous at-the-market offering program (the "2019 ATM Program").

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

The foregoing description of the Sales Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sales Agreement, which is attached hereto as Exhibit 1.1 and is incorporated by reference herein.

On August 11, 2020 Quanterix Corporation (Nasdaq: QTRX), a company digitizing biomarker analysis to advance the science of precision health, reported the closing of its previously announced underwritten public offering of 3,048,774 shares of its common stock at a public offering price of $32.00 per share, including 397,666 shares sold pursuant to the full exercise of the underwriters’ option to purchase additional shares (Press release, Quanterix, AUG 11, 2020, View Source [SID1234563434]). Gross proceeds from the sale of the shares, before deducting underwriting discounts and commissions and offering expenses, were approximately $97.6 million.

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SVB Leerink and Cowen acted as joint bookrunning managers for the offering. Canaccord Genuity acted as co-manager for the offering.

The public offering was made pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC"), as well as a related registration statement on Form S-3MEF filed with the SEC pursuant to Rule 462(b) under the Securities Act of 1933, as amended. The final prospectus supplement and accompanying base prospectus relating to and describing the terms of the offering has been filed with the SEC and is available on the SEC’s website located at View Source, and copies of which can be obtained from SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA, 02110, by telephone at (800) 808-7525, ext. 6218 or by e-mail at [email protected], or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attention: Prospectus Department, by telephone at (833) 297-2926 or by email at [email protected].

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

On August 11, 2020 Context Therapeutics, a clinical-stage biopharmaceutical company dedicated to advancing medicines for hormone driven cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for onapristone extended release (ONA-XR), for the treatment of patients with progesterone receptor positive (PR+) ovarian cancer (Press release, Context Therapeutics, AUG 11, 2020, View Source [SID1234563433]).

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"We are very encouraged by the FDA’s decision to grant us Fast Track Designation for our lead program ONA-XR, which has shown very encouraging activity and tolerability in our ongoing Phase 2 study in advanced PR+ ovarian cancers. We believe this recognition underscores the high unmet medical need for a treatment for patients with PR+ ovarian cancer," said Martin Lehr, Chief Executive Officer of ContextTherapeutics. "With this designation in hand, we plan to be able to quickly advance through the administrative steps of ONA-XR’s development and bring forth a therapy for these patients as soon as possible."

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill unmet medical needs. A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if certain criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a New Drug Application (NDA).

About Onapristone Extended Release
ONA-XR (onapristone extended release) is a potent and specific antagonist of the progesterone receptor that is orally administered. Currently, there are no approved therapies that selectively target PR+ cancers. Preliminary preclinical and clinical data suggest that ONA-XR has anticancer activity by inhibiting progesterone receptor binding to chromatin, downregulating cancer stem cell mobilization and blocking immune evasion. ONA-XR is currently the subject of an ongoing Phase 2 clinical trial in progesterone receptor positive ovarian cancer. Additional Phase 2 clinical trials in breast and endometrial cancers will initiate in 2020. ONA-XR is an investigational drug that has not been approved for marketing by any regulatory authority.