On November 7, 2019 GlycoMimetics, Inc. (Nasdaq: GLYC) reported its financial results for the quarter ended September 30, 2019 and highlighted recent business developments (Press release, GlycoMimetics, NOV 7, 2019, View Source [SID1234550601]). Quarter-end cash and cash equivalents were $170.9 million.

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"In the third quarter of 2019, we continued to progress the late-stage clinical development of our wholly-owned product candidate, uproleselan. Our Company-sponsored Phase 3 trial in relapsed or refractory AML patients and the NCI-sponsored Phase 3 trial for newly diagnosed patients with AML both advanced during the quarter. We are also working with the Duke Cancer Institute towards initiating a single center, proof-of-mechanism Phase 1b trial for GMI-1359, our dual antagonist of E-selectin and CXCR-4, in breast cancer patients with bone metastases," said Rachel King, GlycoMimetics’ Chief Executive Officer.

Ms. King continued, "We are also very much looking forward to the ASH (Free ASH Whitepaper) meeting in December, which has always been an important conference for us, and this year is no different. The key takeaway for us at this year’s ASH (Free ASH Whitepaper) meeting is that data from multiple preclinical and clinical settings show that E-selectin ligand expression on leukemic cells is correlated with poor survival in AML. The data indicate that E-selectin ligand expression is a key driver of environmental-mediated chemoresistance in AML and suggest that uproleselan has the potential to break this chemoresistance, and thereby improve clinical outcomes. Based on this expanding dataset, we are exploring how use of biomarkers may help us in advancing our clinical program.

"Finally, as previously announced, Pfizer reported that its Phase 3 clinical trial evaluating rivipansel in SCD failed to meet its primary endpoint and key secondary endpoints. Of course, this is disappointing, but for some time our operational focus has been on our uproleselan program in AML, and we continue to focus our efforts on diligently and efficiently progressing that exciting clinical program," Ms. King added.

Key Third-Quarter 2019 and Recent Operational Developments:

GlycoMimetics’ pivotal Phase 3 trial of uproleselan in relapsed/refractory AML continued to initiate and activate clinical sites and to enroll patients in the U.S., Australia and now in Europe.
Investigators continued to enroll patients in the NCI-sponsored Phase 3 clinical trial designed to evaluate uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy.
Pfizer announced that the Phase 3 clinical trial evaluating rivipansel in SCD failed to meet the primary endpoint and key secondary endpoints.
As part of a commitment to eliminate certain non-core research and development spending, GlycoMimetics discontinued plans to collaborate with the Haemato Oncology Foundation for Adults in the Netherlands on a Phase 2 trial of uproleselan in newly-diagnosed patients unfit for chemotherapy.
The Company continued to work closely with the Duke Cancer Institute to initiate a Phase 1b proof-of-mechanism clinical trial of GMI-1359 in individuals with breast cancer whose tumors have spread to bone. The trial will evaluate safety and biomarkers of cancer cell mobilization in individuals with hormone receptor positive metastatic breast cancer. The trial will be conducted at Duke University and is expected to initiate during the fourth quarter.
Third Quarter 2019 Financial Results:

Cash position: As of September 30, 2019, GlycoMimetics had cash and cash equivalents of $170.9 million as compared to $209.9 million as of December 31, 2018.
R&D Expenses: The Company’s research and development expenses increased to $10.7 million for the quarter ended September 30, 2019 as compared to $9.7 million for the third quarter of 2018. This increase was primarily the result of expenses relating to the Company’s Phase 3 clinical trial of uproleselan in relapsed or refractory AML patients and supporting the clinical trials of uproleselan conducted by or in collaboration with third parties.
G&A Expenses: The Company’s general and administrative expenses increased to $3.4 million for the quarter ended September 30, 2019 as compared to $2.8 million for the third quarter of 2018. The increase was due to higher patent, legal and non-cash stock-based compensation expenses.
Shares Outstanding: Shares outstanding as of September 30, 2019 were 43,359,949.
The Company will host a conference call and webcast today at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) with passcode 9845948. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.

About Uproleselan (GMI-1271)

Uproleselan (yoo’ pro le’ sel an), currently in a comprehensive Phase 3 development program in AML, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. In a Phase 1/2 clinical trial, uproleselan was evaluated in both newly diagnosed elderly and relapsed or refractory patients with AML. In both populations, patients treated with uproleselan together with standard chemotherapy achieved better-than-expected remission rates and overall survival compared to historical controls, which have been derived from results from third-party clinical trials evaluating standard chemotherapy, as well as lower-than-expected induction-related mortality rates. Treatment in these patient populations was generally well tolerated, with fewer than expected adverse effects.

About GMI-1359

GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules involved in tumor trafficking and metastatic spread. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and multiple myeloma or in solid tumors that metastasize to the bone, such as prostate cancer and breast cancer. GMI-1359 has completed a Phase 1 clinical trial in healthy volunteers. In the fourth quarter of 2019, the Company plans to initiate an exploratory clinical trial in individuals with breast cancer whose tumors have spread to bone.

On November 7, 2019 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported its results for the quarter ended September 30, 2019 (Press release, Corcept Therapeutics, NOV 7, 2019, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-2019-financial [SID1234550631]).

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Financial Highlights

Revenue of $81.5 million, a 26 percent increase from third quarter 2018
GAAP diluted net income of $0.22 per share, compared to $0.14 per share in third quarter 2018
Non-GAAP diluted net income of $0.31 per share, compared to $0.22 per share in third quarter 2018
Cash and investments of $266.9 million, compared to $225.7 million in second quarter 2019
2019 revenue guidance narrowed to $300 – $315 million
Corcept reported quarterly revenue of $81.5 million in the third quarter, compared to $64.4 million in the third quarter of 2018. Third quarter GAAP net income was $26.3 million, compared to $17.7 million in the same period last year. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the third quarter was $37.8 million, compared to $27.9 million in the third quarter of 2018. A reconciliation of GAAP to non-GAAP net income is included below.

The company narrowed 2019 revenue guidance to $300 – $315 million. Guidance had previously been
$285 – $315 million.

Third quarter operating expenses were $48.5 million, compared to $41.5 million in the third quarter of 2018, primarily due to increased spending to recruit and compensate additional personnel and discover and develop new selective cortisol modulators, as well as increased legal expense. Cash and investments were $266.9 million at September 30, 2019, an increase of $41.2 million from June 30, 2019.

"Our Cushing’s syndrome business had an excellent quarter," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "We expect the number of patients receiving Korlym and physicians prescribing the medication to continue to increase. To reach more doctors, we are expanding our sales force. We expect the clinical specialists we are hiring now to begin contributing to our results next year.

"I am also pleased to announce an important advance in our program to treat serious metabolic disorders. In a double-blind, placebo-controlled trial in healthy subjects, our selective cortisol modulator miricorilant significantly reduced the weight gain caused by the commonly prescribed antipsychotic medication olanzapine (Eli Lily’s drug, Zyprexa). We have already initiated one of two planned Phase 2 trials to further test miricorilant’s activity in this indication."

Cushing’s Syndrome

European sites begin dosing patients in Phase 3 trial ("GRACE") of relacorilant to treat patients with Cushing’s syndrome
Double-blind, placebo-controlled, Phase 3 trial of relacorilant in patients whose Cushing’s syndrome is caused by adrenal adenomas to start in the first quarter of next year
"As of today, 42 of 62 planned sites are recruiting patients for GRACE," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "We expect to open an additional 13 sites by the end of the year. The activation pace of ex-US sites, which we expect will provide the majority of enrollments, has refined our estimate of the trial’s completion date. Our plan is to submit our NDA in the fourth quarter of 2021.

We spent substantial time in Europe in the past quarter helping clinical site activation and speaking to investigators. Most important, our investigators are highly enthusiastic about GRACE1, because of relacorilant’s positive Phase 2 efficacy and side effect profile."

Patients in relacorilant’s Phase 2 trial exhibited meaningful improvements in glucose control and hypertension – two of Cushing’s syndrome’s most common and pernicious symptoms. The trial also met a wide range of secondary endpoints, including weight loss, liver function, coagulopathy, insulin resistance, cognitive function, mood and quality of life. These results were achieved without relacorilant causing Korlym’s significant off-target effects – vaginal bleeding, endometrial thickening and low potassium.2

In addition to GRACE, Corcept plans to start a Phase 3, double-blind, placebo-controlled trial of relacorilant in patients whose Cushing’s syndrome is caused by an adrenal adenoma – a population that has not been rigorously studied. Patients with adrenal Cushing’s syndrome typically experience a slower onset of symptoms, but their ultimate health outcomes are poor. Corcept expects to enroll 130 patients at sites in the United States and Europe in the study. Most of the planned investigators and sites are also participating in GRACE.

Metabolic Disease

Positive top-line results from double-blind, placebo-controlled, Phase 1b trial of miricorilant to reduce antipsychotic-induced weight gain
Recruiting underway in double-blind, placebo-controlled, Phase 2 trial of miricorilant to reverse recent antipsychotic-induced weight gain
"Our program to develop miricorilant as a treatment for metabolic disorders is off to an excellent start," said Dr. Grauer. "Antipsychotic medications such as olanzapine are essential to the health of millions of patients, but the weight gain and other metabolic side effects they cause are life-threatening and often lead patients to discontinue treatment. At the first dose level tested in our Phase 1b trial, healthy subjects given olanzapine plus miricorilant gained less weight than subjects receiving olanzapine plus placebo (see Figure 1). In addition, markers of liver damage that often rise temporarily at the start of olanzapine therapy increased less sharply in subjects receiving miricorilant, suggesting that miricorilant may have protective effects in the liver (see Figure 2). Five subjects in the olanzapine alone group were unable to complete the study due to elevated liver enzymes, while one patient in the miricorilant group experienced this problem."

A photo accompanying this announcement is available at View Source

The Phase 1b trial’s first part enrolled 66 healthy subjects, each of whom received olanzapine (10 mg) and either miricorilant (600 mg) or placebo daily. The trial’s duration was two weeks. The second part of the trial, which is planned to start in December, will test a higher dose of miricorilant (900 mg) in 30 healthy subjects. The study’s full results will be presented at a scientific meeting in 2020.

"These preliminary results are especially encouraging given the short duration of treatment and the low dose of miricorilant. They are consistent with the effects we had previously seen in animal studies. Our plan is to confirm these findings and explore the full breadth of miricorilant’s activity," said Dr. Grauer.

In addition to the second part of its Phase 1b trial, Corcept plans to conduct two double-blind, placebo-controlled Phase 2 trials of miricorilant for the treatment of patients with antipsychotic-induced weight gain. The first trial, which is underway, will test miricorilant’s activity in reversing recent weight gain. It is expected to enroll 100 patients with schizophrenia at 20 sites in the United States. Patients will continue to receive their established antipsychotic medication and will have either miricorilant or placebo added to their regimen for 12 weeks. A second Phase 2 trial is planned to start next year. It will enroll patients with long-standing weight gain. A third Phase 2 trial, testing miricorilant’s activity in preventing antipsychotic-induced weight gain, is under consideration.

Next year, Corcept also plans to start a double-blind, placebo-controlled, Phase 2 trial of miricorilant as a treatment for patients with non-alcoholic steatohepatitis (NASH), a serious liver disorder that afflicts millions of people.

Solid Tumors

European Commission designates relacorilant orphan drug for treatment of pancreatic cancer
Phase 3 trial of relacorilant plus nab-paclitaxel in patients with metastatic pancreatic cancer to start upon completion of consultations with the U.S. Food and Drug Administration (FDA)
"We are pleased the European Commission (EC) has joined the FDA in designating relacorilant an orphan drug for the treatment of pancreatic cancer," said Dr. Grauer. "The EC based its decision on the European Medicines Agency’s finding that relacorilant has the potential to significantly benefit patients.

"We presented the clinical data reviewed by the EMA at last year’s ASCO (Free ASCO Whitepaper) meeting and it was indeed promising," said Dr. Grauer. "Seven of 25 patients with metastatic pancreatic cancer treated with relacorilant plus nab-paclitaxel (Celgene’s drug, Abraxane) achieved durable disease control, meaning their tumors either shrank or ceased growing for 16 weeks or longer. Tumor response in two patients lasted more than 50 weeks.3 All of these patients’ tumors had progressed during multiple lines of prior therapy, including treatments with nab-paclitaxel or another taxane. That any of them responded is remarkable. We have sought FDA guidance as to the optimum development path in pancreatic cancer and plan to start a Phase 3 trial promptly upon the conclusion of our discussions."

Corcept’s 180-patient, placebo-controlled Phase 2 trial of relacorilant plus nab-paclitaxel in ovarian cancer continues to enroll patients at sites in the United States and the European Union. Dosing also continues in the company’s Phase 1/2 study of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer.

Conference Call

We will hold a conference call on November 7, 2019, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, dial 1-877-260-1479 from the United States or 1-334-323-0522 internationally approximately ten minutes before the start of the call (passcode 8532239). A replay will be available through November 21, 2019 at 1-888-203-1112 in the United States and 1-719-457-0820 internationally (passcode 8532239).

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system in the body and can be lethal if not treated effectively.

On November 7, 2019 Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) reported its interim financial results in line with guidance for the first nine months of 2019 and business progress for the third quarter of 2019 (Press release, Bavarian Nordic, NOV 7, 2019, View Source [SID1234550647]).

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Paul Chaplin, President & Chief Executive Officer of Bavarian Nordic said: "We set out the year with a clear ambition and strategy to expand our commercial footprint, and with the recently announced acquisition of two commercial vaccines from GlaxoSmithKline (GSK), we turn to the next chapter for Bavarian Nordic to become an independent vaccine company with sustainable profitability. The company is fully focused on the integration of manufacturing of the two new vaccines and establishing the commercial operations to make this a great success. We will move ahead with our planned Phase 3 trial for RSV in 2021 that allows sufficient time to establish an improved commercial scale production and formulation to meet the demands of this blockbuster indication. The recent approval of JYNNEOS was also a tremendous milestone for the Company, offering exciting new business opportunities beyond our long-term partnership with the U.S. government to supply the national stockpile and offering clear synergies with the two acquired commercial vaccines."

TRANSFORMATIVE ACQUISITION OF TWO COMMERCIAL VACCINES FROM GLAXOSMITHKLINE
Announced on October 21, the acquisition of Rabipur/RabAvert and Encepur from GlaxoSmithKline (GSK) is a transformative transaction that will establish a leading infectious disease franchise and make Bavarian Nordic an independent and profitable vaccine company. Specifically, the transition:

Accelerates our vision by 3 years to become a leading and profitable vaccine company
Exploits significant manufacturing synergies between highly complementary technologies and builds on our expertise
Ensures strong cash flow generation, allowing us to continue progressing our promising innovative pipeline
Full details on the acquisition were published in company announcement no. 19/2019.

Closing of the acquisition is expected by December 31, 2019, conditional on antitrust approval as well as on approval of the rights issue by the Company’s shareholders. To resolve on the rights issue, an extraordinary general meeting has been convened on November 27, 2019. Details are available at www.bavarian-nordic.com/egm.

OPERATIONAL HIGHLIGHTS

Delivering our strategy
We set out for the year with a 5-year vision and four strategic imperatives that collectively build on our heritage in vaccine development and manufacturing, aiming to secure and reinforce a sustainable foundation, while also expanding the commercial opportunities. With the acquisition of two commercial vaccines from GSK, the recent approval of JYNNEOS and the advancement of several clinical programs during the year, we are on track to accomplish these targets:

MAINTAIN global leadership of our smallpox vaccine business
EXPAND and rapidly ADVANCE the pipeline of infectious disease programs
ESTABLISH a broad and deep cancer immunotherapy portfolio
EXPAND the commercial footprint and capabilities
Smallpox

In September, the U.S. Food and Drug Administration (FDA) approved JYNNEOS (MVA-BN) for protection against smallpox and monkeypox. The approval was the result of our successful, fifteen-year partnership with the U.S. government to develop a non-replicating smallpox vaccine, suitable for the entire population, presenting significant opportunities for our future business with the U.S. government beyond emergency stockpiling.
As expected, we were awarded a Priority Review Voucher along with the approval.
The additional indication for monkeypox provides new commercial opportunities to be exploited in the coming years as we expand the commercial infrastructure to accommodate sales and distribution of the vaccines acquired from GSK.
A Phase 3 lot-consistency trial, fully funded by BARDA, of the freeze-dried formulation of MVA-BN is ongoing. A prior Phase 2 study showed bioequivalence between the freeze-dried and liquid-frozen formulations of MVA-BN, and the lot-consistency trial was agreed with the FDA as the only Phase 3 study required to support licensure of the freeze-dried formulation, which is anticipated in 2022.
We are on track with the production of bulk vaccine for the current order for freeze-dried MVA-BN from the U.S. Government with the majority of the batches being invoiced in the fourth quarter of 2019 adding up to a total of USD 50 million included in our guided 2019 revenues.
Infectious diseases

The design of the Phase 3 efficacy trial, for our highly promising RSV vaccine candidate for the elderly (≥60 years old), has been finalized with the FDA. The randomized, placebo-controlled trial has an adaptive design and will enroll a total of 12,000 to 14,000 subjects over two seasons. The total number of subjects will depend on the independent analysis performed from the first 6,000 subjects enrolled for the first season. The estimated costs to determine futility after the season one will be USD 40 million and if positive, the second season will cost an additional USD 50-70 million, although after passing the first season threshold there would be approximately a 75% chance of successfully reaching the efficacy endpoint of the trial. The trial is planned to be initiated in 2021 prior to the RSV season, with the initial read out in 2022 and a potential approval in 2024.
In October, we initiated a Phase 1 clinical trial of MVA-BN WEV, a trivalent prophylactic vaccine against three strains of the equine encephalitis virus: Western, Eastern (also referred to as triple E) and Venezuelan. The current program is funded by the U.S. Department of Defense. Equine encephalitis virus is an alpha virus that is spread by mosquitos from infected birds to horses and people that can result in the rare condition of encephalitis in about 5% of the people that become infected. Since October, 31 cases, including 9 deaths have been reported by the US CDC in what is the largest ever recorded outbreak of triple E. Successful Phase 1 results could lead to additional funding of clinical development towards licensure of this important emerging infectious disease.
Our Janssen partnership provides an opportunity to explore and commercialize our MVA-BN platform in three infectious disease indications; HPV, HIV and HBV. A Phase 1/2a trial of the therapeutic HPV vaccine was initiated earlier this year and the first clinical trial of the therapeutic HIV vaccine is anticipated to begin in 2020. Collectively, these programs, along with our Ebola collaboration, represent USD 1 billion in potential future milestone payments, in addition to royalties on future sales.
Ebola continues to rage in the Democratic Republic of Congo, and Janssen recently announced the donation of up to 500,000 doses of the Ad26.ZEBOV/MVA-BN Filo vaccine regimen, which will be introduced to help counter the outbreak. Neighboring countries like Rwanda and Uganda have also expressed their intent to use the vaccine as Ebola cases have been reported around the border. In Uganda, a large trial of the vaccine was initiated in healthcare workers in August. The vaccine regimen has to-date been evaluated in more than 6,500 individuals in Phase 1, 2 and 3 clinical studies, and Janssen is planning to submit a Marketing Authorization Application to the European Medicines Agency this year.
Cancer immunotherapy

In October, we updated on the development of CV301, and the study in bladder cancer, which did not meet the efficacy threshold to expanded enrollment for the second stage. While we continue to follow and support two ongoing, investigator-sponsored Phase 2 trials evaluating CV301 in combination with checkpoint inhibitors in colorectal and pancreatic cancer, the Company’s focus moves to BN-Brachyury and the intravenous and intra-tumoral applications.
BN-Brachyury is currently being evaluated in a Phase 2 trial in patients with advanced chordoma. This trial, which also uses a two-stage design, indicated clinical activity in the first stage, allowing for expansion of enrollment, which was recently completed. Conclusive data from this study are expected within 12 months.
A planned Phase 1 dose escalation study at the National Cancer Institute (NCI) investigating the safety of MVA-BN Brachyury administered intravenously into patients with brachyury positive tumors will initiate shortly.
Commercial manufacturing

Construction of our fill and finish facility is progressing according to plan, and we expect the building to be completed by end of 2019, which is the peak investment year with approximately DKK 270 million in total investments.
The addition of a large-scale fill and finish line to our existing bulk production will significantly expand our manufacturing capabilities, allowing for integration of the products from GSK, while also enabling us to continuously support our partnerships, including fulfilling our smallpox vaccine contract with the U.S. government.
To support simultaneous manufacturing of multiple products, we will invest in a new, independent clean room suite at our existing facility to be initiated in 2020.
FINANCIALS AND OUTLOOK

Financial results for the first nine months in line with expectations

Revenue generated for the nine months ending September 30, 2019 was DKK 372 million/USD 54 million (DKK 319 million/USD 47 million in the first nine months of 2018).
The income before interest and tax (EBIT) was a loss of DKK 298 million/USD 43 million (loss of DKK 261 million/USD 38 million in the first nine months of 2018).
As of September 30, 2019, the Group’s cash preparedness was DKK 1,740 million/USD 254 million (DKK 2,314 million/USD 337 million as of December 31, 2018), including unutilized credit lines of DKK 244 million/USD 36 million (DKK 244 million/USD 36 million as of December 31, 2018).
Danish kroner (DKK) is the Company’s reporting currency. The USD figures provided above are based upon an assumed exchange rate of DKK 6.86 per 1.00 USD, which was the exchange rate as of September 30, 2019.

Outlook for 2019 maintained
The majority of our 2019 revenues are related to the production and release of bulk smallpox vaccine for the U.S. Government, most of which will occur in the fourth quarter of 2019. Hence, we maintain our financial expectations for 2019 as announced on March 21, 2019 with revenues of approximately DKK 600 million/USD 92 million for the full year and a loss before interest and tax (EBIT) of approximately DKK 360 million/USD 55 million. The expected cash preparedness at year-end was revised in October from approximately DKK 1,600 million/USD 246 million to approximately DKK 1,000 million/USD 154 million as a result of the announced acquisition of two vaccines from GlaxoSmithKline.

A detailed guidance for 2020 will be provided in connection with the publication of the Company’s annual report for 2019. However, pending a successful closing of the transaction with GSK, the Company is able to confirm that it will be profitable on an EBITDA basis already in 2020, exclusive of the sale of the Priority Review Voucher, which was awarded the Company by FDA upon the approval of JYNNEOS in September.

The financial expectations are based on an exchange rate of DKK 6.50 per 1.00 USD. For further details regarding assumptions behind the guidance see the Annual Report 2018.

Financial calendar 2019/2020
The date for announcement of the 2019 annual report has been changed to February 20, 2020.

November 27, 2019 Extraordinary General Meeting *
February 20, 2020 2019 Annual Report
April 21, 2020 Annual General Meeting **
May 14, 2020 First quarterly report (Q1) for the three-month period ended March 31, 2020
August 26, 2020 Half-year report (Q2) for the six-month period ended 30 June 2020
November 11, 2020 Third quarterly report (Q3) for the nine-month period ended 30 September 2020
* An extraordinary general meeting has been convened to seek shareholder approval to increase the Company’s share capital through a planned rights issue in the first half of 2020 with pre-emptive rights for the existing shareholders in order to obtain financing of costs associated with the acquisition of Rabipur/RabAvert and Encepur. See www.bavarian-nordic.com/egm for additional information.

** Pursuant to Article 12 of the Articles of Association, shareholders who wish to submit a request for proposals for consideration at the annual general meeting must lodge this with the Company no later than Wednesday, March 11, 2020.

OTHER DEVELOPMENTS

Incentive programs for employees and executive management in Bavarian Nordic
The board of directors has today decided to issue warrants to executive management and certain employees in the Bavarian Nordic Group. The decision is made in accordance with the shareholder authorization for the board of directors adopted as Article 5b of the Articles of Association and the Company’s guidelines regarding incentive programs.

The warrant program entails the issuance of 564,585 warrants in total which entitle the warrant holders to subscribe for up to 564,585 shares in total with a nominal value of DKK 10 each at an exercise price of DKK 185.4 per share. The warrants may be exercised wholly or partly during eight fixed subscription periods during 2023 and 2024.

The value of each warrant equals DKK 45.5 and is calculated on the Black-Scholes model with a risk-free interest rate of -0.69 per cent and on the historical volatility of the shares. The calculation is based on a share price of DKK 154.05.

Furthermore, the Company introduces a three-year incentive program in January 2020 for all employees in the Bavarian Nordic Group, with the exception of employees receiving warrants. The program is a cash bonus program based on the development in the Company’s share price. The incentive program will not have a dilutive effect on the shareholders.

Each employee participating in the program is awarded so-called phantom shares every month of employment until 31 December 2022. The exercise price is DKK 185.4. The phantom shares may be exercised in January 2023, only if the Company’s share price by then exceeds the exercise price by at least DKK 5. In that case, each phantom share will yield a cash bonus equivalent to DKK 1 per point the share price exceeds the exercise price.

Based on the current number of employees in the Group eligible for participating in the program, the program will comprise up to 72,288 phantom shares. The average value of each phantom share granted equals DKK 27.7 calculated on the basis of the Black-Scholes model with a risk-free interest rate of -0.69 per cent and on the historical volatility of the shares. The calculation is based on a share price of DKK 154.05.

Conference call and webcast
The management of Bavarian Nordic will host a conference call today at 2 pm CET (8 am ET) to present the interim results followed by a Q&A session. A listen-only version of the call can be accessed via View Source To join the Q&A session, use one of the following dial-in numbers: Denmark: +45 32 72 80 42, UK: +44 (0) 844 571 8892, USA: +1 631-510-7495. Participant code is 8772778.

On November 7, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended September 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, NOV 7, 2019, View Source [SID1234550663]).

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"In the third quarter, we made meaningful progress towards our clinical and regulatory milestones," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We received IMPD and IND clearance for our global Phase 1 trial for PKD, treated our first patient for LAD-I and continued enrollment of our Phase 1 trial of RP-A501 for Danon disease. We also announced promising long-term data from our Phase 1/2 trial of first-generation RP-L102 for Fanconi Anemia supporting sustained clinical improvement with robust engraftment exceeding the 10% threshold agreed to by regulators for the upcoming global Phase 2 trial. We hope to further optimize these responses with our ‘Process B’ of RP-L102 designed to enable consistent results with commercial-grade product without cytotoxic conditioning. We look forward to data from ‘Process B’ later this year, along with initial Phase 1 data from our RP-L201 trial for LAD-I."

Dr. Shah continued, "We now have four gene therapy candidates in the clinic, meeting one of our 2019 corporate goals and reinforcing our multi-platform strategy. As we advance our first candidate into late-stage development, we remain dedicated to our pursuit of potentially curative treatments for patients contending with rare genetic diseases."

Recent Pipeline Developments

Commencement of registration-enabling Phase 2 study for Fanconi Anemia (FA). Patient enrollment is ongoing in the global Phase 2 trial. The study initiation follows recent alignment from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) on the trial design and the primary endpoint. Resistance to mitomycin-C, a DNA damaging agent, in bone marrow stem cells at a minimum time point of one year will serve as the primary endpoint and potentially as a surrogate endpoint for accelerated approval. Patient dosing is anticipated to begin in the fourth quarter with preliminary Phase 2 data anticipated in 2020.
Long-term Phase 1/2 clinical data of RP-L102 for FA "Process A" presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress. Long-term patient follow-up data demonstrate evidence of increasing and durable engraftment leading to bone marrow restoration exceeding the 10% threshold agreed to by the FDA and EMA for the ongoing registration-enabling Phase 2 trial. In patient 02002, who received what we consider adequate drug product, hemoglobin levels are now similar to those in the first year after birth, suggesting hematologic correction over the long term. Preliminary results from the first two patients receiving "Process B" of RP-L102 are anticipated in the fourth quarter.
Global Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD) initiates enrollment. Rocket received Investigational Medicinal Product Dossier (IMPD) clearance and Investigational New Drug (IND) application approval of RP-L301 during the quarter. The open-label, single-arm, clinical trial will enroll six adult and pediatric transfusion-dependent PKD patients in the U.S. and Europe. The trial is designed to assess the safety, tolerability and preliminary efficacy of RP-L301. Preliminary data are anticipated in 2020.
Patient dosing continues in Phase 1/2 registrational trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. The Phase 2 portion of the trial will evaluate overall survival. Preliminary Phase 1 data will be presented in the fourth quarter.
Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates reporting Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
Anticipated Milestones

FA (RP-L102)
Global Phase 2 trial first patient treatment (4Q19)
Initial Phase 1 data for "Process B" (4Q19)
Additional Phase 1 "Process B" data (1H20)
Preliminary Phase 2 data (2H20)
Danon Disease (RP-A501)
Phase 1 data (2020)
LAD-I (RP-L201)
Initial Phase 1 data (4Q19)
Phase 1 data update (1H20)
Commence enrollment of Phase 2 study (2H20)
PKD (RP-L301)
Preliminary Phase 1 data (2H20)
IMO (RP-L401)
Initiation of clinical study (2H20)
Upcoming Investor Conferences

Barclays Gene Editing & Gene Therapy Summit—November 13, 2019 in New York, N.Y.
Evercore ISI 2nd Annual HealthCONx Conference—December 3, 2019 in Boston, M.A.
Piper Jaffray’s 31st Annual Healthcare Conference—December 5, 2019 in New York, N.Y.
Third Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of September 30, 2019, were $240.6 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $14.8 million for the three months ended September 30, 2019, compared to $13.1 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in clinical trial costs of $1.3 million.
G&A expenses. General and administrative expenses were $4.3 million for the three months ended September 30, 2019, compared to $2.3 million for the three months ended September 30, 2018. The increase was primarily driven by an increase in non-cash stock compensation expense of $1.4 million and an increase in compensation and benefits expense of $0.3 million as a result of increased headcount.
Net loss. Net loss was $19.3 million or $0.38 per share (basic and diluted) for the three months ended September 30, 2019, compared to $16.1 million or $0.40 per share (basic and diluted) for the three months ended September 30, 2018.
Shares outstanding. 50,376,030 shares of common stock were outstanding as of September 30, 2019.
Financial Guidance

Cash position. As of September 30, 2019, we had cash, cash equivalents and investments of $240.6 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.

On November 7, 2019 MedX Health Corp. ("MedX" or the "Company") (TSX-V:MDX) reported that, pursuant to a non-brokered Private Placement Offering (the "Placement Offering"), it has completed an initial Closing of 1,251,000 Units effective November 4, 2019, to raise $150,120 from accredited investors pursuant to a Private Placement Offering announced on November 1, 2019 (Press release, MedX Health, NOV 7, 2019, View Source [SID1234550679]). Each Unit, priced at $0.12, comprises one fully paid common share and one share purchase warrant; each warrant entitles the holder to purchase one additional share at a price of $0.20 during the period up to two years from the closing date of the placement. The securities issued as a result of the Placement Offering will be restricted from trading for four months following each Closing. The Placement Offering is still open to subscribers, and further subscriptions may be accepted for further Closings. This Closing, and any further Closing of the Placement Offering, is subject to all relevant regulatory and other consents and approvals, including acceptance by the TSX Venture Exchange. In connection with the first Closing, a cash commission of $5,136 was paid and 42,800 Agent’s Warrants were issued.

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Following this Closing, MedX has 143,169,762 common shares issued and outstanding.