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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Global Blood Therapeutics Inc | NASDAQ:GBT | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
0.00 | 0.00% | 68.49 | 68.47 | 68.50 | 0 | 01:00:00 |
☒ |
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
☐ |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934
|
Delaware
|
27-4825712
|
|
(State or other jurisdiction of
incorporation or organization)
|
(I.R.S. Employer
Identification No.)
|
Title of each class
|
|
Trading
Symbol(s)
|
|
Name of each exchange
on which registered
|
Common Stock, par value $0.001 per share
|
|
GBT
|
|
The NASDAQ Global Select Market
|
|
Large accelerated filer
|
☒
|
Accelerated filer
|
☐
|
|||
Non-accelerated
filer
|
☐
|
Smaller reporting company
|
☐
|
|||
|
|
Emerging growth company
|
☐
|
|
|
|
|
Page
|
|
|
|
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3
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Item 1.
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3
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3
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4
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5
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6
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7
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Item 2.
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15
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Item 3.
|
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22
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Item 4.
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22
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||
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22
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|||
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Item 1.
|
|
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22
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Item 1A.
|
|
|
|
22
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|
Item 2.
|
|
|
|
59
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|
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Item 3.
|
|
|
|
59
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|
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Item 4.
|
|
|
|
59
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Item 5.
|
|
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|
59
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Item 6.
|
|
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|
59
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|
|
|
|
|
|
|
||
|
|
61
|
|
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
|
(Unaudited)
|
|
|
|
||||
Assets
|
|
|
|
|
|
|
||
Current assets:
|
|
|
||||||
Cash and cash equivalents
|
$ |
315,525
|
$ |
302,237
|
||||
Short-term marketable securities
|
215,295
|
307,732
|
||||||
Accounts receivable, net
|
4,578
|
2,637
|
||||||
Inventories
|
14,927
|
1,277
|
||||||
Prepaid expenses and other current assets
|
14,144
|
14,114
|
||||||
Total current assets
|
564,469
|
627,997
|
||||||
Property and equipment, net
|
37,329
|
27,113
|
||||||
Long-term marketable securities
|
84,378
|
85,030
|
||||||
Operating lease
right-of-use
assets
|
52,082
|
52,775
|
||||||
Restricted cash
|
2,395
|
2,395
|
||||||
Other assets, noncurrent
|
493
|
789
|
||||||
Total assets
|
$ |
741,146
|
$ |
796,099
|
||||
Liabilities and Stockholders’ Equity
|
|
|
|
|
|
|
||
Current liabilities:
|
|
|
||||||
Accounts payable
|
$ |
13,696
|
$ |
10,621
|
||||
Accrued liabilities
|
34,562
|
41,358
|
||||||
Accrued compensation
|
11,269
|
17,578
|
||||||
Other liabilities, current
|
2,721
|
1,896
|
||||||
Total current liabilities
|
62,248
|
71,453
|
||||||
Long-term debt
|
73,688
|
73,559
|
||||||
Operating lease liabilities, noncurrent
|
81,638
|
72,359
|
||||||
Other liabilities, noncurrent
|
—
|
34
|
||||||
Total liabilities
|
217,574
|
217,405
|
||||||
Commitments and contingencies
|
|
|
||||||
Stockholders’ equity:
|
|
|
||||||
Preferred stock, $0.001 par value, 5,000,000 shares authorized as of March 31, 2020
(
and December 31, 2019; no shares issued and outstanding
unaudited
)
|
—
|
—
|
||||||
Common stock, $0.001 par value, 150,000,000 shares authorized as of March 31, 2020 (unaudited) and December 31, 2019, respectively; 60,886,371 and 60,644,380 shares issued and outstanding as of March 31, 2020 (unaudited) and December 31, 2019, respectively
|
61
|
61
|
||||||
Additional
paid-in
capital
|
1,334,238
|
1,316,795
|
||||||
Accumulated other comprehensive income
|
1,215
|
754
|
||||||
Accumulated deficit
|
(811,942
|
) |
(738,916
|
) | ||||
Total stockholders’ equity
|
523,572
|
578,694
|
||||||
Total liabilities and stockholders’ equity
|
$ |
741,146
|
$ |
796,099
|
||||
|
Three Months Ended March 31,
|
|||||||
|
2020
|
|
2019
|
|
||||
Product sales, net
|
$ |
14,118
|
$ |
—
|
||||
Costs and operating expenses:
|
|
|
||||||
Cost of sales
|
135
|
—
|
||||||
Research and development
|
39,773
|
34,468
|
||||||
Selling, general and administrative
|
47,662
|
18,055
|
||||||
Total costs and operating expenses
|
87,570
|
52,523
|
||||||
Loss from operations
|
(73,452
|
) |
(52,523
|
) | ||||
Other income (expense):
|
|
|
||||||
Interest income
|
2,856
|
3,831
|
||||||
Interest expenses
|
(2,314
|
) |
(181
|
) | ||||
Other expenses, net
|
(116
|
) |
(50
|
) | ||||
Total other income, net
|
426
|
3,600
|
||||||
Net loss
|
(73,026
|
) |
(48,923
|
) | ||||
Other comprehensive income:
|
|
|
||||||
Net unrealized gain on marketable securities, net of tax
|
461
|
624
|
||||||
Comprehensive loss
|
$ |
(72,565
|
) | $ |
(48,299
|
) | ||
Basic and diluted net loss per common share
|
$ |
(1.20
|
) | $ |
(0.87
|
) | ||
Weighted-average number of shares used in computing basic and diluted net loss per common share
|
60,787,710
|
56,231,587
|
||||||
|
Common Stock
|
Additional
Paid-
In Capital
|
|
Accumulated
Other
Comprehensive
Income
|
|
Accumulated
Deficit
|
|
Total
Stockholders’
Equity
|
|
|||||||||||||||
|
Shares
|
|
Amount
|
|
||||||||||||||||||||
Balance at December 31, 2019
|
60,644,380
|
$ |
61
|
$ |
1,316,795
|
$ |
754
|
$ |
(738,916
|
) | $ |
578,694
|
||||||||||||
Issuance of common stock upon exercise of stock
options |
33,937
|
—
|
967
|
—
|
—
|
967
|
||||||||||||||||||
Issuance of common stock upon vesting of restricted
share units, net of shares withheld for employee taxes |
160,594
|
—
|
(2,099
|
) |
—
|
—
|
(2,099
|
) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases
|
47,460
|
—
|
1,870
|
—
|
—
|
1,870
|
||||||||||||||||||
Stock-based compensation expense
|
—
|
—
|
16,705
|
—
|
—
|
16,705
|
||||||||||||||||||
Net unrealized gain on marketable securities
|
—
|
—
|
—
|
461
|
—
|
461
|
||||||||||||||||||
Net loss
|
—
|
—
|
—
|
—
|
(73,026
|
) |
(73,026
|
) | ||||||||||||||||
Balance at March 31, 2020
|
60,886,371
|
$ |
61
|
$ |
1,334,238
|
$ |
1,215
|
$ |
(811,942
|
) | $ |
523,572
|
||||||||||||
|
Common Stock
|
Additional
Paid-
In Capital
|
|
Accumulated
Other
Comprehensive
Income
|
|
Accumulated
Deficit
|
|
Total
Stockholders’
Equity
|
|
|||||||||||||||
|
Shares
|
|
Amount
|
|
||||||||||||||||||||
Balance at December 31, 2018
|
55,640,299
|
$ |
56
|
$ |
1,044,941
|
$ |
(48
|
) | $ |
(472,150
|
) | $ |
572,799
|
|||||||||||
Issuance of common stock upon equity offerings, net
of issuance costs |
511,363
|
—
|
21,246
|
—
|
—
|
21,246
|
||||||||||||||||||
Issuance of common stock upon exercise of stock
options |
52,288
|
—
|
817
|
—
|
—
|
817
|
||||||||||||||||||
Issuance of common stock upon vesting of restricted
share units, net of shares withheld for employee taxes |
78,155
|
—
|
(686
|
) |
—
|
—
|
(686
|
) | ||||||||||||||||
Issuance of common stock pursuant to ESPP purchases
|
30,745
|
—
|
1,128
|
—
|
—
|
1,128
|
||||||||||||||||||
Vesting of restricted stock purchases
|
24,195
|
—
|
80
|
—
|
—
|
80
|
||||||||||||||||||
Stock-based compensation expense
|
—
|
—
|
9,453
|
—
|
—
|
9,453
|
||||||||||||||||||
Net unrealized gain on marketable securities
|
—
|
—
|
—
|
624
|
—
|
624
|
||||||||||||||||||
Net loss
|
—
|
—
|
—
|
—
|
(48,923
|
) |
(48,923
|
) | ||||||||||||||||
Balance at March 31, 2019
|
56,337,045
|
$ |
56
|
$ |
1,076,979
|
$ |
576
|
$ |
(521,073
|
) | $ |
556,538
|
||||||||||||
|
March 31, 2020
|
|||||||||||||||
|
Total
|
|
Level 1
|
|
Level 2
|
|
Level 3
|
|
||||||||
Financial Assets:
|
|
|
|
|
||||||||||||
Money market funds
|
$
|
294,863
|
$
|
294,863
|
$ |
—
|
$ |
|
||||||||
Corporate debt securities
|
118,390
|
—
|
118,390
|
—
|
||||||||||||
U.S. government agency securities
|
79,579
|
—
|
79,579
|
—
|
||||||||||||
Certificates of deposits
|
5,808
|
—
|
5,808
|
—
|
||||||||||||
U.S. government securities
|
95,896
|
—
|
95,896
|
—
|
||||||||||||
Total financial assets
|
$ |
594,536
|
$ |
294,863
|
$ |
299,673
|
$ |
—
|
||||||||
|
December 31, 2019
|
|||||||||||||||
|
Total
|
|
Level 1
|
|
Level 2
|
|
Level 3
|
|
||||||||
Financial Assets:
|
|
|
|
|
||||||||||||
Money market funds
|
$
|
250,535
|
$
|
250,535
|
$ |
—
|
$ |
|
||||||||
Corporate debt securities
|
152,149
|
—
|
152,149
|
—
|
||||||||||||
U.S. government agency securities
|
95,032
|
—
|
95,032
|
—
|
||||||||||||
Certificates of deposits
|
6,282
|
—
|
6,282
|
—
|
||||||||||||
U.S. government securities
|
140,244
|
—
|
140,244
|
—
|
||||||||||||
Total financial assets
|
$ |
644,242
|
$ |
250,535
|
$
|
393,707
|
$ |
—
|
||||||||
|
March 31, 2020
|
December 31, 2019
|
||||||||||||||||||||||||||||||
|
Amortized
Cost
|
|
Unrealized
Gains
|
|
Unrealized
(Losses)
|
|
Estimated Fair
Value
|
|
Amortized
Cost
|
|
Unrealized
Gains
|
|
Unrealized
(Losses)
|
|
Estimated Fair
Value
|
|
||||||||||||||||
Financial Assets:
|
|
|
|
|
|
|
|
|
||||||||||||||||||||||||
Money market funds
|
$
|
294,863
|
$ |
—
|
$ |
—
|
$ |
294,863
|
$
|
250,535
|
$ |
—
|
$ |
—
|
$ |
250,535
|
||||||||||||||||
Corporate debt securities
|
118,187
|
287
|
(84
|
) |
118,390
|
151,773
|
384
|
(8
|
) |
152,149
|
||||||||||||||||||||||
U.S. government agency securities
|
79,417
|
181
|
(19
|
) |
79,579
|
94,963
|
73
|
(4
|
) |
95,032
|
||||||||||||||||||||||
Certificates of deposits
|
5,766
|
42
|
—
|
5,808
|
6,239
|
43
|
—
|
6,282
|
||||||||||||||||||||||||
U.S. government securities
|
95,088
|
808
|
—
|
95,896
|
139,978
|
266
|
—
|
140,244
|
||||||||||||||||||||||||
Total
|
$ |
593,321
|
$ |
1,318
|
$ |
(103
|
) | $ |
594,536
|
$ |
643,488
|
$ |
766
|
$ |
(12
|
) | $ |
644,242
|
||||||||||||||
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
Cash and cash equivalents
|
$ |
294,863
|
$ |
251,480
|
||||
Short-term marketable securities
|
215,295
|
307,732
|
||||||
Long-term marketable securities
|
84,378
|
85,030
|
||||||
Total
|
$ |
594,536
|
$ |
644,242
|
||||
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
Raw materials
|
$ |
7,141
|
$ |
700
|
||||
Work-in-process
|
7,217
|
525
|
||||||
Finished goods
|
569
|
52
|
||||||
Total inventories
|
$ |
14,927
|
$ |
1,277
|
||||
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
Laboratory equipment
|
$ |
8,472
|
$ |
8,314
|
||||
Computer equipment
|
2,224
|
2,224
|
||||||
Leasehold improvements
|
13,785
|
13,785
|
||||||
Construction-in-progress
|
31,679
|
19,289
|
||||||
Total property and equipment
|
56,160
|
43,612
|
||||||
Less: accumulated depreciation and amortization
|
(18,831
|
) |
(16,499
|
) | ||||
Property and equipment, net
|
$ |
37,329
|
$ |
27,113
|
||||
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
Accrued research and development costs
|
$ |
9,659
|
$ |
26,480
|
||||
Accrued manufacturing costs
|
18,160
|
9,466
|
||||||
Accrued professional and consulting services
|
4,262
|
4,564
|
||||||
Accrued sales deductions
|
1,998
|
529
|
||||||
Other
|
483
|
319
|
||||||
Total accrued liabilities
|
$ |
34,562
|
$ |
41,358
|
||||
|
March 31, 2020
|
|
December 31, 2019
|
|
||||
Operating lease liabilities, current
|
$ |
2,691
|
$ |
1,866
|
||||
Other payable
|
30
|
30
|
||||||
Total other liabilities, current
|
$ |
2,721
|
$ |
1,896
|
||||
Year ending December 31,
|
Amount
|
|
||
2020 (nine months)
|
$ |
5,063
|
||
2021
|
6,750
|
|||
2022
|
6,750
|
|||
2023
|
31,406
|
|||
2024
|
29,156
|
|||
2025
|
26,906
|
|||
Total minimum payments
|
106,031
|
|||
Less amount representing interest
|
(29,531
|
) | ||
Less amount representing Paydown Fee
|
(1,500
|
) | ||
Long-term debt, gross
|
75,000
|
|||
Discount on notes payable
|
(1,418
|
) | ||
Accretion of Paydown Fee
|
106
|
|||
Long-term debt
|
$ |
73,688
|
||
|
Three Months Ended March 31,
|
|||||||
|
2020
|
|
2019
|
|
||||
ROU assets obtained in exchange for new operating lease upon adoption of ASC 842
|
$ |
—
|
$ |
14,177
|
||||
Cash paid for amounts included in the measurement of lease liabilities
|
1,153
|
1,117
|
||||||
Weighted-average remaining lease term of operating leases (in years)
|
10
|
8.7
|
||||||
Weighted-average discount rate of operating leases
|
8.7
|
% |
13.2
|
% |
Year ending December 31,
|
Amount
|
|
||
2020 (nine months)
|
$ |
5,878
|
(a)
|
|
2021
|
11,736
|
|||
2022
|
12,116
|
|||
2023
|
12,508
|
|||
2024
|
12,913
|
|||
2025
|
13,333
|
|||
Thereafter
|
61,035
|
|||
Total lease payments
|
129,519
|
|||
Less: Imputed interest
(b)
|
(45,190
|
) | ||
Present value of operating lease liabilities
|
$ |
84,329
|
||
(a)
|
Includes our lease payments of $7.1 million, which is partially offset by the expected receipt of the remaining tenant inducements of $1.2 million.
|
|
Number of
Options |
|
Weighted-
Average Exercise Price |
|
||||
Outstanding — December 31, 2019
|
3,573,860
|
$ |
36.24
|
|||||
Options granted
|
621,703
|
66.05
|
||||||
Options exercised
|
(33,937
|
) |
28.50
|
|||||
Options canceled
|
(27,905
|
) |
47.16
|
|||||
Outstanding — March 31, 2020
|
4,133,721
|
$ |
40.72
|
|||||
|
Three Months Ended
March 31,
|
|||||||
|
2020
|
|
2019
|
|
||||
Expected term (in years)
|
6.1
|
6.1
|
||||||
Volatility
|
69.6%-69.9%
|
71.6%-72.2%
|
||||||
Risk-free interest rate
|
1.4%-1.8%
|
2.5%-2.6%
|
||||||
Dividend yield
|
—
|
—
|
|
Number
of RSUs
|
|
Weighted-
Average
Grant Date Fair
Value
|
|
||||
Non-vested
units — December 31, 2019
|
1,848,772
|
$ |
49.19
|
|||||
RSUs granted
|
1,073,766
|
66.16
|
||||||
RSUs vested
|
(192,966
|
) |
45.68
|
|||||
RSUs forfeited
|
(38,413
|
) |
48.18
|
|||||
Non-vested
units — March 31, 2020
|
2,691,159
|
$ |
56.23
|
|||||
|
Three Months Ended
March 31,
|
|||||||
|
20
20
|
|
2019
|
|
||||
Research and development
|
$ |
5,350
|
$ |
4,023
|
||||
Selling, general and administrative
|
11,017
|
5,430
|
||||||
Total stock-based compensation expense
|
$ |
16,367
|
$ |
9,453
|
||||
|
Three Months Ended
March 31,
|
|||||||
|
2020
|
|
2019
|
|
||||
Options to purchase common stock
|
4,133,721
|
3,813,995
|
||||||
Restricted stock subject to future vesting
|
—
|
22,856
|
||||||
Restricted stock units
|
2,691,159
|
1,626,127
|
||||||
Total
|
6,824,880
|
5,462,978
|
||||||
Item 2.
|
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
|
|
Three Months Ended
March 31,
|
$ Change
|
|
% Change
|
|
|||||||||||
|
2020
|
|
2019
|
|
||||||||||||
|
(in thousands, except percentages)
|
|||||||||||||||
Product sales, net
|
$ |
14,118
|
$ |
—
|
$ |
14,118
|
*
|
|||||||||
Costs and operating expenses:
|
|
|
|
|
||||||||||||
Cost of sales
|
135
|
—
|
135
|
*
|
||||||||||||
Research and development
|
39,773
|
34,468
|
5,305
|
15
|
% | |||||||||||
Selling, general and administrative
|
47,662
|
18,055
|
29,607
|
164
|
% | |||||||||||
Total costs and operating expenses
|
87,570
|
52,523
|
35,047
|
67
|
% | |||||||||||
Loss from operations
|
(73,452
|
) |
(52,523
|
) |
(20,929
|
) |
40
|
% | ||||||||
Interest income
|
2,856
|
3,831
|
(975
|
) |
(25
|
)% | ||||||||||
Interest expenses
|
(2,314
|
) |
(181
|
) |
(2,133
|
) |
*
|
|||||||||
Other expenses, net
|
(116
|
) |
(50
|
) |
(66
|
) |
132
|
% | ||||||||
Net loss
|
$ |
(73,026
|
) | $ |
(48,923
|
) | $ |
(24,103
|
) |
49
|
% | |||||
* | Change is not meaningful |
|
Rebates,
co-payment
assistance, Medicare
Part D coverage gap,
product returns and
distributor fees
|
|
Prompt payment
discounts and
chargebacks
|
|
Total
|
|
||||||
Balances at December 31, 2019
|
$ |
529
|
$ |
113
|
$ |
642
|
||||||
Provision related to current period sales
|
1,543
|
339
|
1,882
|
|||||||||
Credit or payments made during the period
|
(75
|
) |
(280
|
) |
(355
|
) | ||||||
Balance at March 31, 2020
|
$
|
1,997
|
|
$
|
172
|
|
$
|
2,169
|
|
|||
• | employee-related expenses, which include salaries, benefits and stock-based compensation; |
• | expenses incurred under agreements with consultants, third-party research firms, manufacturing organizations for products not approved by the FDA, and investigative clinical trial sites that conduct research and development activities on our behalf; |
• | the costs related to production of clinical supplies, including fees paid to contract manufacturers; |
• | laboratory and vendor expenses related to the execution of nonclinical studies and clinical trials; |
• | payments upon achievement of certain clinical development and regulatory milestones in relation with license agreement; and |
• | facilities and other allocated expenses, which include expenses for rent and maintenance of facilities, depreciation and amortization expense and other supplies. |
|
Three Months Ended March 31,
|
$ Change
|
|
% Change
|
|
|||||||||||
|
2020
|
|
2019
|
|
||||||||||||
Costs incurred by development program:
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Oxbryta for the treatment of SCD
|
$ |
23,460
|
$ |
27,725
|
$ |
(4,265
|
) |
(15
|
)% | |||||||
Other preclinical programs
|
9,316
|
5,363
|
3,953
|
74
|
% | |||||||||||
Inclacumab for the treatment of SCD
|
6,997
|
1,380
|
5,617
|
407
|
% | |||||||||||
Total research and development expenses
|
$ |
39,773
|
$ |
34,468
|
$ |
5,305
|
15
|
% | ||||||||
• | employee-related expenses, which include salaries, benefits and stock-based compensation; |
• | fees to third party vendors providing customer support services; |
• | expenses incurred under agreements with consultants; and |
• | facilities and other allocated expenses, which include expenses for rent and maintenance of facilities, depreciation and amortization expense and other supplies. |
• | our ability to successfully commercialize Oxbryta, inclacumab and any other product candidates we may identify and develop in any territories; |
• | the manufacturing, selling, and marketing costs associated with the commercialization of Oxbryta and the potential commercialization of inclacumab and any other product candidates we may identify and develop, including the cost and timing of establishing or maintaining our sales and marketing capabilities in any territory(ies); |
• | the amount and timing of sales and other revenues from Oxbryta, inclacumab and any other product candidates we may identify and develop, including the sales price and the availability of adequate third-party reimbursement; |
• | the time and cost necessary to wind down our completed Phase 3 HOPE Study, to conduct and complete multiple ongoing studies (including our HOPE-KIDS 1 Study, Phase 3 HOPE-KIDS 2 Study and our OLE study in HOPE study countries); |
• | the time and cost necessary to conduct and complete any additional clinical studies required to pursue additional regulatory approvals for Oxbryta for SCD, including our Phase 3 HOPE-KIDS 2 Study (which is intended as our required confirmatory study to move from our current Subpart H approval to a full approval of Oxbryta) and any studies to support potential label expansions into younger SCD pediatric populations, or any other post-marketing studies for Oxbryta for SCD; |
• | the progress, data and results of clinical trials of Oxbryta; |
• | the progress, timing, scope and costs of our nonclinical studies, our clinical trials and other related activities, including our ability to enroll subjects in a timely manner for our ongoing and future clinical trials of Oxbryta, inclacumab or any other product candidate that we may identify and develop; |
• | the costs of obtaining clinical and commercial supplies of Oxbryta, inclacumab and any other product candidates we may identify and develop; |
• | our ability to advance our development programs, including for Oxbryta, inclacumab and any other potential product candidate programs we may identify and pursue, the timing and scope of these development activities, and the availability of approval for any of our other product candidates; |
• | our ability to successfully obtain any additional regulatory approvals from any regulatory authorities, and the scope of any such regulatory approvals, to market and sell Oxbryta, inclacumab and any other product candidates we may identify and develop in any territory(ies); |
• | the cash requirements of any future acquisitions or discovery of product candidates; |
• | the time and cost necessary to respond to technological and market developments; |
• |
the extent to which we may acquire or
in-license
other product candidates and technologies, and the costs and timing associated with any such acquisitions or
in-licenses;
|
• | our ability to attract, hire, and retain qualified personnel; and |
• | the costs of maintaining, expanding, and protecting our intellectual property portfolio. |
|
Three Months Ended
March 31,
|
|||||||
|
2020
|
|
2019
|
|
||||
Cash used in operating activities
|
$ |
(78,622
|
) | $ |
(40,316
|
) | ||
Cash provided by (used in) investing activities
|
91,126
|
(100,651
|
) | |||||
Cash provided by financing activities
|
784
|
22,446
|
||||||
Net increase (decrease) in cash, cash equivalents and restricted cash
|
$ |
13,288
|
$ |
(118,521
|
) | |||
Item 3.
|
Quantitative and Qualitative Disclosures about Market Risk
|
Item 4.
|
Controls and Procedures
|
Item 1.
|
Legal Proceedings
|
Item 1A.
|
Risk Factors.
|
• | the demonstrated efficacy and potential advantages of our drugs compared to alternative treatments; |
• | our ability to offer our drugs for sale at competitive prices; |
• | the availability of third-party coverage and adequate reimbursement; |
• | the convenience and ease of administration of our drugs compared to alternative current and future treatments; |
• | the willingness of the SCD or other target patient populations to try new therapies and of physicians to prescribe these therapies; |
• | the availability of our drugs and our ability to meet market demand, including a reliable supply for long-term chronic treatment; |
• | the strength of labeling, marketing and distribution support; |
• | the clinical indications and approved labeling for which the drug is approved, including labeling restrictions for drugs approved under Subpart H, such as Oxbryta; |
• | the prevalence and severity of any side effects and overall safety profile of the drug; and |
• | any restrictions on the use of the drug, including together with other medications. |
• | the burden of complying with complex and changing foreign regulatory, tax, accounting, compliance and legal requirements; |
• | different medical practices and customs in foreign countries affecting acceptance in the marketplace; |
• | import or export licensing requirements; |
• | longer accounts receivable collection times; |
• | longer lead times for shipping; |
• | language barriers for technical training; |
• | reduced protection of intellectual property rights in some foreign countries, and related prevalence of bioequivalent or generic alternatives to therapeutics; |
• | foreign currency exchange rate fluctuations; |
• | potential resource constraints, including with respect to patients’ ability to obtain reimbursement for our products in foreign markets; and |
• | the interpretation of contractual provisions governed by foreign laws in the event of a contract dispute. |
• | issue untitled or warning letters; |
• | impose civil or criminal penalties; |
• | impose injunctions; |
• | impose fines; |
• | impose additional specialized restrictions on the company’s activities and practices; |
• | suspend regulatory approval; |
• | suspend ongoing clinical trials; |
• | seek voluntary product recalls and impose publicity requirements; |
• | refuse to approve pending applications or supplements to approved applications submitted by us; |
• | impose restrictions on our operations, including closing our contract manufacturers’ facilities; or |
• | seize or detain products. |
• | commercialize Oxbryta and continue related clinical development, including winding down our recently completed Phase 3 HOPE Study and conducting (i) our Phase 2a HOPE-KIDS 1 Study of Oxbryta, (ii) our HOPE-KIDS 2 Study, which we intend to serve as our post-confirmatory study of Oxbryta in SCD (and any other post-marketing studies that may be required by regulatory authorities, if any), and (iii) any additional clinical trials of Oxbryta we may conduct now or in the future in SCD patients or for any other indications for Oxbryta, inclacumab or any other product candidates, if any; |
• | establish and maintain manufacturing and supply relationships with third parties that can provide adequate supplies (in amount and quality) of Oxbryta and inclacumab to support commercialization and further clinical development; |
• | seek and obtain additional regulatory and marketing approvals for Oxbryta for SCD, including for younger pediatric patient populations, or any potential approvals we may pursue; |
• | maintain a sales and marketing organization and enter into selected collaborations to commercialize Oxbryta for SCD or any other approved indication; |
• | maintain a medical affairs organization to advance our engagement with healthcare providers and stakeholders; |
• | advance our other programs, including inclacumab, through nonclinical and clinical development and commence development activities for any additional product candidates we may identify and pursue; and |
• | expand our organization to support our commercialization, research, development and medical activities and our operations as a public company. |
• | our ability to successfully commercialize Oxbryta, inclacumab and any other product candidates we may identify and develop in any territories; |
• | the manufacturing, selling, and marketing costs associated with the commercialization of Oxbryta and the potential commercialization of inclacumab and any other product candidates we may identify and develop, including the cost and timing of establishing or maintaining our sales and marketing capabilities in any territory(ies); |
• | the amount and timing of sales and other revenues from Oxbryta, inclacumab and any other product candidates we may identify and develop, including the sales price and the availability of adequate third-party reimbursement; |
• | the time and cost necessary to wind down our completed Phase 3 HOPE Study, to conduct and complete multiple ongoing studies (including our HOPE-KIDS 1 Study, Phase 3 HOPE-KIDS 2 Study and our OLE study in HOPE study countries; |
• | the time and cost necessary to conduct and complete any additional clinical studies required to pursue additional regulatory approvals for Oxbryta for SCD, including our Phase 3 HOPE-KIDS 2 Study (which is necessary to move from our current Subpart H approval to a full approval) and any studies to support potential label expansions into younger SCD pediatric populations, or any other post-marketing studies for Oxbryta for SCD; |
• | the progress, data and results of clinical trials of Oxbryta; |
• | the progress, timing, scope and costs of our nonclinical studies, our clinical trials and other related activities, including our ability to enroll subjects in a timely manner for our ongoing and future clinical trials of Oxbryta, inclacumab or any other product candidate that we may identify and develop; |
• | the costs of obtaining clinical and commercial supplies of Oxbryta, inclacumab and any other product candidates we may identify and develop; |
• | our ability to advance our development programs, including for Oxbryta, inclacumab and any other potential product candidate programs we may identify and pursue, the timing and scope of these development activities, and the availability of approval for any of our other product candidates; |
• | our ability to successfully obtain any additional regulatory approvals from any regulatory authorities, and the scope of any such regulatory approvals, to market and sell Oxbryta, inclacumab and any other product candidates we may identify and develop in any territory(ies); |
• | the cash requirements of any future acquisitions or discovery of product candidates; |
• | the time and cost necessary to respond to technological and market developments; |
• |
the extent to which we may acquire or
in-license
other product candidates and technologies, and the costs and timing associated with any such acquisitions or
in-licenses;
|
• | our ability to attract, hire, and retain qualified personnel; and |
• | the costs of maintaining, expanding, and protecting our intellectual property portfolio. |
• | sell, transfer or otherwise dispose of any of our business or property, subject to limited exceptions; |
• | make certain changes to our organizational structure; |
• | consolidate or merge with other entities or acquire other entities; |
• | incur additional indebtedness or create encumbrances on our assets; |
• | pay dividends, other than dividends paid solely in shares of our common stock, or make distributions on and, in certain cases, repurchase our stock; |
• | repay subordinated indebtedness; or |
• | make certain investments. |
• | we may not be able to demonstrate to the satisfaction of regulatory authorities (including the EMA) that Oxbryta, inclacumab or any other product candidates we may develop are safe and effective for any proposed indications; |
• | the FDA or comparable foreign regulatory authorities may disagree with our plans or expectations regarding the pathways and endpoints for approval, including the availability of accelerated approval, or the design or implementation of our nonclinical studies or clinical trials; |
• | the populations studied in our clinical programs may not be sufficiently broad or representative to assure safety or demonstrate efficacy in the full population for which we seek approval; |
• | the FDA or comparable foreign regulatory authorities may require additional nonclinical studies or clinical trials beyond those we anticipate; |
• | the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data and results from our nonclinical studies or clinical trials; |
• | the data and results collected from nonclinical studies or clinical trials of Oxbryta, inclacumab and any other product candidates that we may identify and pursue may not be sufficient to support the submission for regulatory approval; |
• | we may be unable to demonstrate to the FDA or comparable foreign regulatory authorities that a product candidate’s risk-benefit ratio for its proposed indication is acceptable; |
• | the FDA or comparable foreign regulatory authorities may find deficiencies with or fail to approve the manufacturing processes, test procedures and specifications, or facilities of third-party manufacturers with which we contract and rely on for all clinical and commercial supplies of Oxbryta, inclacumab and any other product candidates (if any); and |
• | the approval policies or regulations of the FDA or comparable foreign regulatory authorities may change in a manner that renders our development or manufacturing efforts insufficient for approval. |
• | delays or failures in reaching a consensus with regulatory agencies on study design, including clinical endpoints sufficient to support an approval decision; |
• | delays or failures to receive approval for conduct of clinical studies in one or more geographies, which could result in delays in enrollment and availability of data and results; |
• | delays or failures in reaching agreement on acceptable terms with a sufficient number of prospective contract research organizations, or CROs, and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and trial sites; |
• | delays in obtaining required Institutional Review Board, or IRB, or ethics committee approval for each clinical trial site; |
• | delays in recruiting a sufficient number of suitable patients to participate in our clinical trials; |
• | imposition of a clinical hold by any regulatory authority, including if imposed due to safety concerns after an inspection of our clinical trial operations or study sites; |
• | failure by our CROs, clinical sites, participating clinicians or patients, other third parties or us to adhere to clinical trial, regulatory or legal requirements; |
• | failure to perform in accordance with the FDA’s good clinical practices, or GCPs, or applicable regulatory requirements in other countries; |
• | delays in the testing, validation, manufacturing and delivery of sufficient quantities of Oxbryta or our product candidates or study related devices to the clinical sites and patients; |
• |
delays in having patients enroll or complete participation in a study in accordance with applicable protocols or protocol amendments or return for post-treatment
follow-up;
|
• | reduction in the number of participating clinical trial sites or patients, including by dropping out of a trial; |
• | failure to address in an adequate or timely manner any patient safety concerns that arise during the course of a trial; |
• | unanticipated costs or increases in costs of clinical trials of Oxbryta or our product candidates; |
• | the occurrence of serious adverse events or other safety concerns associated with Oxbryta or our product candidates; or |
• | changes in regulatory requirements and guidance that require amending or submitting new clinical protocols or obtaining additional IRB or other approvals to conduct or complete clinical studies of Oxbryta or our product candidates. |
• | reliance on the third party for regulatory compliance and quality assurance; |
• | the possible breach or termination of the manufacturing agreement by the third party or by us, including at a time that is costly or inconvenient for us; |
• |
the inability of the third party to satisfy our ordering requirements as to quality, quantity and/or price, including, without limitation, potential impact on supply chain due to the impact of public health risks, such as the recent spread of
COVID-19;
|
• |
the possible misappropriation of our proprietary information, including our trade secrets and
know-how;
and
|
• | the unwillingness of the third party to extend or renew terms with us when desired. |
• | the research methodology used may not be successful in identifying potential product candidates; |
• | competitors may develop alternatives that render our product candidates obsolete or less attractive; |
• | product candidates we develop may nevertheless be covered by third parties’ patents or other exclusive rights; |
• | the market for a product candidate may change during our program so that such a product may become unreasonable to continue to develop; |
• | a product candidate may on further study be shown to have harmful side effects, lack of potential efficacy or other characteristics that indicate it is unlikely to meet applicable regulatory criteria or remain reasonable to continue to develop; |
• | a product candidate may not be capable of being produced in commercial quantities at an acceptable cost, or at all; and |
• | a product candidate may not be accepted as safe and effective by patients, the medical community or third-party payors, if applicable. |
• | impairment of our business reputation; |
• | withdrawal of clinical trial participants; |
• | costs due to related litigation; |
• | distraction of management’s attention from our primary business; |
• | substantial monetary awards to patients or other claimants; |
• | increased warnings on product labels or additional restrictions imposed by regulatory authorities; |
• | the recall of Oxbryta or our product candidates; |
• | the inability to commercialize Oxbryta or our product candidates; and |
• | decreased demand for Oxbryta or our product candidates, if approved for commercial sale. |
• | restrictions and obligations imposed by privacy regulations, such as provisions under the General Data Protection Regulation 2016/679, known as GDPR, applicable to the collection and use of personal health data in the European Union; |
• | multiple, conflicting, and changing laws and regulations such as tax laws, export and import restrictions, employment laws, regulatory requirements, and any requirements to obtain other governmental approvals, permits, and licenses; |
• | failure by us to obtain and maintain regulatory approvals for the sale or use of our products in various countries; |
• | additional potentially relevant third-party patent rights; |
• | complexities and difficulties in obtaining protection for and enforcing our intellectual property; |
• | difficulties in staffing and managing our current and potential foreign operations; |
• |
complexities associated with managing multiple payor reimbursement regimes, government payors, or patient
self-pay
systems;
|
• | limits in our ability to penetrate international markets; |
• | financial risks, such as longer payment cycles, difficulty collecting accounts receivable, the impact of local and regional financial crises on demand and payment for our products, and exposure to foreign currency exchange rate fluctuations; |
• | natural disasters, political and economic instability, including wars, terrorism, and political unrest, outbreak of disease, boycotts, curtailment of trade, and other business restrictions; |
• | certain expenses including, among others, expenses for travel, translation, and insurance; and |
• | regulatory and compliance risks that relate to maintaining accurate information and control over sales and activities that may fall within the purview of the FCPA, its books and records provisions, or its anti-bribery provisions. |
• | comply with EMA or FDA regulations or similar regulations of comparable foreign regulatory authorities; |
• | provide accurate information to the FDA or EMA or comparable foreign regulatory authorities; |
• | comply with cGMP regulations and manufacturing standards that we have established and comply with applicable healthcare fraud and abuse regulations in the jurisdictions in which we operate; |
• | report financial information or data accurately; or |
• | disclose unauthorized activities to us. |
• | failure to successfully develop and commercialize Oxbryta, inclacumab or any other product candidates, including results relating to our launch and commercialization of Oxbryta in the United States; |
• | adverse results or delays in, or the halting of, our nonclinical studies or clinical trials, especially in our ongoing or future clinical program for Oxbryta for the treatment of SCD; |
• | reports of adverse events from our commercialization or clinical trials of Oxbryta, or from clinical trials of any other product candidates that we may develop; |
• | any delay in the review of, or potential action with respect to, our planned filing of an IND or NDA for inclacumab or for any other product candidates that we may develop and any adverse development or perceived adverse development with respect to the FDA’s regulatory review of such filing; |
• | adverse regulatory decisions affecting Oxbryta, inclacumab or any other product candidates we may develop, including any delay in or denial of potential approval in accordance with our plans and expectations; |
• | inability to obtain additional funding; |
• | failure to prosecute, maintain or enforce our intellectual property rights; |
• | disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; |
• | changes in laws or regulations applicable to Oxbryta or future products; |
• | inability to obtain adequate product supply for Oxbryta or our product candidates or the inability to do so at acceptable prices; |
• | introduction of new products, services or technologies by our competitors; |
• | failure to enter into or perform under strategic collaborations; |
• | failure to meet or exceed any financial projections that we or the investment community may provide; |
• | the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; |
• | announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us or our competitors; |
• | additions or departures of key scientific or management personnel; |
• | significant lawsuits, including patent or stockholder litigation; |
• | changes in the market valuations of similar companies; |
• | sales of our common stock by us or our stockholders in the future; |
• | trading volume of our common stock; and |
• | the other risks described in this “Risk Factors” section. |
• | our ability to successfully commercialize Oxbryta or any of our product candidates, if approved, and the timing and costs of our commercialization activities; |
• | the timing and cost of, and level of investment in, research and development activities relating to Oxbryta and our product candidates, which may change from time to time; |
• | the timing and success or failure of clinical trials for Oxbryta and our product candidates or competing product candidates, or any other change in the competitive landscape of our industry, including consolidation among our competitors or partners; |
• | our ability to obtain and maintain full regulatory approval for Oxbryta in the United States and to obtain regulatory approval of Oxbryta outside of the United States as well as regulatory approval for our product candidates, and the timing and scope of any such approvals we may receive; |
• | the cost of manufacturing Oxbryta and our product candidates, which may vary depending on the quantity of production and the terms of our agreements with manufacturers; |
• | our ability to attract, hire, train and retain qualified personnel; |
• | expenditures that we will or may incur to acquire or develop additional product candidates and technologies; |
• | the level of demand for Oxbryta and our product candidates, if approved, which may vary significantly; |
• | future accounting pronouncements or changes in our accounting policies; |
• | the risk/benefit profile, cost and reimbursement policies with respect to Oxbryta and our products candidates, if approved, and existing and potential future drugs that compete with Oxbryta and our product candidates; |
• | whether Oxbryta or any of our product candidates are subject to any compliance-related challenges or sanctions, or any intellectual-property related challenges; and |
• |
the changing and volatile U.S., European and global economic environments, including economic volatility as a result of the
COVID-19
pandemic.
|
• | authorize “blank check” preferred stock, which could be issued by our board of directors without stockholder approval and may contain voting, liquidation, dividend and other rights superior to our common stock; |
• | create a classified board of directors whose members serve staggered three-year terms; |
• | specify that special meetings of our stockholders can be called only by our board of directors, the chairperson of our board of directors, our chief executive officer or our president; |
• | prohibit stockholder action by written consent; |
• | establish an advance notice procedure for stockholder approvals to be brought before an annual meeting of our stockholders, including proposed nominations of persons for election to our board of directors; |
• | provide that our directors may be removed only for cause; |
• | provide that vacancies on our board of directors may be filled only by a majority of directors then in office, even though less than a quorum; |
• | specify that no stockholder is permitted to cumulate votes at any election of directors; |
• | expressly authorize our board of directors to modify, alter or repeal our amended and restated bylaws; and |
• | require supermajority votes of the holders of our common stock to amend specified provisions of our amended and restated certificate of incorporation and amended and restated bylaws. |
Item 2.
|
Unregistered Sales of Equity Securities and Use of Proceeds
|
a) |
Sales of Unregistered Securities
|
b) |
Use of Proceeds from our Initial Public Offering of Common Stock
|
c) |
Repurchases of Shares or of Company Equity Securities
|
Item 3.
|
Defaults Upon Senior Securities
|
Item 4.
|
Mine Safety Disclosures
|
Item 5.
|
Other Information
|
Item 6.
|
Exhibits
|
Exhibit
Number
|
|
|
Incorporated by Reference
|
Filed
Herewith
|
|
|||||||||||||||
Exhibit Description
|
Form
|
|
Date
|
|
Number
|
|
||||||||||||||
3.1
|
S-1/A
|
7/31/2015
|
3.2
|
|
||||||||||||||||
3.2
|
S-1/A
|
7/31/2015
|
3.4
|
|
||||||||||||||||
4.1
|
S-1/A
|
7/31/2015
|
4.1
|
|
||||||||||||||||
10.1#
|
8-K
|
1/9/2020
|
10.1
|
|
||||||||||||||||
10.2#
|
8-K
|
1/9/2020
|
10.2
|
|
||||||||||||||||
10.3#
|
S-8
|
1/23/2020
|
99.1
|
|
||||||||||||||||
10.4#
|
S-8
|
1/23/2020
|
99.3
|
|
||||||||||||||||
10.5#
|
—
|
—
|
—
|
X
|
||||||||||||||||
10.6#
|
—
|
—
|
—
|
X
|
||||||||||||||||
31.1
|
—
|
—
|
—
|
X
|
||||||||||||||||
31.2
|
—
|
—
|
—
|
X
|
||||||||||||||||
32.1*
|
—
|
—
|
—
|
X
|
||||||||||||||||
101.SCH
|
Inline XBRL Taxonomy Extension Schema Document
|
—
|
—
|
—
|
X
|
|||||||||||||||
101.CAL
|
Inline XBRL Taxonomy Extension Calculation Linkbase Document
|
—
|
—
|
—
|
X
|
|||||||||||||||
101.DEF
|
Inline XBRL Taxonomy Extension Definition Linkbase Document
|
—
|
—
|
—
|
X
|
|||||||||||||||
101.LAB
|
Inline XBRL Taxonomy Extension Label Linkbase Document
|
—
|
—
|
—
|
X
|
|||||||||||||||
101.PRE
|
Inline XBRL Taxonomy Extension Presentation Linkbase Document
|
—
|
—
|
—
|
X
|
|||||||||||||||
104
|
Cover Page Interactive Data File (formatted as inline XBRL with applicable taxonomy extension information contained in Exhibits 101.)
|
—
|
—
|
—
|
X
|
* |
The certification attached as Exhibit 32.1 that accompanies this Quarterly Report on Form
10-Q
is not deemed filed with the SEC and is not to be incorporated by reference into any filing of Global Blood Therapeutics, Inc. under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Form
10-Q,
irrespective of any general incorporation language contained in such filing.
|
# | Represents management compensation plan, contract or arrangement. |
|
|
Global Blood Therapeutics, Inc.
|
||||
Date: May 6, 2020
|
|
By:
|
/s/ Ted W. Love, M.D.
|
|||
|
|
|
Ted W. Love, M.D.
President and Chief Executive Officer
(Principal Executive Officer)
|
|||
Date: May 6, 2020
|
|
By:
|
/s/ Jeffrey Farrow
|
|||
|
|
|
Jeffrey Farrow
Chief Financial Officer
(Principal Financial Officer)
|
1 Year Global Blood Therapeutics Chart |
1 Month Global Blood Therapeutics Chart |
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