Blog: SCHOLAR ROCK HOLDING CORP : Termination of a Material Definitive Agreement, Regulation FD Disclosure, Other Events, Financial Statements and Exhibits (form 8-K) – Marketscreener.com

Item 1.02 Termination of a Material Definitive Agreement.

Scholar Rock Holding Corporation (the “Company”) previously entered into an Open
Market Sale AgreementSM (the “Sale Agreement”) with Jefferies LLC (the “Sales
Agent”) on March 9, 2021, pursuant to which the Company was able to issue and
sell from time to time in an “at-the-market” offering shares of the Company’s
common stock, par value $0.001 per share (the “Common Stock”). As of June 16,
2022, 500,000 shares of Common Stock have been issued and sold under the Sale
Agreement.

On June 16, 2022, the Company provided notice to the Sales Agent that it was
terminating the Sale Agreement, effective immediately. The Sale Agreement
provided for termination thereof by either party upon ten (10) calendar days’
prior written notice to the other party; however, the Sales Agent has waived
such ten (10) day notice requirement. The Company will not incur any early
termination penalties in connection with the termination of the Sale Agreement.

Item 7.01. Regulation FD Disclosure.

On June 17, 2022, the Company issued a press release announcing positive
24-month topline results from its TOPAZ Phase 2 clinical trial for apitegromab.
A copy of the press release is attached hereto as Exhibit 99.1.

On June 17, 2022, the Company will host a conference call and webcast at 8:30 am
ET to discuss the 24-month topline results from the TOPAZ Phase 2 clinical
trial. A copy of the presentation slides to be used by the Company during the
conference call and webcast is attached hereto as Exhibit 99.2. A live webcast
of the conference call may be accessed by visiting the Investors & Media section
of the Company’s website at http://investors.scholarrock.com.

The information in this report furnished pursuant to Item 7.01 and Exhibit 99.1
shall not be deemed “filed” for the purposes of Section 18 of the Securities
Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to
the liabilities of that section nor shall such information be deemed
incorporated by reference in any filing under the Securities Act of 1933, as
amended, or the Exchange Act, regardless of the general incorporation language
of such filing, except as shall be expressly set forth by specific reference in
such filing. It may only be incorporated by reference in another filing under
the Exchange Act or the Securities Act of 1933, as amended, if such subsequent
filing specifically references the information furnished pursuant to Item 7.01
and Exhibit 99.1 of this report.


Item 8.01. Other Events.


TOPAZ Phase 2 Clinical Trial Update

On June 17, 2022, the Company announced new data from the Phase 2 TOPAZ trial
extension period evaluating patient outcomes after 24-months of treatment, which
support sustained and continued improvement with apitegromab for non-ambulatory
patients with Types 2 and 3 SMA receiving an SMN therapy.

TOPAZ evaluated apitegromab across a broad age range (2-21 years) of patients
with Types 2 and 3 SMA. All 35 non-ambulatory patients (Cohorts 2 and 3) and 12
of 23 ambulatory patients (Cohort 1) were receiving nusinersen maintenance
therapy. The primary efficacy endpoint for the non-ambulatory population was
mean change from baseline in HFMSE. Additional endpoints included mean change
from baseline in RULM, an assessment specifically designed for upper limb
function in patients with SMA. The HFMSE is a validated measure for the
assessment of gross motor function in SMA, while the RULM is validated to
evaluate upper limb motor performance by evaluating tasks which correspond to
the ability to perform various everyday activities with their hands and arms.

For this 24-month evaluation, an observed case analysis was conducted, which
pooled all the non-ambulatory patients (Cohorts 2 and 3) and was based upon the
available data for given timepoints. This analysis population included patients
receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab
(inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year
2) and did not exclude any patients who had missed apitegromab doses due to
study site access restrictions from COVID-19.

Non-ambulatory patients (age range of 2 to 21 years old) with valid HFMSE
assessments had sizable, sustained gains in HFMSE scores at 24 months from
baseline (prior to first dose of apitegromab), while RULM scores continued to
increase at 24 months. The mean change from baseline results for non-ambulatory
patients showed:



                                 12-Month Data     24-Month Data    24-Month Data
                                                       Pooled       *excluding pts
                                                   non-ambulatory    w/scoliosis
                                                        pts            surgery
Mean Change from Baseline in       3.6 points        4.0 points       4.4 points
HFMSE (95% CI)                 (95% CI: 1.2, 6.0)  (95% CI: 1.5,    (95% CI: 2.0,
                                      N=32              6.5)             6.9)
                                                        N=29             N=28
Mean Change from Baseline in       1.3 points        1.9 points       2.3 points
RULM (95% CI)                  (95% CI: 0.2, 2.3)  (95% CI: 0.8,    (95% CI: 1.2,
                                      N=31              3.0)             3.4)
                                                        N=33             N=30



*Three patients in the non-ambulatory group underwent scoliosis surgery in year
2, which has been reported to negatively impact HFMSE scores for a considerable
period afterwards. This analysis excluded post-surgery data of these patients.

Dose response continued to be observed across the 24 months of apitegromab
administration based upon HFMSE scores and pharmacodynamic data (target
engagement as measured by serum latent myostatin concentrations), with signs
that that there may be further HFMSE increases as non-ambulatory patients
originally receiving the low dose switched to the high dose treatment.

Data at 24-months for ambulatory patients with Type 3 SMA (Cohort 1) suggest
stability of Revised Hammersmith Scale (RHS) scores in patients receiving 20
mg/kg of apitegromab and nusinersen. The mean RHS change from baseline at
24-months was -0.7 points (95% CI: -3.1, 1.7) for the apitegromab and nusinersen
subgroup (n=10) and -2.8 points (95% CI: -8.4, 2.8) for the apitegromab
monotherapy subgroup (n=11). A subset of individuals in Cohort 1(n=21) had RHS
improvements, as reflected by 42.9% (9/21) and 23.8% (5/21) of patients having
?1-point and ?3-point RHS increases from baseline at 24 months respectively.

Of the 55 patients who completed the 24-month TOPAZ extension period, 54 have
opted to continue treatment in the 36-month extension period.

Consistent with the 12-month safety data, no serious safety risks were
identified as part of the analysis of the cumulative 24-month data. The
incidence and severity of adverse events were consistent with the underlying
patient population and background therapy. The five most common
treatment-emergent adverse events (TEAEs) were headache, pyrexia, upper
respiratory tract infection, cough, and nasopharyngitis. No deaths or serious
adverse reactions have been observed with apitegromab. A total of 14 serious
TEAEs have been reported over the 24-month treatment period, all assessed by the
respective trial investigator as unrelated to apitegromab.

Item 9.01. Financial Statements and Exhibits.




(d) Exhibits



Exhibit
  No.      Description
  99.1       Press Release issued by Scholar Rock Holding Corporation, dated June
           17, 2022
  99.2       Presentation Slide Deck
104        Cover Page Interactive Data File (embedded within the Inline XBRL
           document)

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