Biogen: New Spinraza Data In Atrophy After Incomplete Response To Novartis' Treatment

 Biogen Inc 

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 announced new Spinraza (nusinersen) data that were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA.

RESPOND is an ongoing two-year, phase 4 open-label study to evaluate clinical outcomes and safety following treatment with Spinraza in infants and toddlers with spinal muscular atrophy (SMA) after treatment with Novartis AG's gene therapy 

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 Zolgensma (onasemnogene abeparvovec). 

Interim efficacy results at six months from 29 study participants treated with Spinraza show: 

Most participants demonstrated improved motor function measured by increased mean total Hammersmith Infant Neurological Examination Section 2 (HINE-2) score from baseline.

• • Participants with two SMN2 copies (n=24) improved by a mean of over 5 points on HINE-2
  • All participants with three SMN2 copies (n=3) improved; a mean change from baseline was not calculated due to the small number of participants.
• Most participants (25/27) with investigator-reported suboptimal motor function at baseline improved.

After a median of 230.5 days in the study, serious adverse events (AEs) were reported in 13/38 (34%) participants. 

No serious AEs were considered related to Spinraza or led to study withdrawal. 

William Blair writes that overall interim results suggest the added benefit of Spinraza treatment and support management commentary that sales of the $1.79 billion in FY22 franchise are stabilizing. 

The analyst says Spinraza will remain a significant topline contributor for Biogen and its development partner Ionis Pharmaceuticals Inc 

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 and bolster revenues.

It says that the ongoing results from RESPOND and the risdiplam switch study to high-dose Spinraza (ASCEND) study will be critical in creating a competitive dynamic whereby Spinraza could have a path to "return to growth."

https://www.benzinga.com/analyst-ratings/analyst-color/23/07/33094946/biogen-reports-new-spinraza-data-in-atrophy-patients-after-incomplete-response-to-n

AstraZeneca: Adverse Events In Late-Stage Lung Cancer Drug Trial

 AstraZeneca Plc 

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 reported high-level results from the TROPION-Lung01 Phase 3 trial of datopotamab deruxtecan (Dato-DXd) in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) treated with at least one prior therapy.

Dato-DXd demonstrated a statistically significant improvement for the dual primary endpoint of progression-free survival (PFS) compared to docetaxel, the current standard of care chemotherapy.

For the dual primary endpoint of overall survival (OS), the data were not mature, and an early trend was observed in favor of datopotamab deruxtecan versus docetaxel that did not meet the prespecified threshold for statistical significance at this interim analysis. 

All-grade interstitial lung disease was generally consistent with prior clinical trials, with the majority being low-grade. Some Grade 5 events were observed (Death related to AE). Probably investors are reacting to this.

Citing BI pharma analyst, Bloomberg noted that though progression-free survival benefit was reported as statistically significant, the lack of numerical detail — a minimum 2-3 month benefit was targeted — will lead to questions regarding clinical significance.

Datopotamab deruxtecan is a specifically engineered TROP2-directed DXd antibody-drug conjugate (ADC) jointly developed by AstraZeneca and Daiichi Sankyo DSKNY.

In June, the company shared updated results from the TROPION-Lung02 Phase Ib trial, showing additional enrolment and follow-up from the initial presentation that Dato-DXd plus Merck & Co Inc's Keytruda (pembrolizumab) with or without platinum-based chemotherapy demonstrated promising clinical activity and no new safety signals in both previously untreated or pretreated patients with advanced or metastatic NSCLC without actionable genomic alterations.

The doublet therapy demonstrated objective response rates of 57% and 50%, respectively, with a disease control rate of 91% across cohorts.

https://www.benzinga.com/general/biotech/23/07/33093239/astrazeneca-shares-under-pressure-on-some-adverse-events-in-late-stage-lung-cancer-drug-trial

Apple's ambitions to enter banking sector in jeopardy? Goldman wants to end partnership

 Little by little, Apple is entering the financial sector. The tech giant has already launched a credit card, a deferred payment system and a lucrative savings account. Now, however, the tech giant is in danger of losing an important partner from the banking sector. U.S. major bank Goldman Sachs wants to end cooperation with the iPhone maker.

Recap: Goldman Sachs announced in late 2022 that it planned to wind down its consumer business. At the time, the U.S. bank still seemed to attach great importance to its relationship with Apple.

• In fact, the bank extended its collaboration until the end of the decade. Through that collaboration, the bank supports financial services the Cupertino-based company plans to offer its customers, including deferred payments and .
• "It's a very, very solid partnership with a lot of opportunity," David Solomon, CEO of Goldman Sachs, said last October.

Today: According to the Wall Street Journal, Goldman Sachs wants to end its partnership with Apple.

• The bank is reportedly in talks with American Express to acquire the credit card it launched with Apple. The talks have reportedly been ongoing for several months.
• For now, no deal has been reached and there is nothing to suggest that Goldman Sachs will achieve its goal anytime soon, according to several people aware of the matter.
• Moreover, it would take some time for the transfer of business to become a reality. In any case, Apple would have to agree to it.

Ambitions scaled back

If Goldman Sachs says goodbye to the Apple Card (and credit cards in general) it would mean the end of its credit business aimed at consumers. The financial institution has already stopped making personal loans and also wants to sell Greensky, a provider of renovation loans that the bank bought in 2022, according to the .

• In doing so, Goldman Sachs would completely shelve its grand plans to become a full-service bank.
  • The loss of some $3 billion since 2020, caused by the switch to consumer operations, certainly played a determining role.
• Goldman Sachs seems to want to return to its first love: high-interest savings accounts.

Implications for Apple

An eventual end to the partnership between Apple and Goldman Sachs does not yet mean the end of the tech giant's financial services. As you could read above, the major bank is looking for a new partner to take over certain operations, including credit cards.

• Therefore, Apple's credit card, deferred payment service and savings account are not doomed to disappear.
• On the other hand, we can imagine that transferring Apple's partnership to another bank is likely to disrupt the tech giant's financial business and delay its plans in that area.
  • That may cause for consumers.
  • But it will also delay the rollout of the Apple Card in new countries.
  
  

https://www.marketscreener.com/quote/stock/APPLE-INC-4849/news/Are-Apple-s-ambitions-to-enter-the-banking-sector-in-jeopardy-Goldman-Sachs-wants-to-end-partnershi-44253668/

Reneo started at Buy by B of A

 https://finviz.com/quote.ashx?t=RPHM&p=d

Healthcare hits 2-yr M&A high, bucking trend

 According to data released by LSEG Deals Intelligence, the healthcare sector saw a 35% increase on last year’s deals, reaching $174.6 billion in the first half of 2023 – a two-year high that bucks the trend at a time when global dealmaking fell 38% to a post-pandemic low.

Indeed, healthcare dealmaking accounts for 14% of global M&A, although the number of deals is down 12% compared to this time last year. And M&A activity within the biotechnology, healthcare equipment, and health services sectors accounts for 85% of overall dealmaking within the sector as a whole.

Pfizer’s $42.1 billion acquisition of Seagen was the largest deal announced this year, while CVS Health’s $10.7 billion acquisition of Oak Street Health and Merck’s $10.3 billion purchase of Prometheus Biosciences were also in the top three. Merck’s deal was preceded by GSK’s decision to acquire Bellus Health for approximately $2 billion.

It was also within healthcare that the highest number of companies went from small cap to large cap in the FTSE Russell US Indexes Reconstitution in June, with six healthcare companies among the 22 companies moving up from the Russell 2000 to the Russell 1000.

Matt Toole, Director at LSEG Deals Intelligence, commented that “[h]uge amounts of cash reserves amassed during the coronavirus pandemic have been deployed to fuel growth and boost investor confidence.”

He caveated, however that, “[m]ore broadly, economic pressures have continued to dampen the global dealmaking environment, with fears of recession and rising interest rates biting.”

This can be seen in the number of deals in worldwide dealmaking having decreased 11% so far this year – a three-year low. Nonetheless, second quarter-to-date 2023 M&A activity increased 23% compared to the first quarter, which had ranked as the slowest first quarter since the first quarter of a decade ago in 2013.

Certainly, the pharma industry had also been seeing a slowdown in major acquisitions. Now, as these investments become more regular, it is thought that the industry could enter into another period of consolidation and expansion within their chosen niches.

Meanwhile, in May 2023, CVS Health decided to shut down its clinical trials business, just two years after launching it in the midst of the COVID-19 pandemic. Its acquisition of primary care provider Oak Street Health and home care company Signify Health for $8 billion resulted in a downgrade of its earnings forecasts for the year, which likely had a bearing on its plans for the clinical unit, the phased exit from which is expected by 31st December 2024.

https://pharmaphorum.com/news/healthcare-hits-2-yr-ma-high-bucking-trend

NLS to Proceed with Phase 3 for Mazindol ER for Treatment of Narcolepsy

  NLS Pharmaceutics Ltd. (Nasdaq:NLSP)(Nasdaq:NLSPW) ("NLS" or the "Company"), a Swiss clinical-stage biopharmaceutical company focused on the discovery and development of innovative therapies for patients with rare and complex central nervous system disorders, today announced that the U.S. Food and Drug Administration (FDA) has reviewed the full protocol for the NLS-1031 study, part of the Phase 3 program for Mazindol ER, called AMAZE. In addition, the Company is pleased to announce that the Phase 3 clinical trial protocol to evaluate the safety and efficacy of Mazindol ER in patients with narcolepsy type 1 received approval from the independent Institutional Review Board ("IRB"). The AMAZE Program will encompass two almost-identical double-blind Phase 3 studies (N=50 each) investigating Mazindol ER versus placebo in adult patients with narcolepsy commencing this summer at multiple sites exclusively in the U.S.

Based on the FDA's recommendations, both Phase 3 trials will measure the weekly cataplexy episodes as the primary endpoint over 8 weeks of treatment and excessive daytime sleepiness as a secondary objective using the Patient-Reported Outcomes Measurement Information System (PROMIS-SRI) and the Epworth Sleepiness Scale (ESS).

https://www.biospace.com/article/releases/nls-pharmaceutics-to-proceed-with-phase-3-clinical-program-amaze-for-mazindol-er-for-the-treatment-of-narcolepsy-following-fda-review-and-irb-approval-of-the-full-study-protocol/

MannKind shares fall after fire that may delay clinical trial

 Shares of MannKind Corp. (MNKD) fell 1.5% premarket on Monday after the biopharma company said a fire at a German manufacturing facility may delay the planned initiation later this year of a phase 2/3 clinical study of MNKD-101, a treatment for nontuberculous myobacterial lung disease. The facility was producing clofazimine inhalation solution, the investigational product that MannKind is developing as MNKD-101, MannKind said in a Securities and Exchange Commission filing Monday. Clinical supplies of MNKD-101 will be delayed an estimated three to six months, according to the filing. The company is "evaluating several mitigation strategies and will update the projected timing for this clinical study at a later date," the filing said.

https://www.morningstar.com/news/marketwatch/20230703151/mannkind-shares-fall-after-fire-that-may-delay-clinical-trial